Archive for April, 2019

EMERGENCY RESPONSE FOR A CRYONICS INSTITUTE MEMBER WHO IS IN CRITICAL CONDITION OR LEGALLY DECEASEDCritical Condition

If the person is in critical condition or hospice care, collect all required legal documents relating to suspension and confirm plans for standby and transport as soon as possible.

If the person has been legally pronounced dead and is currently a CI member, then entirely cover and cool his or her head with bags of crushed ice.Do NOT place ice on a Member until there has been a legal pronouncement of death -- attempt to obtain a pronouncement as soon as possible.

*Requirements

In order to fund a suspension at the $28,000 fee, a member must sign all required CI documents themselves with their signature witnessed by a notary. The membership documents and funding must be in place for a minimum of two weeks otherwise, the suspension fee will cost $35,000 and other nonmember restrictions may apply.

In either situation, contact the Cryonics Institute as soon as possible at:

Also see the listing below for exit codes for countries that do not use 00

Calling from Outside North America: + 1 586 791 5961 where +represents the exit code for your country,see http://www.howtocallabroad.com/codes.html "

Excerpt from:
CI MEMBER | Cryonics Institute

How do Stem Cellsfunction?Stem cells have the capacity to migrate to injured tissues, a phenomenon calledhoming. This occurs by injury or disease signals that are released from the distressed cells and tissue. Once stem cells arrive,they dock on adjacent cells to commence performing their job to repair the problem.

Stem cells serve as a cell replacementwhere they change into the required cell type such as a muscle cell, bone orcartilage. This is ideal for traumaticinjuries and many orthopedic indications.

They do not express specific human leukocyte antigens (HLAs) which helpthem avoid the immune system. Stemcells dock on adjacent cells and release proteins called growth factors, cytokines and chemokines. These factors help control many aspects of the healing and repairprocess systemically.

Stem cells control the immune system and regulate inflammation which is a keymediator of disease, aging, and is ahallmark of autoimmune diseases such as rheumatoid arthritis and multiple sclerosis.

They help to increase new blood vesselformation so that tissues receive proper blood flow and the correct nutrients needed to heal as in stroke, peripheral arterydisease and heart disease.

Stem cells provide trophic support forsurrounding tissues and help hostendogenous repair. This works wellwhen used for orthopedics. In case ofdiabetes, it may help the remaining beta cells in the pancreas to reproduce orfunction optimally.

As CSN research evolves, the field ofregenerative medicine and stem cells offers the greatest hope for those suffering from degenerative diseases, conditions for which there is currently no effective treatment or conditions that have failed conventional medical therapy.

Stem cell treatment is a complex process allowing us to harvest the bodys own repair mechanism to fight against degeneration, inflammation and general tissue damage. Stem cells are cells that can differentiate into other types of tissue to restore function and reduce pain.

Adult stem cells are found in abundance in adipose (fat) tissue, where more than 5million stem cells reside in every gram. These stem cells are called adult mesenchymal stem cells.

Our medical doctors extract stromal vascular fraction (SVF) from your own body to provide treatment using your very own cells. This process is calledautologous mesenchymal stem celltherapy. Our multi-specialty team deploys SVF under an institutional review board (IRB). This is an approved protocol that governs investigational work and the focus is to maintain safety of autologous use of SVF for various degenerative conditions.

How do we perform the stem cell treatment?Our procedure is very safe and completed in a single visit to our clinic. On the day of treatment, our physicians inject a localanaesthetic and harvest approximately 60 cc (2 oz.) of stromalvascular fraction (SVF) from under the skin of your flanks or abdomen. The extracted SVF is then refined in a closed system using strictCSN protocols to produce pure stromalvascular fraction (SVF). SVF containsregenerative cells including mesenchymal and hematopoietic stem cells, macrophages, endothelial cells, immune regulatory cells, and important growth factors that facilitate your stem cell activity. CSN technology allows us to isolate high numbers of viable stem cells that we can immediately deploy directly into a joint, trigger point, and/or byintravascular infusion. Specific deployment methods have been developed that are unique for each condition being treated.

During the refinement process, thesubcutaneous harvested cells andtheir connecting collagen matrix willbe separated, leaving purified free stem cells. About half of the SVF will be pure stem cells, while the remainder will be acombination of other regenerative cellsand growth factors. Before the SVF isre-injected into your body during the final part of the process we perform a qualityand quantity test which will examine the cell count and viability.

Perfecting the stem cell treatmentOur team records cell numbers and viability so that we can gain a better understanding of what constitutes a successful treatment. Although it is not yet possible to predict what number of cells that will be recovered in a harvest, it is very important that we know the total cell count and cell viability. It is only with this data that we will beginto understand why treatments are verysuccessful, only slightly successful orunsuccessful.

While vigilant about patient safety, we are also learning and sharing with the CSN data bank about which diseases respond best and which deployment methods are most effective with over 80 other clinics.

This data collection from all over the world makes the Cell Surgical Network the worlds largest regenerative medicineclinical research organization.

Network physicians have the opportunity to share their data, as well as their clinical experiences, thus helping one anotherto achieve higher levels of scientificunderstanding and optimizing medical protocols.

Injecting into thevascular system and/ora jointWe will administer the stem cell treatment with two methods:

The belief is that for many degenerative joint conditions IV and intra-articulardeployment is superior because each of these conditions have a local pathology and a central pathology. The local resident stem cell population has been working very hard to repair the damage and over the course of time these stem cells have become worn out, depleted and slowly die. This essentially causes a state of stem cell depletion. When we inject our mix of stem cells, cytokines and growth factors (known as SVF)inflammation is decreased and theregenerative process improved.

The stem cells that we have injected will then bring the level of stem cells closer to the normal level, thus restoring the natural balance and allow the body to heal itself.

Caplan et al, The MSC: An Injury Drugstore, DOI 10.1016/j .stem.2011.06.008

How long does it last?Many studies have shown the healing and regenerative ability of stem cells. Forexample, a study in World Journal of Plastic Surgery (Volume 5[2]; May 2016) followed a woman with knee arthritis. Before and after analysis of MRI images confirmed new growth of cartilage tissue. Unlike steroids, lubricants, and other injectable treatments, stem cells actually repair damaged tissue.

As published in Experimental andTherapeutic Medicine (Volume 12[2]; August 2016), numerous studies with hundreds of patients showed continuous improvement of arthritis for two years. Patients showed improvement three months after a single treatment and they continued to show improvement for two full years. This is why stem cells are often referred to as regenerative medicine.

No one can guarantee results for this or any other treatment. Outcomes will vary from patient to patient. Each potential patient must be assessed individually to determine the potential for optimum results from this regenerative therapy. To learn more about stem cell therapy, please contact us by clicking here or calling our clinic at 604-708-CELL (604-708-2355).

Like Loading...

Read the original here:
Vancouver Stem Cell Treatment Centre | Stem Cells

April 30, 2019

Children born with a rare genetic disorder called X-linked severe combined immunodeficiency (X-SCID) dont have a functioning immune system. As a result, they cant fight off infections. Without treatment, an infant with X-SCID will usually die within the first year or two of life.

The best option for treatment of newly diagnosed infants with X-SCID has been stem-cell transplantation from a genetically matched sibling. But less than a quarter of children with X-SCID have a matched donor available. For those without a matched donor, standard treatment has been a half-matched bone marrow transplant from a parent. But most infants receiving this type of transplant only have part of their immune system, called T lymphocytes, restored. These infants will need lifelong injections of protective antibodies. In addition, as they grow into young adulthood, they may have chronic medical problems that affect growth, nutrition, and quality of life.

To develop a better approach to fix the immune systems of children with X-SCID, researchers have used gene therapy to alter patients own blood stem cells. An engineered virus brings a healthy copy of the gene into the stem cells to replace the mutated gene that causes the disease.

Early results from trials of gene therapy for X-SCID resulted in life-saving correction of T lymphocytes. But similar to bone marrow transplant from a parent, the immune restoration was incomplete. In addition, in those first gene therapy studies, almosta third of the children developed leukemia. The approach accidentally stimulated cells to grow uncontrollably. In later studies, improved design of the engineered virus didnt cause cancer, but also didnt fully restore a healthy immune system.

In 2010, Dr. Harry Malech of NIHs National Institute of Allergy and Infectious Diseases (NIAID) and Dr. Brian Sorrentino of St. Jude Childrens Research Hospital reported a new and safer version of gene therapy for X-SCID. They designed a harmless engineered virus (called a lentivector) that could deliver genes into cells without activating other genes that can cause cancer. Before the altered stem cells were returned to their bodies, patients were given low doses of the chemotherapy drug busulfan. This made it easier for the new stem cells to grow in the bone marrow. In young adults and children treated at the NIH Clinical Center, the new therapy proved to be both safe and effective at restoring the full range of immune functions.

Based on this work, a team led by Dr. Ewelina Mamcarz of St. Jude Childrens Research Hospital began treatment in 2015 of newly diagnosed infants with X-SCID using the lentivector and busulfan. The work was funded in part by NHLBI. The team described the treatment of eight infants with the disorder on April 18, 2019, in the New England Journal of Medicine.

By 3 to 4 months after infusion of the repaired stem cells, 7 of the 8 infants had normal levels of multiple types of immune cells in their blood. The last infant required a second stem-cell infusion, after which his immune-cell levels rose to a normal range.

The infants new immune systems were able to fight off infections that the researchers had detected before the gene therapy. Four of the eight discontinued immune-system boosting medications that theyd previously needed. Of those four, three developed antibodies in response to vaccination, indicating a fully functional immune system.

A year and a half after gene therapy, all children were healthy and growing normally.

The broad scope of immune function that our gene therapy approach has restored to infants with X-SCID as well as to older children and young adults in our continuing study at NIH is unprecedented, Malech says.

The researchers will continue to follow the participants over time. They plan to track how the childrens immune systems develop and look for any late side effects.

References:Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. PMID: 30995372.

Funding:NIHs National Institute of Allergy and Infectious Diseases (NIAID); National Heart, Lung, and Blood Institute (NHLBI); and National Cancer Institute (NCI); American Lebanese Syrian Associated Charities; California Institute of Regenerative Medicine; and Assisi Foundation of Memphis.

Link:
Gene therapy reverses rare immune disorder | National ...

Global Gene Therapy Market report offers clients the most efficient and dependable insight into the Gene Therapy market, ranging across different major players.

Pune, India April 29, 2019

The Global Gene Therapy Industry, 2014-2024 Market Research Report is a professional and in-depth study on the current state of the Global Gene Therapy Market with a focus on the market conditions. The report provides key statistics on the market status of the Gene Therapy manufacturer and is a valuable source of guidance and direction for companies and individuals interested in the industry. Firstly, the report provides a basic overview of the industry including its definition, applications and manufacturing technology. Then, the report explores the international major industry players in detail.

The report includes Global key players of Gene Therapy Market in which at least 7 companies are included:SangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.For complete companies list, please ask for sample pages

To know more about Gene Therapy market Report request sample@ https://www.reportsnreports.com/contacts/requestsample.aspx?name=2160345

This report lists major product type of Gene Therapy market in Global Market.Ex vivoIn Vivo

This report focuses on the status and outlook for key applications and End users are also listed:CancerMonogenicInfectious diseaseCardiovascular diseaseOther

Get access to the complete report on Gene Therapy market spread across 139 pages and different major key players available @ https://www.reportsnreports.com/contacts/discount.aspx?name=2160345

The Gene Therapy Market report initially provides a basic overview of the industry that covers definition, applications and manufacturing technology post which the report explores into the international players in the market. In this part, the report presents the company profile, product specifications, capacity, production value and 2014-2019 market shares for each company. The report depicts the global market of Gene Therapy Industry including capacity, production, production value, cost and profit, supply and demand and import-export. The total market is further divided by company, by country, and by application or type for the competitive landscape analysis. The report also estimates 2019-2024 market development trends of Gene Therapy Industry. Analysis of upstream raw materials, downstream demand, and current market dynamics is also carried out. In the end, the report makes some important proposals for a new project of Gene Therapy Industry before evaluating its feasibility. Overall, the report provides an in-depth insight of 2014-2024 Global Gene Therapy Industry covering all important parameters.

Purchase a Copy of this Report at https://www.reportsnreports.com/purchase.aspx?name=2160345

Reasons to Purchase this Report:Estimates 2019-2024 Gene Therapy market development trends with the recent trends and SWOT analysisMarket dynamics scenario, along with growth opportunities of the market in the years to comeMarket segmentation analysis including qualitative and quantitative research incorporating the impact of economic and policy aspectsRegional and country level analysis integrating the demand and supply forces that are influencing the growth of the market.Market value (USD Million) and volume (Units Million) data for each segment and sub-segmentCompetitive landscape involving the market share of major players, along with the new projects and strategies adopted by players in the past five yearsComprehensive company profiles covering the product offerings, key financial information, recent developments, SWOT analysis, and strategies employed by the major market players1-year analyst support, along with the data support in excel format

Browse an In-depth TOC and list of tables and figure available in the report.

About Us:ReportsnReports.com is your single source for all market research needs. Our database includes 500,000+ market research reports from over 95 leading global publishers & in-depth market research studies of over 5000 micro markets. With comprehensive information about the publishers and the industries for which they publish market research reports, we help you in your purchase decision by mapping your information needs with our huge collection of reports. Feel free to Call us at +1 888 391 5441 or Email us at sales@reportsandreports.com

Contact Info:Name: Vishal KalraOrganization: ReportsnReportsAddress: Tower B5, office 101, Magarpatta SEZ, Hadapsar, PunePhone: +1 888 391 5441Website: https://www.reportsnreports.com/reports/2160345-gene-therapy-market-insights-2019-global-and-chinese-analysis-and-forecast-to-2024.html

This content is not produced by Reuters Editorial News. It is produced by VC NewsNetwork. For content enquiry, please reach us at

Originally posted here:
Gene Therapy Market Emerging Trends, Growth and New ...

Menopause hormone therapy and your heart

Are you taking or considering hormone therapy to treat bothersome menopause symptoms? Understand potential risks to your heart and whether hormone therapy is right for you.

Long-term hormone replacement therapy used to be routinely prescribed for postmenopausal women to relieve hot flashes and other menopause symptoms. Hormone replacement therapy was also thought to reduce the risk of heart disease.

Before menopause, women have a lower risk of heart disease than men do. But as women age, and their estrogen levels decline after menopause, their risk of heart disease increases. In the 1980s and 1990s, experts advised older women to take estrogen and other hormones to keep their hearts healthy.

However, hormone replacement therapy or menopause hormone therapy, as it's now called has had mixed results. Many of the hoped-for benefits failed to materialize for large numbers of women. The largest randomized, controlled trial to date actually found a small increase in heart disease in postmenopausal women using combined (both estrogen and progestin) hormone therapy. For women in this study using estrogen alone, there was no increased risk in heart disease.

Other studies suggest that hormone therapy, especially estrogen alone, may not affect or may even decrease the risk of heart disease when taken early in postmenopausal years. However, these studies can be confusing to interpret into practice, since study outcomes can be affected by many factors, such as the ages of the study participants, the time elapsed since menopause and the duration of hormone therapy use. Continued research will help doctors more clearly understand the relationship between menopause hormone therapy and heart disease.

If you're having a tough time with symptoms of menopause but worry about how hormone therapy will affect your heart, talk with your doctor to put your personal risk into perspective. Consider these points:

Menopause hormone therapy risks may vary depending on:

If you've already had a heart attack, menopause hormone therapy is not for you. If you already have heart disease or you have a history of blood clots, the risks of hormone therapy have been clearly shown to outweigh any potential benefits.

Talk with your doctor about these strategies to reduce the risks of menopause hormone therapy:

Women of all ages should take heart disease seriously. Among U.S. women, nearly 1 in 3 deaths each year is due to heart and blood vessel (cardiovascular) disease.

Most healthy women who are within five years of menopause can safely take short-term hormone therapy for menopausal symptoms without significantly increasing the risk of heart disease. If you experience classic menopausal symptoms, including intolerable hot flashes, vaginal dryness or insomnia, talk to your doctor about how you can relieve troublesome symptoms without putting your health at risk.

.

See the article here:
Menopause hormone therapy and your heart - Mayo Clinic

Today, induced pluripotent stem cells are mostly used to understand how certain diseases occur and how they work. By using IPS cells, one can actually study the cells and tissues affected by the disease without causing unnecessary harm to the patient.For example, its extremely difficult to obtain actual brain cells from a living patient with Parkinsons Disease. This process is even more complicated if you want to study the disease in its early stages before symptoms begin presenting themselves.

Fortunately, with genetic reprogramming, researchers can now achieve this. Scientists can do a skin biopsy of a patient with Parkinsons disease and create IPS cells. These IPS cells can then be converted into neurons, which will have the same genetic make-up as the patients own cells.

Because of IPS cells, researchers can now study conditions like Parkinsons disease to determine what went wrong and why. They can also test out new treatment methods in hopes of protecting the patient against the disease or curing it after diagnosis.

In addition, IPS cells have also been looked to as a way to replace cells that are often destroyed by certain diseases. However, there is still research to be done here.

Read more:
What Are Induced Pluripotent Stem Cells? - Stem Cell: The ...

There are many ways in which human stem cells can be used in research and the clinic. Studies of human embryonic stem cells will yield information about the complex events that occur during human development. A primary goal of this work is to identify how undifferentiated stem cells become the differentiated cells that form the tissues and organs. Scientists know that turning genes on and off is central to this process. Some of the most serious medical conditions, such as cancer and birth defects, are due to abnormal cell division and differentiation. A more complete understanding of the genetic and molecular controls of these processes may yield information about how such diseases arise and suggest new strategies for therapy. Predictably controlling cell proliferation and differentiation requires additional basic research on the molecular and genetic signals that regulate cell division and specialization. While recent developments with iPS cells suggest some of the specific factors that may be involved, techniques must be devised to introduce these factors safely into the cells and control the processes that are induced by these factors.

Human stem cells are currently being used to test new drugs. New medications are tested for safety on differentiated cells generated from human pluripotent cell lines. Other kinds of cell lines have a long history of being used in this way. Cancer cell lines, for example, are used to screen potential anti-tumor drugs. The availability of pluripotent stem cells would allow drug testing in a wider range of cell types. However, to screen drugs effectively, the conditions must be identical when comparing different drugs. Therefore, scientists must be able to precisely control the differentiation of stem cells into the specific cell type on which drugs will be tested. For some cell types and tissues, current knowledge of the signals controlling differentiation falls short of being able to mimic these conditions precisely to generate pure populations of differentiated cells for each drug being tested.

Perhaps the most important potential application of human stem cells is the generation of cells and tissues that could be used for cell-based therapies. Today, donated organs and tissues are often used to replace ailing or destroyed tissue, but the need for transplantable tissues and organs far outweighs the available supply. Stem cells, directed to differentiate into specific cell types, offer the possibility of a renewable source of replacement cells and tissues to treat diseases including maculardegeneration, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis, and rheumatoid arthritis.

Figure 3. Strategies to repair heart muscle with adult stem cells. Click here for larger image.

2008 Terese Winslow

For example, it may become possible to generate healthy heart muscle cells in the laboratory and then transplant those cells into patients with chronic heart disease. Preliminary research in mice and other animals indicates that bone marrow stromal cells, transplanted into a damaged heart, can have beneficial effects. Whether these cells can generate heart muscle cells or stimulate the growth of new blood vessels that repopulate the heart tissue, or help via some other mechanism is actively under investigation. For example, injected cells may accomplish repair by secreting growth factors, rather than actually incorporating into the heart. Promising results from animal studies have served as the basis for a small number of exploratory studies in humans (for discussion, see call-out box, "Can Stem Cells Mend a Broken Heart?"). Other recent studies in cell culture systems indicate that it may be possible to direct the differentiation of embryonic stem cells or adult bone marrow cells into heart muscle cells (Figure 3).

Cardiovascular disease (CVD), which includes hypertension, coronary heart disease, stroke, and congestive heart failure, has ranked as the number one cause of death in the United States every year since 1900 except 1918, when the nation struggled with an influenza epidemic. Nearly 2,600 Americans die of CVD each day, roughly one person every 34 seconds. Given the aging of the population and the relatively dramatic recent increases in the prevalence of cardiovascular risk factors such as obesity and type 2 diabetes, CVD will be a significant health concern well into the 21st century.

Cardiovascular disease can deprive heart tissue of oxygen, thereby killing cardiac muscle cells (cardiomyocytes). This loss triggers a cascade of detrimental events, including formation of scar tissue, an overload of blood flow and pressure capacity, the overstretching of viable cardiac cells attempting to sustain cardiac output, leading to heart failure, and eventual death. Restoring damaged heart muscle tissue, through repair or regeneration, is therefore a potentially new strategy to treat heart failure.

The use of embryonic and adult-derived stem cells for cardiac repair is an active area of research. A number of stem cell types, including embryonic stem (ES) cells, cardiac stem cells that naturally reside within the heart, myoblasts (muscle stem cells), adult bone marrow-derived cells including mesenchymal cells (bone marrow-derived cells that give rise to tissues such as muscle, bone, tendons, ligaments, and adipose tissue), endothelial progenitor cells (cells that give rise to the endothelium, the interior lining of blood vessels), and umbilical cord blood cells, have been investigated as possible sources for regenerating damaged heart tissue. All have been explored in mouse or rat models, and some have been tested in larger animal models, such as pigs.

A few small studies have also been carried out in humans, usually in patients who are undergoing open-heart surgery. Several of these have demonstrated that stem cells that are injected into the circulation or directly into the injured heart tissue appear to improve cardiac function and/or induce the formation of new capillaries. The mechanism for this repair remains controversial, and the stem cells likely regenerate heart tissue through several pathways. However, the stem cell populations that have been tested in these experiments vary widely, as do the conditions of their purification and application. Although much more research is needed to assess the safety and improve the efficacy of this approach, these preliminary clinical experiments show how stem cells may one day be used to repair damaged heart tissue, thereby reducing the burden of cardiovascular disease.

In people who suffer from type1 diabetes, the cells of the pancreas that normally produce insulin are destroyed by the patient's own immune system. New studies indicate that it may be possible to direct the differentiation of human embryonic stem cells in cell culture to form insulin-producing cells that eventually could be used in transplantation therapy for persons with diabetes.

To realize the promise of novel cell-based therapies for such pervasive and debilitating diseases, scientists must be able to manipulate stem cells so that they possess the necessary characteristics for successful differentiation, transplantation, and engraftment. The following is a list of steps in successful cell-based treatments that scientists will have to learn to control to bring such treatments to the clinic. To be useful for transplant purposes, stem cells must be reproducibly made to:

Also, to avoid the problem of immune rejection, scientists are experimenting with different research strategies to generate tissues that will not be rejected.

To summarize, stem cells offer exciting promise for future therapies, but significant technical hurdles remain that will only be overcome through years of intensive research.

Previous|VII. What are the potential uses of human stem cells and the obstacles that must be overcome before these potential uses will be realized?|Next

Original post:
Stem Cell Basics VII. | stemcells.nih.gov

Adult stem cells are the bodys toolbox, called into action by normal wear and tear on the body, and when serious damage or disease attack. Researchers believe that adult stem cells also have the potential, as yet untapped, to be tools in medicine. Scientists and physicians are working towards being able to treat patients with their own stem cells, or with banked donor stem cells that match them genetically.

Grown in large enough numbers in the lab, then transplanted into the patient, these cells could repair an injury or counter a diseaseproviding more insulin-producing cells for people with type 1 diabetes, for example, or cardiac muscle cells to help people recover from a heart attack. This approach is called regenerative medicine.

A number of challenges must be overcome before the full therapeutic potential of adult stem cells can be realized. Scientists are exploring practical ways of harvesting and maintaining most types of adult stem cells. Right now, scientists do not have the ability to grow the cells in the amounts needed for treatment. More work is also needed to find practical ways to direct the different kinds of cells to where theyre needed in the body, preferably without the need for surgery or other invasive methods.

Research in all aspects of adult stem cells and their potential is underway at Childrens Hospital Boston. Realizing that potential will require years of research, but promising strides are being made.

Read this article:
Why are Adult Stem Cells Important? Boston Children's ...

By: Ian Murnaghan BSc (hons), MSc - Updated: 12 Sep 2015| *Discuss

One of the biggest hurdles in stem cell research involves the use of embryonic stem cells. While these stem cells have the greatest potential in terms of their ability to differentiate into many different kinds of cells in the human body, they also bring with them enormous ethical controversies. The extraction of embryonic stem cells involves the destruction of an embryo, which upsets and outrages some policy makers and researchers as well as a number of public members. Not only that, but actually obtaining them is a challenge in itself and one that has become more difficult in places such as the United States, where policies have limited the availability of embryonic stem cells for use.

Although researchers have focused on harnessing the power of adult stem cells, there have still been many difficulties in the practical aspects of these potential therapies. In an ideal world, we would be able to use embryonic stem cells without destroying an embyro. Now, however, this ideal hope may actually have some realistic basis. In recent medical news, there has been important progress in the use of embryonic stem cells.

There are still many more tests and research that must be conducted to verify the safety and reliability of the procedure but it is indeed hopeful that funding can now increase for stem cell research. If you are an avid reader of health articles, you will probably be able to stay up-to-date on the latest developments related to this medical news. This newest research into embryonic stem cells holds promise and hope for appeasing the controversy around embryonic stem cell use and allowing for research to finally move forward with fewer challenges and controversies. For those who suffer from one of the many debilitating diseases and conditions that stem cell treatments may help or perhaps cure one day, this is welcome news.

You might also like...

Share Your Story, Join the Discussion or Seek Advice..

Time for Change - 12-Sep-15 @ 12:54 PM

Ellie - 25-Apr-14 @ 6:02 PM

Title:

(never shown)

Excerpt from:
Creating Embryonic Stem Cells Without Embryo Destruction

Thank you for visiting PromoCodeWatch on your hunt for Life Extension promo codes. We hope that one of our 13 Life Extension coupons for April, 2019 helped you save on your purchase. You can rest assured that weve searched everywhere to find all available Life Extension deals. This includes tracking mentions of Life Extension coupons on social media outlets like Twitter and Instagram, visiting blogs and forums related to Life Extension products and services, and scouring top deal sites for the latest Life Extension promo codes.

When shopping online for Life Extension products and services, it is a wise decision to visit PromoCodeWatch before checking out. Weve saved our visitors an average of 2 million dollars a year, many of which never knew Life Extension discounts were so easily available until visiting our site. Now that you are in the know, bookmark this page and check PromoCodeWatch before all of your online purchases.

If you seek more than just Life Extension coupon codes, we provide coupons and discounts for over 50,000 brands and retailers. Check out these related stores, or visit our complete directory to search our database of over one million coupon codes.

See the rest here:
70% Off Life Extension Promo Codes | Top 2019 Coupons @PromoCodeWatch

Good news: you heard wrong! With CI, the minimum fee for cryopreservation at CI (which includes vitrification perfusion and long term storage) is $28,000 a one-time fee, due at time of death. And though the fee can be paid in cash, usually a member has a life insurance policy made that pays the amount to CI upon death. A term life insurance policy in the amount of the minimum fee often costs around $30 per month for a person starting their policy in good health at middle age. Funding at a higher level can be used to defray additional costs, including transportation (which is not included in CIs base fee) or even a cryonics standby team to perform rapid cooling and cardiopulmonary support upon pronouncement of death.

Advice from an insurance professional is recommended before selecting a policy.

A person who wishes to become a Lifetime CI Member can make a single membership payment of $1,250 with no further payment required. If a new member would rather pay a smaller amount up front, in exchange for funding a slightly higher cryopreservation fee later on ($35,000), he or she can join with a $75 initiation fee, and pay annual dues of only $120, which are also payable in quarterly installments of $35. (And such a dues-paying member can upgrade to Lifetime Membership at any time, saving $7,000 and future any dues.) Members at a distance may have to pay local funeral director fees and transportation costs to Michigan to be cryopreserved. These payments are not made to CI, and are not included in the figures outlined above.

Take a look at our Membership FAQ and the membership application forms to find out more. And if you've got any questions, or want to talk about making special arrangements? Give us a call at (586) 791-5961 or drop us an email at CIHQ@aol.com. We're more than happy to help.

Visit link:
Frequently Asked Questions | Cryonics Institute

About Conference

About Conference

In continuation to 1st successful past scientific meeting, 2nd EuroSciCon Conference on Cell and Gene Therapy will be held on April 08-09, 2019 on Paris, France.

EuroSciCon suggests every single person to attend "Genetherapy 2019 in the midst of April 08-09, 2019 at Paris, France which merges brief keynote introductions, speaker talks, Exhibitions, Symposia, Workshops.

Genetherapy 2019 will gather world-class educators, researchers, analysts, Molecular Biologist, Gene therapists , Young Researchers working in the related fields to consider, exchange views and their experiences before an extensive worldwide social occasion of individuals. The social gathering warmly welcomes Presidents, CEO's, Delegates and present day experts from the field of Gene therapy and Public wellbeing and other pertinent organization positions to take an interest in these sessions, B2B get together and board talks. The assembly of this event will be revolving around the topic Exploring the possibilities and breakthroughs in cell and gene therapy.

EuroSciCon is the longest running independent life science events company with a predominantly academic client base. Our multi professional and multi-specialty approach creates a unique experience that cannot be found with a specialist society or commercially. EuroSciCon are corporate members of the following organizations: Royal Society of Biology, IBMS Company.

This global meeting gives the chance to Molecular Biologist, Gene Therapists, young researchers, specialists and analysts throughout the world to assemble and take in the most recent advances in the field of Cell and Gene Therapy and to trade innovative thoughts and encounters.

2 days of scientific exchange

100+ abstracts submitted

20+ scientific sessions

50+ worldwide professionals

80+ healthcare experts

Genetherapy 2019 is the yearly gathering directed with the help of the Organizing Committee Members and individuals from the Editorial Board of the supporting cell and Gene therapy related journals.

Reason to attend?

Genetherapy 2019 is relied upon to give young researchers and scientists a platform to present their revolutions in the field of Cell and Gene Therapy. This conference invites Presidents, CEO's, Delegates and present day specialists from the field of Cell and Gene Therapy and Public wellbeing and other pertinent organization positions to take an interest in this sessions, B2B get together and board talks.

About City:

Paris, the world's most popular city destination, has plenty of must-see places but make sure you spend at least a day strolling off the beaten path, as this is the only way to discover the real Paris: a lively cosmopolitan but undeniably French city.

The city is known for its cafe culture and designer boutiques along the Rue du Faubourg Saint-Honor. Paris is the city of love, inspiration, art and fashion. It has a population of more than 2million people and is divided into 20 districts. Paris has a lot of interesting architecture and museums to offer; among them the famous tourist place to visit is the Eiffel Tower. A significant number of the acclaimed roads and city building areas structures where changed by Haussmann and Napoleon III (Charles Louis Napoleon Bonaparte). The lanes where made much wider, places and squares where fabricated and the structures totally modified. Paris has a nickname called La Ville-Lumiere. The famous places to visit in Paris are Notre Dame Cathedralwhich is Roman Catholic Cathedral situated in the eastern half of the city, Louvre Museum which is located at the heart of Paris , Champs Elysees which is a Arc of Triumph, Montmartre which is a hill located at the north of Paris and its height is 130 metre, it is best known White Domed Basilica of the sacred heart at the top, Quartier Latin which is called the famous private garden located on the left bank of the seine around the Sorbonne, Disneyland Paris which is located 32 km from central Paris , it has two theme parks Disneyland and Walt Disney studios.

Track 1: Cell Science Research

Cell Science Research examines cells their physiological properties, their structure, the organelles they contain, interchanges with their condition, their life cycle, division, end and cell work.

Related Keyword:

Gene Therapy Conferences|Cell and Gene Therapy Conferences|International Gene Therapy Conferences|Top cell and gene Therapy research conferences

Track 2: Cell & Gene Therapy

Quality treatment is described as a plan of approaches that modify the announcement of a man's characteristics or repair bizarre characteristics. Each system incorporates the association of a specific nucleic destructive (DNA or RNA). Nucleic acids are frequently not taken up by cells, henceforth exceptional transporters; implied 'vectors' are required. Vectors can be of either mainstream or non-viral nature however Cell treatment is portrayed as the association of living whole cells into the patient for the treatment of a disease. The start of the cells can be from a comparable individual (autologous source) or from another individual (allogeneic source). Cells can be gotten from undifferentiated life forms, for instance, bone marrow or induced pluripotent central microorganisms (iPSCs), rethought from skin fibroblasts or adipocytes. Youthful microorganisms are associated with respect to bone marrow transplantation particularly. Distinctive methods incorporate the utilization of basically create cells, isolated in vitro (in a dish) from essential microorganisms.

Related Keyword:

Cell and Gene Therapy Conferences|Molecular Biology Conferences|Top Molecular Biology Conferences

Track 3: Regenerative Medicine

Regenerative Medicine implies a social affair of biomedical approaches to manage investigate and clinical applications which are away to supplant or "recouping" human cells, tissues or organs to restore or set up conventional limits which were vexed on account of afflictions. The field of Regenerative medication has pulled in much thought as it holds the assurance of recuperating hurt tissues and organs in the body by supplanting hurt tissue or by strengthening the body's own repair segments to patch hurt tissues or organs. It in like manner may enable analysts to create tissues and organs in the lab and safely install them inside the body. Regenerative courses of action subsequently can be a dynamic progress in the field of therapeutic administrations.

Related Keyword:

Genetic Regenerative Medicine Conferences|Top Cell and Gene therapy Conferences|International Cell Conferences|Top Cell Therapy Conferences

Track 4: Immunotherapy

Due to rapidly pushing field of tumor immunology as of late, there has been age of a couple of new procedures for treating development called Immunotherapies. Immunotherapy is a sort of treatment that extends the nature of safe response against tumors either by enabling the activities of specific sections of safe structure or by checking signals conveyed by illness cells that cover safe responses. A couple of sorts of immunotherapy are also called as biologic treatment or biotherapy. Late movements in development immunotherapies have given new supportive systems. These consolidate tumor-related macrophages as treatment centers in oncology, in-situ commencement of platelets with checkpoint inhibitors for post-careful development immunotherapy, safe checkpoint blockade and related endocrinopathies and some more.

Related Keyword :

Top Immunotherapy Conferences|International Genetic Immunotherapy Conferences|Top Gene Therapy Conferences

Track 5 : Genetics and stem cell biology

An undifferentiated mass of cell in a multicellular animal which is prepared for offering rise to uncertain number of cells of a comparable sort, and from which certain diverse sorts of cell rise by detachment. Undifferentiated life forms can isolate into specific cell creates. The two describing characteristics of an undifferentiated cell are endless self-restoration and the ability to isolate into a specific adult. There are two critical classes of youthful microorganisms: pluripotent that can end up being any cell in the adult body, and multipotent that is kept to transforming into a more limited masses of cells.

Related Keyword:

Stem Cell Biology Conferences|Genetic Cell Biology Conferences|Top Stem Cell Biology Conferences|International Stem Cell Therapy Conferences

Track 6: Epigenetics

The examination of changes in living creatures caused by alteration of quality verbalization instead of adjustment of the inherited code itself. Epigenetics are unfaltering heritable characteristics that can't be cleared up by changes in DNA progression.

Related Keyword:

International Epigenetic Conferences|Top Gene therapy Conferences|Genomic Conferences

Track 7: Human Genomics

The human genome is the total arrangement of nucleic corrosive groupings for people, encoded as DNA inside the 23 chromosome combines in cell cores and in a little DNA particle found inside individual mitochondria. Human genomes incorporate both protein-coding DNA genes and noncoding DNA

Related Keyword:

International human genomics conference|Top Genomic Conferences|Genome Sequencing Conferences

Track 8: Next Generation Sequencing

Deoxyribonucleic destructive, for the most part known as DNA, contains the outlines of life. Inside its structures are the codes required for the party of proteins and non-coding RNA these sub-nuclear mechanical assemblies impact all the natural systems that make and care forever. By understanding the game plan of DNA, examiners have had the ability to outline the structure and limit of proteins and what's more RNA and have gotten a cognizance of the essential purposes behind ailment. Front line Sequencing (NGS) is an able stage that has enabled the sequencing of thousands to countless iotas in the meantime. This compelling device is evolving fields, for instance, redid medicine, inherited infections, and clinical diagnostics by offering a high throughput elective with the capacity to progression various individuals meanwhile.

Related Keyword:

Gene therapy Conferences|Cell And Genetic Sequencing Conferences|Top Next Generation Sequencing Conferences

Track 9: Gene Editing and CRISPR Based Technologies

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) Technology is a champion among the most fit yet clear mechanical assembly for genome changing. It urges and empowers investigators to easily change DNA groupings and modify quality limits. It has various potential applications that join helping innate disseminates, treating and keeping the spread of diseases and improving yields. CRISPR broadly used as CRISPR-Cas9 where CRISPRs are particular stretches out of DNA and Cas9 is the protein which is an aggravate that exhibitions like a few nuclear scissors, fit for cutting DNA strands. The assurance of CRISPR advancement anyway raises moral stresses as it isn't 100% compelling. Regardless, the progression of CRISPR-Cas9 has disturbed the designed science industry these days, being a clear and great quality changing device.

Related Keyword:

Top Gene Editing And CRISPR Conferences|Genomic Editing Technology Conferences|Cell Microbiology Conferences

Track 10: Proteomics

Proteomics is the broad scale examination of proteomes. A proteome is a course of arrangement of proteins made in a living being, structure, or regular setting. We may imply, for instance, the proteome of a creature composes (for example, Homo sapiens) or an organ (for example, the liver). The proteome isn't relentless; it fluctuates from cell to cell and changes after some time. To some degree, the proteome reflects the key transcriptome. Regardless, protein activity (regularly reviewed by the reaction rate of the systems in which the protein is incorporated) is similarly changed by various components despite the verbalization level of the appropriate quality.

Related Keyword:

International Proteomics Conferences|Top Microbiology Conferences|Cell Microbiology Conferences

Track 11: Viral Gene Therapy

Customary strategies for quality treatment fuse transfection. It twisted up clearly inefficient and confined fundamentally in view of movement of value into right now duplicating cells invitro. Quality treatment utilizes the transport of DNA into cells by techniques for vectors, for instance, natural nanoparticles or viral vectors and non-viral systems. The Several sorts of contaminations vectors used as a piece of value treatment are retrovirus, adenovirus, disease adeno-related and herpes simplex contamination. While other recombinant viral vector structures have been delivered, retroviral vectors remain the most surely understood vector system for quality treatment traditions and most prominent application on account of their certain significance as the essential vectors made for compelling quality treatment application and the soonest phases of the field of value treatment.

Related Keyword:

Gene therapy Conferences|Cell And Genetic Sequencing Conferences|Top Next Generation Sequencing Conferences

Track 12: Cell Therapy of Cardiovascular Disorders

Cardiovascular contaminations have transformed into a growing clinical issue all around. the other test in the treatment of the cardiovascular disease is cell transplantation or cell cardiomyoplasty. Exceptional ischaemic harm and relentless cardiomyopathies incite unending loss of cardiovascular tissue and in the end heart disillusionment. Force medications wide mean to tighten the over the top changes that happen when harm and to cut back shot segments of vas diseases. Regardless, they don't improve the patient's close to home fulfillment or the figure more than coordinate. Unmistakable sorts of undifferentiated living beings have been used for primary microorganism treatment.

Related Keyword:

Genomic Conferences International Human|Top Genomic Conferences|Genome Sequencing Conferences

Track 13: Regulatory and Safety Aspects of Cell and Gene Therapy

Cell treatment things require a combination of prosperity examinations. Comparable living being and quality things are heterogeneous substances. There are a few zones that particularly ought to be tended to as it is extremely not the same as that of pharmaceuticals. These range from making group consistency, thing soundness to thing prosperity, quality and sufficiency through pre-clinical, clinical examinations and displaying endorsement. This review plots the present headings/administers in US, EU, India, and the related challenges in making SCBP with highlight on clinical point.

Related Keyword:

Gene therapy Conferences|Cell and Gene Therapy Conferences|International Gene therapy Conferences|Top Genetic research conference

Track 14: Markets & Future Prospects for Cell & Gene Therapy

The immense number of associations related with cell treatment has extended development incredibly in the midst of the past couple of years. More than 500 associations have been recognized to be locked in with cell treatment and 305 of these are profiled 291 co-tasks. Of these associations, 170 are related with fundamental microorganisms. The Profiles of 72 academic establishments in the US related with cell treatment close by their business facilitated efforts. Allogeneic development with in excess of 350 clinical preliminaries is prepared to order the commercialization of cell medicines in publicize. Advance R&D in cell and quality treatment is depended upon to bloom given the normally based purposes of intrigue.

Related Keyword:

Gene therapy Conferences International Human|Top Gene therapy Conferences|Genome Sequencing Conferences

Track 15: Gene therapy for Diseases

Gene therapy is the addition of particular genes at some particular locales into a person's cells or tissues to treat an illness, in which the inadequate or non-working quality is then supplanted with the working quality.

Related Keyword:

Gene therpy Conferences|Cell and Gene Therapy Conferences|International cell and Gene therapy Conferences|Top Gene Therapy research conferences

Track 16: Stem Cell therapy

Stem Cell therapyis the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use. Recent studies are going on for the treatment ofSpinal cordinjury as well. Thus,Stem cell therapyhas a great scope in future as well.

Related Keyword:

Stem Cell Biology Conferences|Genetic Cell Biology Conferences|Top Stem Cell Biology Conferences|International Stem Cell Therapy Conferences

Track 17: Gene Editing

Gene Editing is where the defective gene is being expelled or supplanted from the genome, in order to change the imperfect type of quality to a working structure. Different methods, for example, gene substitution, gene knock out, gene knock down are utilized for this reason. Additionally, site coordinated mutagenesis has been broadly utilized for gene altering purposes.

Related Keyword:

International Gene Therapy Conferences|Top Gene Editing And CRISPR Conferences|Genomic Editing Technology Conferences

Visit link:
International Cell and Gene therapy Conferences | Gene ...

En Espaol

The term stem cell by itself can be misleading. In fact, there are many different types of stem cells, each with very different potential to treat disease.

Stem CellPluripotentEmbryonic Stem CellAdult Stem CelliPS CellCancer Stem Cell

By definition, all stem cells:

Pluripotent means many "potentials". In other words, these cells have the potential of taking on many fates in the body, including all of the more than 200 different cell types. Embryonic stem cells are pluripotent, as are induced pluripotent stem (iPS) cells that are reprogrammed from adult tissues. When scientists talk about pluripotent stem cells, they mostly mean either embryonic or iPS cells.

Embryonic stem cells come from pluripotent cells, which exist only at the earliest stages of embryonic development. In humans, these cells no longer exist after about five days of development.

When isolated from the embryo and grown in a lab dish, pluripotent cells can continue dividing indefinitely. These cells are known as embryonic stem cells.

James Thomson, a professor in the Department of Cell and Regenerative Biology at the University of Wisconsin, derived the first human embryonic stem cell lines in 1998. He now shares a joint appointment at the University of California, Santa Barbara, a CIRM-funded institution.

Adult stem cells are found in the various tissues and organs of the human body. They are thought to exist in most tissues and organs where they are the source of new cells throughout the life of the organism, replacing cells lost to natural turnover or to damage or disease.

Adult stem cells are committed to becoming a cell from their tissue of origin, and cant form other cell types. They are therefore also called tissue-specific stem cells. They have the broad ability to become many of the cell types present in the organ they reside in. For example:

Unlike embryonic stem cells, researchers have not been able to grow adult stem cells indefinitely in the lab, but this is an area of active research.

Scientists have also found stem cells in the placenta and in the umbilical cord of newborn infants, and they can isolate stem cells from different fetal tissues. Although these cells come from an umbilical cord or a fetus, they more closely resemble adult stem cells than embryonic stem cells because they are tissue-specific. The cord blood cells that some people bank after the birth of a child are a form of adult blood-forming stem cells.

CIRM-grantee IrvWeissman of the Stanford University School of Medicine isolated the first blood-forming adult stem cell from bone marrow in 1988 in mice and later in humans.

Irv Weissman explains the difference between an adult stem cell and an embryonic stem cell (video)

An induced pluripotent stem cell, or iPS cell, is a cell taken from any tissue (usually skin or blood) from a child or adult and is genetically modified to behave like an embryonic stem cell. As the name implies, these cells are pluripotent, which means that they have the ability to form all adult cell types.

Shinya Yamanaka, an investigator with joint appointments at Kyoto University in Japan and the Gladstone Institutes in San Francisco, created the first iPS cells from mouse skin cells in 2006. In 2007, several groups of researchers including Yamanaka and James Thomson from the University of Wisconsin and University of California, Santa Barbara generated iPS cells from human skin cells.

Cancer stem cells are a subpopulation of cancer cells that, like stem cells, can self-renew. However, these cellsrather than growing into tissues and organspropagate the cancer, maturing into the many types of cells that are found in a tumor.

Cancer stem cells are a relatively new concept, but they have generated a lot of interest among cancer researchers because they could lead to more effective cancer therapies that can treat tumors resistant to common cancer treatments.

However, there is still debate on which types of cancer are propelled by cancer stem cells. For those that do, cancer stem cells are thought to be the source of all cells that make up the cancer.

Conventional cancer treatments, such as chemotherapy, may only destroy cells that form the bulk of the tumor, leaving the cancer stem cells intact. Once treatment is complete, cancer stem cells that still reside within the patient can give rise to a recurring tumor. Based on this hypothesis, researchers are trying to find therapies that destroy the cancer stem cells in the hopes that it truly eradicates a patients cancer.

John Dick from the University of Toronto first identified cancer stem cells in 1997. Michael Clarke, then at the University of Michigan, later found the first cancer stem cell in a solid tumor, in this case, breast cancer. Now at Stanford University School of Medicine, Clarke and his group have found cancer stem cells in colon cancer and head and neck cancers.

Find out More:

Catriona Jamieson talks about therapies based on cancer stem cells (4:32)

Stanford Publication: The true seeds of cancer

UCSD Publication: From Bench to Bedside in One Year: Stem Cell Research Leads to Potential New Therapy for Rare Blood Disorder

Updated 2/16

Visit link:
Stem Cell Key Terms | California's Stem Cell Agency

Plan your Stem Cell Therapy in India with Tour2India4Health Consultants

Stem cell therapy in India is performed by highly skilled and qualified doctors and surgeons in India. Our hospitals have state-of-art equipment that increase success rate of stem cell treatment in India. Tour2India4Health is a medical value provider that offers access to the stem cell therapy best hospitals in India for patients from any corner of the world. We offer low cost stem cell therapy at the best hospitals in India.

Stem cells have the ability to differentiate into specific cell types. The two defining characteristics of a stem cell are perpetual self-renewal and the ability to differentiate into a specialized adult cell type.

Serving as a sort of repair system, they can theoretically divide without limit to replenish other cells for as long as the person or animal is still alive. When a stem cell divides, each "daughter" cell has the potential to either remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

There are three classes of stem cells i.e totipotent, pluripotent and multipotent (also known as unipotent).

Many different terms are used to describe various types of stem cells, often based on where in the body or what stage in development they come from. You may have heard the following terms:

Adult Stem Cells or Tissue-specific Stem Cells: Adult stem cells are tissue-specific, meaning they are found in a given tissue in our bodies and generate the mature cell types within that particular tissue or organ. It is not clear whether all organs, such as the heart, contain stem cells. The term adult stem cells is often used very broadly and may include fetal and cord blood stem cells.

Fetal Stem Cells: As their name suggests, fetal stem cells are taken from the fetus. The developing baby is referred to as a fetus from approximately 10 weeks of gestation. Most tissues in a fetus contain stem cells that drive the rapid growth and development of the organs. Like adult stem cells, fetal stem cells are generally tissue-specific, and generate the mature cell types within the particular tissue or organ in which they are found.

Cord Blood Stem Cells: At birth the blood in the umbilical cord is rich in blood-forming stem cells. The applications of cord blood are similar to those of adult bone marrow and are currently used to treat diseases and conditions of the blood or to restore the blood system after treatment for specific cancers. Like the stem cells in adult bone marrow, cord blood stem cells are tissue-specific.

Embryonic Stem Cells: Embryonic stem cells are derived from very early embryos and can in theory give rise to all cell types in the body. While these cells are already helping us better understand diseases and hold enormous promise for future therapies, there are currently no treatments using embryonic stem cells accepted by the medical community.

Induced Pluripotent Stem Cells (IPS cells): In 2006, scientists discovered how to reprogram cells with a specialized function (for example, skin cells) in the laboratory, so that they behave like an embryonic stem cell. These cells, called induced pluripotent cells or IPS cells, are created by inducing the specialized cells to express genes that are normally made in embryonic stem cells and that control how the cell functions.

Embryonic stem cells are derived from the inner cell mass of a blastocyst: the fertilized egg, called the zygote, divides and forms two cells; each of these cells divides again, and so on. Soon there is a hollow ball of about 150 cells called the blastocyst that contains two types of cells, the trophoblast and the inner cell mass. Embryonic stem cells are obtained from the inner cell mass.

Stem cells can also be found in small numbers in various tissues in the fetal and adult body. For example, blood stem cells are found in the bone marrow that give rise to all specialized blood cell types. Such tissue-specific stem cells have not yet been identified in all vital organs, and in some tissues like the brain, although stem cells exist, they are not very active, and thus do not readily respond to cell injury or damage.

Stem cells can also be obtained from other sources, for example, the umbilical cord of a newborn baby is a source of blood stem cells. Recently, scientists have also discovered the existence of cells in baby teeth and in amniotic fluid that may also have the potential to form multiple cell types. Research on these cells is at a very early stage.

Stem cell therapy is the use of stem cells to treat certain diseases. Stem cells are obtained from the patients own blood bone marrow, fat and umbilical cord tissue or blood. They are progenitor cells that lead to creation of new cells and are thus called as generative cells as well.

The biological task of stem cells is to repair and regenerate damaged cells. Stem cell therapy exploits this function by administering these cells systematically and in high concentrations directly into the damaged tissue, where they advance its self-healing. The process that lies behind this mechanism is largely unknown, but it is assumed that stem cells discharge certain substances which activate the diseased tissue. It is also conceivable that single damaged somatic cells, e.g. single neurocytes in the spinal cord or endothelium cells in vessels, are replaced by stem cells. Most scientists agree that stem cell research has great life-saving potential and could revolutionize the study and treatment of diseases and injuries.

Stem cell therapy is useful in certain degenerative diseases like

If stem cell therapy is an option, a detailed treatment plan is prepared depending on the type of treatment necessary. Once the patient has consented to the treatment plan, an appointment is scheduled for bone marrow extraction. Please note that this is a minimally invasive surgical procedure, so it is important that patients do not take any blood-thinning medication in the ten days prior to the appointment. It is necessary for each patient to consult their own doctor before discontinuing this type of medication.

The treatment procedure include:

Bone Marrow Extraction: Bone marrow is extracted from the hip bone by the physicians. This procedure normally takes around 30 minutes. First, local anesthetic is administered to the area of skin where the puncture will be made. Then, a thin needle is used to extract around 150-200 ml of bone marrow. The injection of local anesthetic can be slightly painful, but the patient usually does not feel the extraction of bone marrow.

Isolation, Analysis and Concentration of the Stem Cells in the Laboratory: The quality and quantity of the stem cells contained in the collected bone marrow are tested at the laboratory. First, the stem cells are isolated. Then a chromatographical procedure is used to separate them from the red and white blood corpuscles and plasma. The sample is tested under sterile conditions so that the stem cells, which will be administered to the patient, are not contaminated with viruses, bacteria or fungi. Each sample is also tested for the presence of viral markers such as HIV, hepatitis B and C and cytomegalia. The cleaned stem cells are counted and viability checks are made. If there are enough viable stem cells, i.e. more than two million CD34+ cells with over 80 percent viability, the stem cell concentrate is approved for patient administration.

Stem Cell Implantation: The method of stem cell implantation depends on the patient's condition. There are four different ways of administering stem cells:

Intravenous administration:

It is important to understand that while stem cell therapy can help alleviate symptoms in many patients and slow or even reverse degenerative processes, it does not work in all cases. Based on additional information, patient's current health situation and/or unforeseen health risks, the medical staff can always, in the interest of the individual patient, propose another kind of stem cell transplantation or in exceptional situations cancel the treatment.

Allogeneic Stem Cell Transplantation: Allogeneic stem cell transplantation involves transferring the stem cells from a healthy person (the donor) to your body after high-intensity chemotherapy or radiation. It is helpful in treating patients with high risk of relapse or who didnt respond to the prior treatment. Allogeneic stem cell transplant cost in India is comparatively less when contrasted with alternate nations.

Autologous Stem Cell Transplant: Patients own blood-forming stem cells are collected and then it is treated with high doses of chemotherapy. The high-dose treatment kills the cancer cells. They are used to replace stem cells that have been damaged by high doses of chemotherapy, used to treat the patient's underlying disease.

The side effects of stem cell therapy differ from person to person. Listed below are the side effects of stem cell therapy :

According to the Indian Council of Medical Research, all is considered to be experimental, with the exception of bone marrow transplants. However, the guidelines that were put into place in 2007 are largely non-enforceable. Regardless, stem cell therapy is legalized in India. Umbilical cord and adult stem cell treatment are considered permissible. Embryonic stem cell therapy and research is restricted.

There is about a 60% to 80% overall success rate in the use of stem cell therapy in both India and around the world. However, success rates vary depending on the disease being treated, the institute conducting the procedures, and the condition of the patient. In order to receive complete information you will have to contact the medical institutes and ask specific questions concerning the patient's condition.

Mrs. Selina Naidoo with her Son from Malaysia

Tour2India4Health has proved to be a blessing in disguise for me. A medical tourism company with everything at par with our expectations has given me the most satisfactory and relieving experience of my life. I went to them for my sons surgery who was suffering from a serious illness and stem cell therapy was the only choice I had. Trust it was heart wrenching to leave my son under any hands on the operation table. Nevertheless, courageously I had to because thats what I was here for and thats what could get my son a new and healthy life. Sitting at a corner outside the operation theatre was taking my heartbeats away with every second. Finally, the surgery was over and I was there in front of the doctor with closed eyes. He declared that the surgery was successful and my son is fine but needs some extra care and some cautious post operative measures for recovery. All through our stay in the hospital, everything went on brilliantly and after my son recovered completely, I came back to my home country. Even after that for many months, I received regular calls to verify and virtually monitor the health of my child. Now, its been 5 years and when I see my child today it feels as if no surgery was ever done on him. Thanks to the doctor who treated him and to the entire team of nurses and travel professionals who displayed extra warmth and care. Thanks is just a small word to say as a mother of a child.

India is the most preferable destination for patients who are looking for low cost stem cell therapy. Indian doctors and healthcare professionals are renowned world over for their skills with many of them holding high positions in leading hospitals in US, UK and other countries around the world. There are significant numbers of highly skilled experts in India, including many who have relocated to India after having worked in the top hospitals across the world.

The Cost of stem cell treatment in India are generally about a tenth of the costs in US and are significantly cheaper compared with even other medical travel destinations like Thailand

*The price for the Stem Cell Therapy is an average collected from the 15 best corporate hospitals and 10 Top Stem Cell Experts of India.

*The final prices offered to the patients is based on their medical reports and is dependent on the current medical condition of the patient, type of room, type of therapy, hospital brand and the surgeon's expertise.

We have worked out special packages of the Stem Cell Therapy for our Indian and International patients. You can send us your medical reports to avail the benefits of these special packages.

You would be provided with 3 TOP RECOMMENDED SURGEONS / HOSPITALS FOR YOUR STEM CELL THERAPY in India.

There are many reasons for India becoming a popular medical tourism spot is the low cost stem cell treatment in the area. When in contrast to the first world countries like, US and UK, medical care in India costs as much as 60-90% lesser, that makes it a great option for the citizens of those countries to opt for stem cell treatment in India because of availability of quality healthcare in India, affordable prices strategic connectivity, food, zero language barrier and many other reasons.

The maximum number of patients for stem cell therapy comes from Nigeria, Kenya, Ethiopia, USA, UK, Australia, Saudi Arabia, UAE, Uzbekistan, Bangladesh.

Cities where top and world renowned Stem Cell Therapy hospitals and clinics situated are :

We have PAN-India level tie ups with TOP Hospitals for Stem Cell Therapy across 15+ major cities in India. We can provide you with multiple top hospitals & best surgeons recommendations for Stem Cell Therapy in India.

India has now been recognized as one of the leaders in medical field of research and treatment. Tour2India4Health Group was established with an aim of providing best medical services to its patients and since then has been working hard in maintaining itself as one of the most professional healthcare tourism providers in India. With a number of world-renowned medical facilities affiliated, we have the resources to offer you the finest medical treatment in India, and help your speedy recovery. Tour2India4Health Group has always believed and practiced providing its patients best surgery and treatment procedure giving a second chance to live a more better and normal life. Our team serves the clientele most comfortable and convenient measures of healthcare services thus, making your medical tour to India very fruitful experience.

Our facilitation:

We has been operating patients from all major countries like USA, United Kingdom, Italy, Australia, Canada, Spain, New Zealand, and Kuwait etc. We have network of selected medical centers, surgeons and physicians around various cities in India, who qualify our assessment criteria to ensure that our core values of Safety, Excellence and Trust are maintained in all our services.

Below are the downloadable links that will help you to plan your medical trip to India in a more organized and better way. Attached word and pdf files gives information that will help you to know India more and make your trip to India easy and memorable one.

Best Stem Cell Therapy in India, Cost of Stem Cell Therapy in India, Stem Cell Therapy Best Hospitals in India, Success Rate of Stem Cell Treatment in India, Stem Cell Therapy Treatment Cost in India, Allogeneic Stem cell Transplant Cost in India, autologous Stem Cell Transplant Cost in India, Stem Cell Therapy in India, Low Cost Stem Cell Therapy India, Stem Cell Benefits in India, Top Stem Cell Centers in India, Best Doctors for Stem Cell Therapy in India, List of Best Stem Cell Treatment Clinics in India, Allogeneic stem cell transplantation, Allogeneic Stem Cell Transplant Cost in India, Autologous Stem Cell Transplant, Autologous Stem Cell Transplant Cost in India

Read more from the original source:
Advance Stem Cell Therapy in India | Stem Cell Treatment ...

News Release

Wednesday, July 6, 2016

Findings could inform breast cancer disparities.

The largest study ever to investigate how genetic and biological factors contribute to breast cancer risk among black women launched today. This collaborative research project will identify genetic factors that may underlie breast cancer disparities. The effort is funded by the National Cancer Institute (NCI), part of the National Institutes of Health.

This effort is about making sure that all Americans no matter their background reap the same benefits from the promising advances of precision medicine.

Douglas R. Lowy, M.D., Acting Director, NCI

The Breast Cancer Genetic Study in African-Ancestry Populations initiative does not involve new patient enrollment but builds on years of research cooperation among investigators who are part of the African-American Breast Cancer Consortium, the African-American Breast Cancer Epidemiology and Risk (AMBER) Consortium, and the NCI Cohort Consortium. These investigators, who come from many different institutions, will share biospecimens, data, and resources from 18 previous studies, resulting in a study population of 20,000 black women with breast cancer.

This effort is about making sure that all Americans no matter their background reap the same benefits from the promising advances of precision medicine. The exciting new approaches to cancer prevention, diagnosis, and treatment ring hollow unless we can effectively narrow the gap of cancer disparities, and this new research initiative will help us do that, said Douglas R. Lowy, M.D., acting director of NCI. Im hopeful about where this new research can take us, not only in addressing the unique breast cancer profiles of African-American women, but also in learning more about the origin of cancer disparities.

Survival rates for women with breast cancer have been steadily improving over the past several decades. However, these improvements have not been shared equally; black women are more likely to die of their disease. Perhaps of most concern is that black women are more likely than white women to be diagnosed with aggressive subtypes of breast cancer. The rate of triple-negative breast cancer, an aggressive subtype, is twice as high in black women as compared to white women.

The exact reasons for these persistent disparities are unclear, although studies suggest that they are the result of a complex interplay of genetic, environmental, and societal factors, including access to health care. Large studies are needed to comprehensively examine these factors, and NCI is supporting several such efforts.

As part of the study, the genomes of 20,000 black women with breast cancer will be compared with those of 20,000 black women who do not have breast cancer. The genomes will also be compared to those of white women who have breast cancer. The project will investigate inherited genetic variations that are associated with breast cancer risk in black women compared to white women. In addition, researchers will examine gene expression in breast cancer tumor samples to investigate the genetic pathways that are involved in tumor development.

This $12 million grant in combination with previous investments should help advance our understanding of the social and biological causes that lead to disparities in cancer among underserved populations, said Robert Croyle, Ph.D., director of NCIs Division of Cancer Control and Population Sciences (DCCPS), which is administering the grant. A better understanding of the genetic contributions to differences in breast cancer diagnoses and outcomes among African-Americans may lead to better treatments and better approaches to cancer prevention.

A number of studies have suggested that genetic factors may influence breast cancer disparities, so were hopeful that this project can help to shed further light on this matter. said Damali Martin, Ph.D., program director for the DCCPS Genomic Epidemiology Branch. Dr. Martins office is working directly with the grant recipients as well as the consortia groups that have been researching black women and breast cancer.

The grant has been awarded to Wei Zheng, M.D., Ph.D., of Vanderbilt University, Nashville, Tennesee; Christopher Haiman, Sc.D., of the University of Southern California, Los Angeles; and Julie Palmer, Sc.D., of Boston University. Additionally, minority scientists from various institutions, including from one Historically Black College and University medical school, are playing an important role in this study, and they have been involved in previous research that this study builds upon. For example, the Southern Community Cohort Study, a contributing study for this grant, represents a 15-year partnership between Vanderbilt and historically black Meharry Medical College in Nashville, Tennessee. In addition, this grant will provide training opportunities for scientists from minority populations.

Support for ongoing research in this area represents NCIs continued commitment to fund a comprehensive portfolio of research aimed at reducing cancer risk, incidence, and mortality, as well as improving quality of life for cancer survivors across all demographic groups.

The National Cancer Institute leads the National Cancer Program and the NIHs efforts to dramatically reduce the prevalence of cancer and improve the lives of cancer patients and their families, through research into prevention and cancer biology, the development of new interventions, and the training and mentoring of new researchers. For more information about cancer, please visit the NCI website at http://www.cancer.gov or call NCI's Cancer Information Service at 1-800-4-CANCER.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

ReferenceBreast Cancer Genetic Study in African-Ancestry Populations, Grant Number R01CA202981

###

See the article here:
NIH launches largest-ever study of breast cancer genetics ...

Don't think that either is superior however, each is superior at specific things, obviously.

But here is some fodder for thought. Interesting Genetic Fact:

The female population currently outweighs the male population by 1% or so making the ratio 51% to 49% roughly. However, the male population is predicted to catch up and perhaps surpass the female population only slightly with modern medicine. Why? Because the sex chromosomes, XX for woman and XY for men, carry different genes. The X chromosomes carry large amounts of DNA information while the Y chromosome which is shorter than the X chromosome, only carries a few bits of genetic information such as the gene for becoming male. Essentially, we all start out female! It is the presence of the testis gene, called SRY, that determines the male gender.

Because the X chromosome carries large amounts of genetic information while the Y does not, males are more likely to suffer from disease and abnormalities than women. In genetics, two genes come together to determine a trait. One or both can be dominant or recessive. Disease genes are recessive as are abnormalities but if a male receives a recessive gene for a disease or abnormality, he is likely to express that gene given the lack of extra DNA information from the Y chromosome. Therefore, more male die in infancy than females. Does this make females genetically superior? Modern medicine will help combat early deaths from a genetic standpoint, helping even out the population. Let's not forget that females also develop faster overall, emotionally, physically, and intellectually.

See more here:
Serious question about female genetics? | Yahoo Answers

They were born without a working germ-fighting system, every infection a threat to their lives. Now eight babies with "bubble boy disease" have had it fixed by a gene therapy made from one of the immune system's worst enemies HIV, the virus that causes AIDS.

Astudyout Wednesday details how scientists turned this enemy virus into a savior, altering it so it couldn't cause disease and then using it to deliver a gene the boys lacked.

"This therapy has cured the patients," although it will take more time to see if it's a permanent fix, said Dr. Ewelina Mamcarz, one of the study leaders at St. Jude Children's Research Hospital in Memphis.

Omarion Jordan, who turns 1 later this month, had the therapy in December to treat severe combined immunodeficiency syndrome, or SCID.

"For a long time we didn't know what was wrong with him. He just kept getting these infections," said his mother, Kristin Simpson. Learning that he had SCID "was just heartbreaking ... I didn't know what was going to happen to him."

Omarion now has a normal immune system. "He's like a normal, healthy baby," Simpson said. "I think it's amazing."

Study results were published by the New England Journal of Medicine. The treatment was pioneered by a St. Jude doctor who recently died, Brian Sorrentino.

SCID is caused by a genetic flaw that keeps the bone marrow from making effective versions of blood cells that comprise the immune system. It affects 1 in 200,000 newborns, almost exclusively males. Without treatment, it often kills in the first year or two of life.

"A simple infection like the common cold could be fatal," Mamcarz said.

The nickname "bubble boy disease" comes from a famous case in the 1970s a Texas boy who lived for 12 years in a protective plastic bubble to isolate him from germs. A bone marrow transplant from a genetically matched sibling can cure SCID, but most people lack a suitable donor. Transplants also are medically risky the Texas boy died after one.

Doctors think gene therapy could be a solution. It involves removing some of a patient's blood cells, using the modified HIV to insert the missing gene, and returning the cells through an IV. Before getting their cells back, patients are given a drug to destroy some of their marrow so the modified cells have more room to grow.

When doctors first tried it 20 years ago, the treatment had unintended effects on other genes, and some patients later developed leukemia. The new therapy has safeguards to lower that risk.

A small study of older children suggested it was safe. The new study tried it in infants, and doctors are reporting on the first eight who were treated at St. Jude and at UCSF Benioff Children's Hospital San Francisco.

Within a few months, normal levels of healthy immune system cells developed in seven boys. The eighth needed a second dose of gene therapy but now is well, too. Six to 24 months after treatment, all eight are making all the cell types needed to fight infections, and some have successfully received vaccines to further boost their immunity to disease.

No serious or lasting side effects occurred.

Omarion is the 10th boy treated in the study, which is ongoing. It's sponsored by the American Lebanese Syrian Associated Charities, the California Institute of Regenerative Medicine, the Assisi Foundation of Memphis and the federal government.

"So far it really looks good," but patients will have to be studied to see if the results last, said Dr. Anthony Fauci, head of the National Institute of Allergy and Infectious Diseases, which helped develop the treatment. "To me, this looks promising."

Rights to it have been licensed by St. Jude to Mustang Bio. Doctors say they have no estimate on what it might cost if it does become an approved treatment.

A similar technique harnessing a modified version of HIV is also being studied as a possible cure for sickle cell anemia, CBS News chief medical correspondent Dr. Jon LaPook reports. In a clinical trial at the National Institutes of Health, nine adults with sickle cell anemia have undergone the gene therapy. So far, all are responding well.

Read the original here:
Gene therapy might be a cure for "bubble boy disease ...

Healthy Aging Medical Centers of Essex County New Jersey offers a host of advanced treatment programs to help their patients look and feel their very best including Bioidentical Hormone Replacement Therapy, Testosterone Replacement Therapy, Functional Medicine Services, Cosmetic and Medical Aesthetic procedures, and more.

Learn more about the services offered by Dr. Rand at Healthy Aging Medical Centers of West Orange New Jersey, schedule yourAGE MANAGEMENT CONSULTATIONwith Dr. Rand today. Healthy Aging Medical Centers pride themselves in offering the very best individualized treatment protocols available, serving patients in the Northern New Jersey areas of Essex County, Bergen County, Passaic County, Hudson County, Union County, Middlesex County, and Somerset County New Jersey.

If you are sick and tired of being sick and tired, dont wait any longer to learn about the benefits of Bioidentical Hormone Replacement Therapy as part of a total wellness program offered by Dr. Rand at Healthy Aging Medical Centers in Essex County New Jersey. Call today and learn more about the amazing possibilities of Anti-Aging medicine from someone who has experienced the benefits first hand, Doctor Johanan Rand, M.D.

Continue reading here:
New Jersey Anti Aging Programs for Women and Men

Michael Passineau, PhD, is a man who speaks in metaphors. For good reason he works within a realm of medicine not many people understand. Director of the Gene Therapy Program at Allegheny Health Network (AHN) and a leading force behind AHNs scientific research to address clinical needs, Passineau leans into the way a good metaphor can bring clarity to challenging conceptsincluding the nature of his work.

I think of clinicians as chefs, he says. At the end of each day, theyve done something tangible. Theyve made a meal. Researchers, on the other hand, are a bit like sculptors. We can work for years on something, but once its complete, its permanent.

For over a decade, his research has revolved around gene therapy more specifically, the use of ultrasound technology, instead of viral administration, to deliver therapeutic DNA into the cells of salivary glands. The goal: restore saliva flow to patients who suffer from radiation-induced dry mouth, or xerostomia.

Supported by grants from the National Institutes of Health (NIH), Passineau, radiation oncologist Dr. Mark Trombetta, and their research team are on track to petition the U.S Food and Drug Administration for Investigational New Drug (IND) status, which would allow their work to move out of the lab and into Phase 1 clinical trials with humans.

Xerostomia is an iatrogenic complication, meaning it is caused by treatment in this case, head and neck radiation to treat cancer. When the beam of radiation passes through the head, it damages the salivary glands, resulting in chronic dry mouth. This can lead to permanent loss of function in the salivary glands, difficulty eating, and loss of teeth.

Its anything but trivial, says Passineau. To illustrate how this condition impacts a persons quality of life, I often have donors and executives take a piece of surgical gauze and chew on it while I describe my research. After about five minutes, they understand how difficult it really is.

While there are a few existing medications used to treat xerostomia, they are difficult to administer, and their effects are not long lasting. Most people deal with the condition by carrying a water bottle at all times or by taking saliva substitutes. Unfortunately, these options dont work particularly well.

Were all made of trillions of cells, says Passineau, beginning an attempt to explain gene therapy in a nutshell.

Each cell has a role to play, whether its beating heart muscle, growing hair and nails, or perceiving light signals in your retina, he continues. The way those biochemical machines are engineered is dictated by your genomic DNA, which is DNA you get from your parents. Those genes code for proteins, which are the gears and springs that make cells function as biochemical machines.

Gene therapy means reprogramming the cell changing the machine code telling the cell how to work. That requires getting new DNA to travel inside the cell. Passineau says this is one of the most difficult tasks in the world since our cells are designed to repel foreign DNA.

In nature, foreign DNA gets into human cells only through viral infection or during conception. Virally administered gene therapy approaches have been developed, but one drawback is that after a viral vector is introduced into the body, the immune system fights back and will also react to the vector on subsequent treatments, making them ineffective.

Thats where Passineaus research comes in. Weve developed a method of delivering genes that doesnt require viral administration, he explains. Instead, we use soundwaves.

To understand how ultrasonic administration, or sonoporation, works, Passineau turns again to metaphor.

Picture an agricultural pond, with a thick layer of algae on it. If you throw a ping-pong ball into the middle, it will just sit on top, he says. That is very much what a cell membrane is like the outer covering is rather rigid. So, to deliver the genes, we have to get through the cell membrane. It is only seven nanometers thick but its the longest seven nanometers in nature for someone like me.

Passineaus ground-breaking research uses soundwaves to temporarily alter the permeability of the cell membrane, allowing for the transfer of therapeutic DNA into the cell.

Lets understand how this works in our pond metaphor before getting into what that means for gene therapy.

Imagine we explode a grenade above the pond, Passineau says. For a moment, the layer of slime would open up, and youd see down to the bottom of the pond. Then, it would close again.

In Passineaus lab, the grenade is a mix of a gene drug for xerostomia known as Aquaporin-1 and a solution of microbubbles. Used routinely in cardiac imaging and other medical applications, microbubbles have a resonant frequency that can be used to create the desired explosion.

The classic example is a crystal glass if an opera singer hits the right frequency for that glass, it will vibrate. If she really turns up the volume, the glass will shatter, because it cant absorb the energy, Passineau says. That same thing happens with the microbubbles.

So after administering the microbubble and Aquaporin-1 solution to the treatment site, a low-frequency ultrasound beam is used to create an ultrasonic acoustic field in which the bubbles vibrate. Turn up the power, and the bubbles implode. That opens up the cell membrane long enough for the gene drug to get in, before it closes back up.

For gene therapy researchers like Passineau, the membrane around our cells is the longest seven nanometers in nature.

Sonoporation works well for what were doing in the salivary glands, but not so well for the heart, and certainly not for the brain, Passineau points out. However, we have other applications we intend to use this research for.

He explains that one promising use involves Sjogrens syndrome, an autoimmune disease affecting nearly 4 million Americans (90 percent of whom are women). The diseases debilitating symptoms include severe dry mouth, which may be treatable with Passineaus gene therapy technique.

Another research area he says he is excited about is the use of gene therapy to combat obesity and overeating. Do you remember when you were a kid and youd eat too fast and your mom would tell you to slow down because your brain didnt know whether or not your stomach was full yet? he asks. Well, that was absolutely true.

He explains that, when we eat, our intestines stretch and release a protein called peptide YY (PYY), which circulates through the blood, eventually entering the saliva and interacting with receptors on your tongue.

Think of your appetite as a glass of water, he says. To feel full, you have to fill the glass with PYY. Some people have bigger glasses than others, but if we can use gene therapy to modify saliva and make the glass start half full, then a person would feel full without needing to eat as much.

Passineau adds that poor health outcomes and high costs associated with obesity make this an attractive target for research investment. Obesity adds billions of dollars to the cost of medical care in the U.S. each year, and some studies estimate the cost as high as $190 billion per year.

If gene therapy was this easy, everyone would be doing it. Instead, as Passineau points out, it is one of the most difficult tasks in the world.

At AHN, research is a small but important piece of the operation, Passineau says. Its important to note that everything we do in research is driven by physicians who have recognized clinical needs, and who have partnered with us to develop novel solutions.

Similarly, looking at the value that research can deliver, and its potential impact on both health and overall health care costs, Passineau says that federal funding has an essential role in advancing further discoveries in areas like gene therapy and sonoporation. Government investment really is the lifeblood of what drives research, he says.

Another impact on the success and pace of advances in medical research is whether talented, driven young people decide to take this path. Passineau admits that, like the process of research itself, the path to becoming a successful researcher can be long and sometimes feels like three steps forward, two steps back. But if the work feels meaningful, its all worth it.

I landed where I am today because I figured out what I was good at, he says. Inventing, solving big picture problems and helping people.

Go here to see the original:
Highmark Health Blog | Gene Therapy Research: Dr. Passineau

Frequently Asked QuestionsWhatis gene therapy?

Some diseases are caused by errors (mutations) inspecific genes. Gene therapy delivers DNA into cells to replacemutated (bad) or missing genes or to add new, good genes.

Scientists are investigating a number of differentways to do this. Right now, gene therapy is only done through research studiescalled clinical trials. Unlike medicine, gene therapy directly addresses the underlyinggenetic problem, not just the symptoms.

Genes are in the nucleus of every living cell. A gene is an instruction manual for the body. Itgives the direction to make the proteins that make the body work.

A gene cannot be inserted directly into a cell.Instead, a carrier called a vector is genetically engineered to deliver thegene. Viruses are usually used as the vectors because they are very good atinfecting cells and inserting the gene(s) into the cells DNA. Types of viralvectors are retrovirus, adenovirus, adeno-associated virus and herpes simplexvirus.

No. The virus is specially engineered to remove the infectious piece. We only keep the part of the virus that is good at burrowinginto a cells nucleus. Once the virus delivers the gene into the cell, thevirus slips away.

It is not for all genetic diseases. It is only forsome diseases caused by a single gene mutation. Some diseases that might betreated with gene therapy are:

The goal is to cure a disease or make changes so thebody can better fight off disease. It does not correct 100% of your childs cells.Instead, every time a cell with the good gene reproduces, it carries a copyof the new healthy gene.

The vector can be injected or given by IV directlyinto a specific tissue. Or a sample of cells can be removed and exposed to thevector in a laboratory. The cells with the vector are then returned to thepatient.

1) Stem cells are collected in one of two ways: by bone marrow aspiration, or by purifying blood drawn through a central line in a process called apheresis.

2) Before the infusion, most children have chemotherapy. This makes room for the new cells by getting rid of the existing cells in the bone marrow.

3) In the laboratory, the stem cells from the blood or bone marrow are exposed to a virus or other type of vector containing the desired genes.

4) Once the stem cells take up the vector and merge the genes into cells DNA, the cells are given back to the patient in an IV infusion.

Bone marrow transplants usestem cells from another person (a donor). Gene therapy uses your childs owncells. Using your childs own cells is a benefit because there is no risk ofrejection, or graft vs. host disease, like there is with donor cells. Genetherapy is still only offered through clinical trials and at only a fewresearch hospitals and centers.

Gene therapy is still very new,and is mostly used to treat children who cannot be cured by standardtreatments. Gene therapy is not for everydisease or a good fit for every patient. Your childneeds to meet certain criteria for safety reasons. Your childs doctor willtalk to you about whether your child is a good fit for a gene therapy clinicaltrial.

Your child will have 410 daysof chemotherapy before the infusion. This is called chemotherapy conditioning.It clears out bone marrow to make room for the new stem cells. This has typicalside effects from chemotherapy, like nausea/vomiting, mouth sores and pain.

Your child has the transfusionon the Bone Marrow Transplant floor (6 West) at the Jimmy Fund Clinic. It is given one timeintravenously (through an IV), just like a blood transfusion. It takes 1530minutes. The amount of time your childwill stay in the hospital depends on many factors. Most children stay 46weeks.

Your child will have bloodtests to check for the vector in the cells, and to see how the cells are responding. Your child will come in forfollow-ups frequently. Your childs care team will talk to you about when youshould call your childs doctor. Always call with questions or concerns or ifyou notice signs of an infection.

Many research studies areunderway to test gene therapy as a safe treatment for a growing number ofdiseases. Improvements have already beenmade in safety. Early gene therapy trials showed a high risk of turning ononcogenes that cause cancer. Now, experts have retooled the vector to lower thelikelihood of turning on oncogenes.

Learn more

Read more from the original source:
Gene Therapy Questions | FAQs - Dana-Farber/Boston ...

Gene editing method

CRISPR gene editing is a method by which the genomes of living organisms may be edited. It is based on a simplified version of the bacterial CRISPR/Cas (CRISPR-Cas9) antiviral defense system. By delivering the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, allowing existing genes to be removed and/or new ones added.[1] The Cas9-gRNA complex corresponds with the CAS III CRISPR-RNA complex in the accompanying diagram.

While genomic editing in eukaryotic cells has been possible using various methods since the 1980s, the methods employed had proved to be inefficient and impractical to implement on a larger scale. Genomic editing leads to irreversible changes to the gene. Working like genetic scissors, the Cas9 nuclease opens both strands of the targeted sequence of DNA to introduce the modification by one of two methods. Knock-in mutations, facilitated via Homology Directed Repair (HDR), is the traditional pathway of targeted genomic editing approaches.[2] This allows for the introduction of targeted DNA damage and repair. HDR employs the use of similar DNA sequences to drive the repair of the break via the incorporation of exogenous DNA to function as the repair template.[2] This method relies on the periodic and isolated occurrence of DNA damage at the target site in order for a repair to commence. Knock-out mutations caused by Cas9/CRISPR results in the repair of the double-strand break by means of NHEJ (Non-Homologous End Joining). NHEJ can often result in random deletions or insertions at the repair site disrupting or altering gene functionality. Therefore, genomic engineering by CRISPR-Cas9 allows researchers the ability to generate targeted random gene disruption.

Because of this, the precision of genomic editing is a great concern. With the discovery of CRISPR and specifically the Cas9 nuclease molecule, efficient and highly selective editing is now a reality. Cas9 allows for a reliable method of creating a targeted break at a specific location as designated by the crRNA and tracrRna guide strands.[3] Cas9 derived from Streptococcus pyogenes bacteria has facilitated the targeted genomic modification in eukaryotic cells. The ease with which researchers can insert Cas9 and template RNA in order to silence or cause point mutations on specific loci has proved invaluable to the quick and efficient mapping of genomic models and biological processes associated with various genes in a variety of eukaryotes. A newly engineered variant of the Cas9 nuclease has been developed that significantly reduces off-target manipulation. Called spCas9-HF1 (Streptococcus pyogenes Cas9 High Fidelity 1), it has a success rate of modification in vivo of 85% and undetectable off-target manipulations as measured by genome wide break capture and targeted sequencing methods used to measure total genomic changes.[4][5]

CRISPR-Cas genome editing techniques have many potential applications, including medicine and crop seed enhancement. The use of CRISPR-Cas9-gRNA complex for genome editing[6] was the AAAS's choice for breakthrough of the year in 2015.[7] Bioethical concerns have been raised about the prospect of using CRISPR for germline editing.[8]

In the early 2000s, researchers developed zinc finger nucleases (ZFNs), synthetic proteins whose DNA-binding domains enable them to create double-stranded breaks in DNA at specific points. In 2010, synthetic nucleases called transcription activator-like effector nucleases (TALENs) provided an easier way to target a double-stranded break to a specific location on the DNA strand. Both zinc finger nucleases and TALENs require the creation of a custom protein for each targeted DNA sequence, which is a more difficult and time-consuming process than that for guide RNAs. CRISPRs are much easier to design because the process requires making only a short RNA sequence.[9]

Whereas RNA interference (RNAi) does not fully suppress gene function, CRISPR, ZFNs and TALENs provide full irreversible gene knockout.[10] CRISPR can also target several DNA sites simultaneously by simply introducing different gRNAs. In addition, CRISPR costs are relatively low.[10][11][12]

CRISPR-Cas9 genome editing is carried out with a Type II CRISPR system. When utilized for genome editing, this system includes Cas9, crRNA, tracrRNA along with an optional section of DNA repair template that is utilized in either non-homologous end joining (NHEJ) or homology directed repair (HDR).

CRISPR-Cas9 often employs a plasmid to transfect the target cells.[13] The main components of this plasmid are displayed in the image and listed in the table. The crRNA needs to be designed for each application as this is the sequence that Cas9 uses to identify and directly bind to the cell's DNA. The crRNA must bind only where editing is desired. The repair template is designed for each application, as it must overlap with the sequences on either side of the cut and code for the insertion sequence.

Multiple crRNAs and the tracrRNA can be packaged together to form a single-guide RNA (sgRNA).[14] This sgRNA can be joined together with the Cas9 gene and made into a plasmid in order to be transfected into cells.

CRISPR-Cas9 offers a high degree of fidelity and relatively simple construction. It depends on two factors for its specificity: the target sequence and the PAM. The target sequence is 20 bases long as part of each CRISPR locus in the crRNA array.[13] A typical crRNA array has multiple unique target sequences. Cas9 proteins select the correct location on the host's genome by utilizing the sequence to bond with base pairs on the host DNA. The sequence is not part of the Cas9 protein and as a result is customizable and can be independently synthesized.[15][16]

The PAM sequence on the host genome is recognized by Cas9. Cas9 cannot be easily modified to recognize a different PAM sequence. However this is not too limiting as it is a short sequence and nonspecific (e.g. the SpCas9 PAM sequence is 5'-NGG-3' and in the human genome occurs roughly every 8 to 12 base pairs).[13]

Once these have been assembled into a plasmid and transfected into cells the Cas9 protein with the help of the crRNA finds the correct sequence in the host cell's DNA and depending on the Cas9 variant creates a single or double strand break in the DNA.[17]

Properly spaced single strand breaks in the host DNA can trigger homology directed repair, which is less error prone than the non-homologous end joining that typically follows a double strand break. Providing a DNA repair template allows for the insertion of a specific DNA sequence at an exact location within the genome. The repair template should extend 40 to 90 base pairs beyond the Cas9 induced DNA break.[13] The goal is for the cell's HDR process to utilize the provided repair template and thereby incorporate the new sequence into the genome. Once incorporated, this new sequence is now part of the cell's genetic material and passes into its daughter cells.

Many online tools are available to aid in designing effective sgRNA sequences.[18][19]

Delivery of Cas9, sgRNA, and associated complexes into cells can occur via viral and non-viral systems. Electroporation of DNA, RNA, or ribonucleocomplexes is a common technique, though it can result in harmful effects on the target cells.[20] Chemical transfection techniques utilizing lipids have also been used to introduce sgRNA in complex with Cas9 into cells.[21] Hard-to-transfect cells (e.g. stem cells, neurons, and hematopoietic cells) require more efficient delivery systems such as those based on lentivirus (LVs), adenovirus (AdV) and adeno-associated virus (AAV).[22][23]

Several variants of CRISPR-Cas9 allow gene activation or genome editing with an external trigger such as light or small molecules.[24][25][26] These include photoactivatable CRISPR systems developed by fusing light-responsive protein partners with an activator domain and a dCas9 for gene activation,[27][28] or fusing similar light responsive domains with two constructs of split-Cas9,[29][30] or by incorporating caged unnatural amino acids into Cas9,[31] or by modifying the guide RNAs with photocleavable complements for genome editing.[32]

Methods to control genome editing with small molecules include an allosteric Cas9, with no detectable background editing, that will activate binding and cleavage upon the addition of 4-hydroxytamoxifen (4-HT),[24] 4-HT responsive intein-linked Cas9s[33] or a Cas9 that is 4-HT responsive when fused to four ERT2 domains.[34] Intein-inducible split-Cas9 allows dimerization of Cas9 fragments[35] and Rapamycin-inducible split-Cas9 system developed by fusing two constructs of split Cas9 with FRB and FKBP fragments.[36] Furthermore, other studies have shown to induce transcription of Cas9 with a small molecule, doxycycline.[37][38] Small molecules can also be used to improve Homology Directed Repair (HDR),[39] often by inhibiting the Non-Homologous End Joining (NHEJ) pathway.[40] These systems allow conditional control of CRISPR activity for improved precision, efficiency and spatiotemporal control.

Cas9 genomic modification has allowed for the quick and efficient generation of transgenic models within the field of genetics. Cas9 can be easily introduced into the target cells via plasmid transfection along with sgRNA in order to model the spread of diseases and the cell's response and defense to infection.[41] The ability of Cas9 to be introduced in vivo allows for the creation of more accurate models of gene function, mutation effects, all while avoiding the off-target mutations typically observed with older methods of genetic engineering. The CRISPR and Cas9 revolution in genomic modeling doesn't only extend to mammals. Traditional genomic models such as Drosophila melanogaster, one of the first model species, have seen further refinement in their resolution with the use of Cas9.[41] Cas9 uses cell-specific promoters allowing a controlled use of the Cas9. Cas9 is an accurate method of treating diseases due to the targeting of the Cas9 enzyme only affecting certain cell types. The cells undergoing the Cas9 therapy can also be removed and reintroduced to provide amplified effects of the therapy.[42]

CRISPR-Cas9 can be used to edit the DNA of organisms in vivo and entire chromosomes can be eliminated from an organism at any point in its development. Chromosomes that have been deleted in vivo are the Y chromosomes and X chromosomes of adult lab mice and human chromosomes 14 and 21, in embryonic stem cell lines and aneuploid mice respectively. This method might be useful for treating genetic aneuploid diseases such as Down Syndrome and intersex disorders.[43]

Successful in vivo genome editing using CRISPR-Cas9 has been shown in several model organisms, such as Escherichia coli,[44] Saccharomyces cerevisiae,[45] Candida albicans,[46] Caenorhadbitis elegans,[47] Arabidopsis,[48] Danio rerio,[49] Mus musculus.[50][51] Successes have been achieved in the study of basic biology, in the creation of disease models,[47] and in the experimental treatment of disease models.[52]

Concerns have been raised that off-target effects (editing of genes besides the ones intended) may obscure the results of a CRISPR gene editing experiment (the observed phenotypic change may not be due to modifying the target gene, but some other gene). Modifications to CRISPR have been made to minimize the possibility of off-target effects. In addition, orthogonal CRISPR experiments are recommended to confirm the results of a gene editing experiment.[53][54]

CRISPR simplifies creation of animals for research that mimic disease or show what happens when a gene is knocked down or mutated. CRISPR may be used at the germline level to create animals where the gene is changed everywhere, or it may be targeted at non-germline cells.[55][56][57]

CRISPR can be utilized to create human cellular models of disease. For instance, applied to human pluripotent stem cells CRISPR introduced targeted mutations in genes relevant to polycystic kidney disease (PKD) and focal segmental glomerulosclerosis (FSGS).[58] These CRISPR-modified pluripotent stem cells were subsequently grown into human kidney organoids that exhibited disease-specific phenotypes. Kidney organoids from stem cells with PKD mutations formed large, translucent cyst structures from kidney tubules. The cysts were capable of reaching macroscopic dimensions, up to one centimeter in diameter.[59] Kidney organoids with mutations in a gene linked to FSGS developed junctional defects between podocytes, the filtering cells affected in that disease. This was traced to the inability of podocytes ability to form microvilli between adjacent cells.[60] Importantly, these disease phenotypes were absent in control organoids of identical genetic background, but lacking the CRISPR modifications.[58]

A similar approach was taken to model long QT syndrome in cardiomyocytes derived from pluripotent stem cells.[61] These CRISPR-generated cellular models, with isogenic controls, provide a new way to study human disease and test drugs.

CRISPR-Cas technology has been proposed as a treatment for multiple human diseases, especially those with a genetic cause.[62] Its ability to modify specific DNA sequences makes it a tool with potential to fix disease-causing mutations. Early research in animal models suggest that therapies based on CRISPR technology have potential to treat a wide range of diseases,[63] including cancer,[64] beta-thalassemia,[65] sickle cell disease,[66] hemophilia,[67] cystic fibrosis,[68] Duchenne's muscular dystrophy,[69] Huntington's,[70][71] and heart disease.[72] CRISPR may have applications in tissue engineering and regenerative medicine, such as by creating human blood vessels that lack expression of MHC class II proteins, which often cause transplant rejection.[73]

CRISPR-Cas-based "RNA-guided nucleases" can be used to target virulence factors, genes encoding antibiotic resistance and other medically relevant sequences of interest. This technology thus represents a novel form of antimicrobial therapy and a strategy by which to manipulate bacterial populations.[74][75] Recent studies suggested a correlation between the interfering of the CRISPR-Cas locus and acquisition of antibiotic resistance[76] This system provides protection of bacteria against invading foreign DNA, such as transposons, bacteriophages and plasmids. This system was shown to be a strong selective pressure for the acquisition of antibiotic resistance and virulence factor in bacterial pathogens.[76]

Therapies based on CRISPRCas3 gene editing technology delivered by engineered bacteriophages could be used to destroy targeted DNA in pathogens. [77] Cas3 is more destructive than the better known Cas9[78][79]

Research suggests that CRISPR is an effective way to limit replication of multiple herpesviruses. It was able to eradicate viral DNA in the case of Epstein-Barr virus (EBV). Anti-herpesvirus CRISPRs have promising applications such as removing cancer-causing EBV from tumor cells, helping rid donated organs for immunocompromised patients of viral invaders, or preventing cold sore outbreaks and recurrent eye infections by blocking HSV-1 reactivation. As of August2016[update], these were awaiting testing.[80]

CRISPR may revive the concept of transplanting animal organs into people. Retroviruses present in animal genomes could harm transplant recipients. In 2015, a team eliminated 62 copies of a retrovirus's DNA from the pig genome in a kidney epithelial cell.[81] Researchers recently demonstrated the ability to birth live pig specimens after removing these retroviruses from their genome using CRISPR for the first time.[82]

As of 2016[update] CRISPR had been studied in animal models and cancer cell lines, to learn if it can be used to repair or thwart mutated genes that cause cancer.[83]

The first clinical trial involving CRISPR started in 2016. It involved removing immune cells from people with lung cancer, using CRISPR to edit out the gene expressed PD-1, then administrating the altered cells back to the same person. 20 other trials were under way or nearly ready, mostly in China, as of 2017[update].[64]

In 2016, the United States Food and Drug Administration (FDA) approved a clinical trial in which CRISPR would be used to alter T cells extracted from people with different kinds of cancer and then administer those engineered T cells back to the same people.[84]

Using "dead" versions of Cas9 (dCas9) eliminates CRISPR's DNA-cutting ability, while preserving its ability to target desirable sequences. Multiple groups added various regulatory factors to dCas9s, enabling them to turn almost any gene on or off or adjust its level of activity.[81] Like RNAi, CRISPR interference (CRISPRi) turns off genes in a reversible fashion by targeting, but not cutting a site. The targeted site is methylated, epigenetically modifying the gene. This modification inhibits transcription. These precisely placed modifications may then be used to regulate the effects on gene expressions and DNA dynamics after the inhibition of certain genome sequences within DNA. Within the past few years, epigenetic marks in different human cells have been closely researched and certain patterns within the marks have been found to correlate with everything ranging from tumor growth to brain activity.[6] Conversely, CRISPR-mediated activation (CRISPRa) promotes gene transcription.[85] Cas9 is an effective way of targeting and silencing specific genes at the DNA level.[86] In bacteria, the presence of Cas9 alone is enough to block transcription. For mammalian applications, a section of protein is added. Its guide RNA targets regulatory DNA sequences called promoters that immediately precede the target gene.[87]

Cas9 was used to carry synthetic transcription factors that activated specific human genes. The technique achieved a strong effect by targeting multiple CRISPR constructs to slightly different locations on the gene's promoter.[87]

In 2016, researchers demonstrated that CRISPR from an ordinary mouth bacterium could be used to edit RNA. The researchers searched databases containing hundreds of millions of genetic sequences for those that resembled Crispr genes. They considered the fusobacteria Leptotrichia shahii. It had a group of genes that resembled CRISPR genes, but with important differences. When the researchers equipped other bacteria with these genes, which they called C2c2, they found that the organisms gained a novel defense.[88]

Many viruses encode their genetic information in RNA rather than DNA that they repurpose to make new viruses. HIV and poliovirus are such viruses. Bacteria with C2c2 make molecules that can dismember RNA, destroying the virus. Tailoring these genes opened any RNA molecule to editing.[88]

CRISPR-Cas systems can also be employed for editing of micro-RNA and long-noncoding RNA genes in plants.[89]

Gene drives may provide a powerful tool to restore balance of ecosystems by eliminating invasive species. Concerns regarding efficacy, unintended consequences in the target species as well as non-target species have been raised particularly in the potential for accidental release from laboratories into the wild. Scientists have proposed several safeguards for ensuring the containment of experimental gene drives including molecular, reproductive, and ecological.[90] Many recommend that immunization and reversal drives be developed in tandem with gene drives in order to overwrite their effects if necessary.[91] There remains consensus that long-term effects must be studied more thoroughly particularly in the potential for ecological disruption that cannot be corrected with reversal drives.[92]

Unenriched sequencing libraries often have abundant undesired sequences. Cas9 can specifically deplete the undesired sequences with double strand breakage with up to 99% efficiency and without significant off-target effects as seen with restriction enzymes. Treatment with Cas9 can deplete abundant rRNA while increasing pathogen sensitivity in RNA-seq libraries.[93]

As of November2013[update], SAGE Labs (part of Horizon Discovery group) had exclusive rights from one of those companies to produce and sell genetically engineered rats and non-exclusive rights for mouse and rabbit models.[94] By 2015[update], Thermo Fisher Scientific had licensed intellectual property from ToolGen to develop CRISPR reagent kits.[95]

As of December2014[update], patent rights to CRISPR were contested. Several companies formed to develop related drugs and research tools.[96] As companies ramp up financing, doubts as to whether CRISPR can be quickly monetized were raised.[97] In February 2017 the US Patent Office ruled on a patent interference case brought by University of California with respect to patents issued to the Broad Institute, and found that the Broad patents, with claims covering the application of CRISPR-Cas9 in eukaryotic cells, were distinct from the inventions claimed by University of California.[98][99][100]Shortly after, University of California filed an appeal of this ruling.[101][102]

In March 2017, the European Patent Office (EPO) announced its intention to allow broad claims for editing all kinds of cells to Max-Planck Institute in Berlin, University of California, and University of Vienna,[103][104] and in August 2017, the EPO announced its intention to allow CRISPR claims in a patent application that MilliporeSigma had filed.[103] As of August2017[update] the patent situation in Europe was complex, with MilliporeSigma, ToolGen, Vilnius University, and Harvard contending for claims, along with University of California and Broad.[105]

As of March 2015, multiple groups had announced ongoing research to learn how they one day might apply CRISPR to human embryos, including labs in the US, China, and the UK, as well as US biotechnology company OvaScience.[106] Scientists, including a CRISPR co-discoverer, urged a worldwide moratorium on applying CRISPR to the human germline, especially for clinical use. They said "scientists should avoid even attempting, in lax jurisdictions, germline genome modification for clinical application in humans" until the full implications "are discussed among scientific and governmental organizations".[107][108] These scientists support further low-level research on CRISPR and do not see CRISPR as developed enough for any clinical use in making heritable changes to humans.[109]

In April 2015, Chinese scientists reported results of an attempt to alter the DNA of non-viable human embryos using CRISPR to correct a mutation that causes beta thalassemia, a lethal heritable disorder.[110][111] The study had previously been rejected by both Nature and Science in part because of ethical concerns.[112] The experiments resulted in successfully changing only some of the intended genes, and had off-target effects on other genes. The researchers stated that CRISPR is not ready for clinical application in reproductive medicine.[112] In April 2016, Chinese scientists were reported to have made a second unsuccessful attempt to alter the DNA of non-viable human embryos using CRISPR - this time to alter the CCR5 gene to make the embryo HIV resistant.[113]

In December 2015, an International Summit on Human Gene Editing took place in Washington under the guidance of David Baltimore. Members of national scientific academies of America, Britain and China discussed the ethics of germline modification. They agreed to support basic and clinical research under certain legal and ethical guidelines. A specific distinction was made between somatic cells, where the effects of edits are limited to a single individual, versus germline cells, where genome changes could be inherited by descendants. Heritable modifications could have unintended and far-reaching consequences for human evolution, genetically (e.g. gene/environment interactions) and culturally (e.g. Social Darwinism). Altering of gametocytes and embryos to generate inheritable changes in humans was defined to be irresponsible. The group agreed to initiate an international forum to address such concerns and harmonize regulations across countries.[114]

In November 2018, Jiankui He announced that he had edited two human embryos, to attempt to disable the gene for CCR5, which codes for a receptor that HIV uses to enter cells. He said that twin girls, Lulu and Nana, had been born a few weeks earlier. He said that the girls still carried functional copies of CCR5 along with disabled CCR5 (mosaicism) and were still vulnerable to HIV. The work was widely condemned as unethical, dangerous, and premature.[115] An international group of scientists called for a global moratorium on genetically editing human embryos.[116]

Policy regulations for the CRISPR-Cas9 system vary around the globe. In February 2016, British scientists were given permission by regulators to genetically modify human embryos by using CRISPR-Cas9 and related techniques. However, researchers were forbidden from implanting the embryos and the embryos were to be destroyed after seven days.[117]

The US has an elaborate, interdepartmental regulatory system to evaluate new genetically modified foods and crops. For example, the Agriculture Risk Protection Act of 2000 gives the USDA the authority to oversee the detection, control, eradication, suppression, prevention, or retardation of the spread of plant pests or noxious weeds to protect the agriculture, environment and economy of the US. The act regulates any genetically modified organism that utilizes the genome of a predefined "plant pest" or any plant not previously categorized.[118] In 2015, Yinong Yang successfully deactivated 16 specific genes in the white button mushroom, to make them non-browning. Since he had not added any foreign-species (transgenic) DNA to his organism, the mushroom could not be regulated by the USDA under Section 340.2.[119] Yang's white button mushroom was the first organism genetically modified with the CRISPR-Cas9 protein system to pass US regulation.[120] In 2016, the USDA sponsored a committee to consider future regulatory policy for upcoming genetic modification techniques. With the help of the US National Academies of Sciences, Engineering and Medicine, special interests groups met on April 15 to contemplate the possible advancements in genetic engineering within the next five years and any new regulations that might be needed as a result.[121] The FDA in 2017 proposed a rule that would classify genetic engineering modifications to animals as "animal drugs", subjecting them to strict regulation if offered for sale, and reducing the ability for individuals and small businesses to make them profitably.[122][123]

In China, where social conditions sharply contrast with the west, genetic diseases carry a heavy stigma.[124] This leaves China with fewer policy barriers to the use of this technology.[125][126]

In 2012, and 2013, CRISPR was a runner-up in Science Magazine's Breakthrough of the Year award. In 2015, it was the winner of that award.[81] CRISPR was named as one of MIT Technology Review's 10 breakthrough technologies in 2014 and 2016.[127][128] In 2016, Jennifer Doudna, Emmanuelle Charpentier, along with Rudolph Barrangou, Philippe Horvath, and Feng Zhang won the Gairdner International award. In 2017, Jennifer Doudna and Emmanuelle Charpentier were awarded the Japan Prize for their revolutionary invention of CRISPR-Cas9 in Tokyo, Japan. In 2016, Emmanuelle Charpentier, Jennifer Doudna, and Feng Zhang won the Tang Prize in Biopharmaceutical Science.[129]

Go here to read the rest:
CRISPR gene editing - Wikipedia

While ethicists debate the applications of blockbuster gene-editing tool Crispr in human healthcare, an inventor of the tool believes it has a more immediate application: improving our food.

"I think in the next five years the most profound thing we'll see in terms of Crispr's effects on people's everyday lives will be in the agricultural sector," Jennifer Doudna, the University of California Berkeley geneticist who unearthed Crispr in early experiments with bacteria in 2012, told Business Insider.

Crispr has dozens of potential uses, from treating diseases like sickle cell to certain inherited forms of blindness. The tool recently made headlines when a scientist in China reportedly used it to edit the DNA of a pair of twin baby girls.

Then there are Crispr's practical applications the kinds of things we might expect to see in places like grocery stores and farmers' fields within a decade, according to Doudna.

Crispr's appeal in food is straightforward: it's cheaper and easier than traditional breeding methods, including those that are used to make genetically modified crops (also known as GMOs) currently. It's also much more precise. Where traditional breeding methods hack away at a crop's genome with a dull blade, tools like Crispr slice and reshape with scalpel-like precision.

Want a mushroom that doesn't brown? A corn crop that yields more food per acre? Both already exist, though they haven't yet made it to consumers' plates. What about a strawberry with a longer shelf life or tomatoes that do a better job of staying on the vine?

"I think all of those things are coming relatively quickly," Doudna said.

Read more: The 10 people transforming healthcare

Work on Crispr'd produce has been ongoing for about half a decade, but it's only recently that US regulators have created a viable path for Crispr'd products to come to market.

Back in 2016, researchers at Penn State used Crispr to make mushrooms that don't brown. Last spring, gene-editing startup Pairwise scored $125 million from agricultural giant Monsanto to work on Crispr'd produce with the goal of getting it in grocery stores within the decade. A month later, Stefan Jansson, the chief of the plant physiology department at Sweden's Umea University, grew and ate the world's first Crispr'd kale.

More recently, several Silicon Valley startups have been experimenting with using Crispr to make lab-grown meat.

Read more: Startups backed by celebrities like Bill Gates are using Crispr to make meat without farms

Memphis Meats, a startup with backing from notable figures like Bill Gates and Richard Branson that has made real chicken strips and meatball prototypes from animal cells (and without killing any animals), is using the tool. So is New Age Meats, another San Francisco-based startup that aims to create real meat without slaughter.

Last spring, the US Department of Agriculture issued a new ruling on crops that exempts many Crispr-modified crops from the oversight that accompanies traditional GMOs. So long as the edited DNA in those crops could also have been created using traditional breeding techniques, the Crispr'd goods are not subject to those additional regulatory steps, according to the agency.

"With this approach, USDA seeks to allow innovation when there is no risk present," secretary of agriculture Sonny Perdue said in a statement. Genome editing tools like Crispr, he added, "will help farmers do what we aspire to do at USDA: do right and feed everyone."

Read more: A controversial technology could save us from starvation if we let it

Although several researchers and scientists have cheered the decision, many anti-GMO activists have not been pleased.

Despite the pushback, Doudna believes that Crispr'd food could help dispel some of the fear around GMOs and increase awareness about the role of science in agriculture.

"I hope this brings that discussion into a realm where we can talk about it in a logical way," she said. "Isn't it better to have technology that allows for precise manipulation of a plant genome, rather than relying on random changes?"

See the original post:
Jennifer Doudna: We will eat the first Crispr'd food In 5 ...

Cell Therapy World Asia 2019

Asia-Pacifics ONLY Cell Therapy Focused Regional Event!

Tokyo, Japan

Cell Therapy World Asia 2019 is bringing together Asias best of best in cell therapy development and manufacturing. This will be the most targeted and the only regional conference that will attract cell therapy companies in South Korea, Japan, China, India, Singapore, Taiwan and the rest of Asia to discuss and debate on best practices and innovations in this space.

Event Highlights200+Key Stakeholders from TOP Cell Therapy Companies 50+ Asia-Pacificcell therapy companies to attend 30+ Key opinion leaders to share their insights 20+ Hours of Networking 15+ Technology Showcase

What is in it for you?

Sales and Marketing Opportunities @ Cell Therapy WorldAsia 2019

To ensure your target audience in Korea and Asia gets to hear your product philosophy and successful case studies at the conference, its important to discuss with us about your potential involvement early! Get involved by taking your first step, contact:

Speaking OpportunitiesAarthi AsokanConference ProducerT: (65) 3109 0159E: aarthi.asokan@imapac.com

Sponsorship OpportunitiesMatthew YongBusiness Development ManagerT: (65) 3109 0123E: matthew.yong@imapac.com

Delegate & Media RegistrationAkanksha MittalMarketing ManagerT: (65) 3109 0158E: akanksha.mittal@imapac.com

See the original post:
Cell Therapy World Asia 2019 - IMAPAC - Imagine your impact

News Release

Wednesday, April 17, 2019

NIH scientists and funding contributed to development of experimental treatment

A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. Eight infants with the disorder, called X-linked severe combined immunodeficiency (X-SCID), received an experimental gene therapy co-developed by National Institutes of Health scientists. They experienced substantial improvements in immune system function and were growing normally up to two years after treatment. The new approach appears safer and more effective than previously tested gene-therapy strategies for X-SCID.

These interim results from the clinical trial, supported in part by NIH, were published today in The New England Journal of Medicine.

Infants with X-SCID, caused by mutations in the IL2RG gene, are highly susceptible to severe infections. If untreated, the disease is fatal, usually within the first year or two of life. Infants with X-SCID typically are treated with transplants of blood-forming stem cells, ideally from a genetically matched sibling. However, less than 20% of infants with the disease have such a donor. Those without a matched sibling typically receive transplants from a parent or other donor, which are lifesaving, but often only partially restore immunity. These patients require lifelong treatment and may continue to experience complex medical problems, including chronic infections.

"A diagnosis of X-linked severe combined immunodeficiency can be traumatic for families," said Anthony S. Fauci, M.D., director of NIHs National Institute of Allergy and Infectious Diseases (NIAID). These exciting new results suggest that gene therapy may be an effective treatment option for infants with this extremely serious condition, particularly those who lack an optimal donor for stem cell transplant. This advance offers them the hope of developing a wholly functional immune system and the chance to live a full, healthy life.

To restore immune function to those with X-SCID, scientists at NIAID and St. Jude Childrens Research Hospital in Memphis, Tennessee, developed an experimental gene therapy that involves inserting a normal copy of the IL2RG gene into the patients own blood-forming stem cells. The Phase 1/2 trial reported today enrolled eight infants aged 2 to 14 months who were newly diagnosed with X-SCID and lacked a genetically matched sibling donor. The study was conducted at St. Jude and the Benioff Childrens Hospital of the University of California, San Francisco. Encouraging early results from a separate NIAID-led study at the NIH Clinical Center informed the design of the study in infants. The NIH study is evaluating the gene therapy in older children and young adults with X-SCID who previously had received stem cell transplants.

The gene therapy approach involves first obtaining blood-forming stem cells from a patients bone marrow. Then, an engineered lentivirus that cannot cause illness is used as a carrier, or vector, to deliver the normal IL2RGgene to the cells. Finally, the stem cells are infused back into the patient, who has received a low dose of the chemotherapy medication busulfan to help the genetically corrected stem cells establish themselves in the bone marrow and begin producing new blood cells.

Normal numbers of multiple types of immune cells, including T cells, B cells and natural killer (NK) cells, developed within three to four months after gene therapy in seven of the eight infants. While the eighth participant initially had low numbers of T cells, the numbers greatly increased following a second infusion of the genetically modified stem cells. Viral and bacterial infections that participants had prior to treatment resolved afterwards. The experimental gene therapy was safe overall, according to the researchers, although some participants experienced expected side effects such as a low platelet count following chemotherapy.

"The broad scope of immune function that our gene therapy approach has restored to infants with X-SCID as well as to older children and young adults in our study at NIH is unprecedented," said Harry Malech, M.D., chief of the Genetic Immunotherapy Section in NIAIDs Laboratory of Clinical Immunology and Microbiology. Dr. Malech co-led the development of the lentiviral gene therapy approach with St. Judes Brian Sorrentino, M.D., who died in late 2018. These encouraging results would not have been possible without the efforts of my good friend and collaborator, the late Brian Sorrentino, who was instrumental in developing this treatment and bringing it into clinical trials, said Dr. Malech.

Compared with previously tested gene-therapy strategies for X-SCID, which used other vectors and chemotherapy regimens, the current approach appears safer and more effective. In these earlier studies, gene therapy restored T cell function but did not fully restore the function of other key immune cells, including B cells and NK cells. In the current study, not only did participants develop NK cells and B cells, but four infants were able to discontinue treatment with intravenous immunoglobulins infusions of antibodies to boost immunity. Three of the four developed antibody responses to childhood vaccinations an indication of robust B-cell function.

Moreover, some participants in certain early gene therapy studies later developed leukemia, which scientists suspect was because the vector activated genes that control cell growth. The lentiviral vector used in the study reported today is designed to avoid this outcome.

Researchers are continuing to monitor the infants who received the lentiviral gene therapy to evaluate the durability of immune reconstitution and assess potential long-term side effects of the treatment. They also are enrolling additional infants into the trial. The companion NIH trial evaluating the gene therapy in older children and young adults also is continuing to enroll participants.

The gene therapy trial in infants is funded by the American Lebanese Syrian Associated Charities (ALSAC), and by grants from the California Institute of Regenerative Medicine and the National Heart, Lung, and Blood Institute, part of NIH, under award number HL053749. The work also is supported by NIAID under award numbers AI00988 and AI082973, and by the Assisi Foundation of Memphis. More information about the trial in infants is available on ClinicalTrials.gov using identifier NCT01512888. More information about the companion trial evaluating the treatment in older children and young adults is available using ClinicalTrials.gov identifier NCT01306019.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

E Mamcarz et al. Lentiviral gene therapy with low dose busulfan for infants with X-SCID. The New England Journal of Medicine DOI: 10.1056/NEJMoa1815408 (2019).

###

Read the original here:
Gene therapy restores immunity in infants with rare ...

Researchers from St. Jude Childrens Research Hospital have cured babies with bubble boy disease through gene therapy. Angela Gosnell, Knoxville News Sentinel

MEMPHIS, Tenn. Researchers from St. Jude Childrens Research Hospital have cured babies with bubble boy disease through gene therapy involving a re-engineeredvirus, according to a newly published study.

St. Jude performed the therapy oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1) a genetic condition also known as "bubble boy" disease according to a study published in the New England Journal of Medicine's April 18 issue.

The diseaseprevents babies from developing an immune system to fight even routine infections.In January 2018, St. Jude researchers reported that babies in the trial developed fully functioning immune systems but would be monitored further to confirm its long-term benefits.

Corresponding authors Dr. Ewelina Mamcarz and Dr. Stephen Gottschalk from St. Jude Children's Research Hospital. St. Jude performed a new therapy oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1), a genetic condition called "bubble boy" disease, according to a study published in the New England Journal of Medicine's April 18 issue.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

Previous infections cleared in all infants, and all continued to grow normally, the study said of the results.

St. Jude and UCSF Benioff Childrens Hospital San Francisco treated the children enrolled in the clinical trial with gene therapy developed by St. Judes Brian Sorrentino, the studys senior author,who led groundbreaking gene therapy research before his death in November at 60 years old.

Brian Sorrentino(Photo: Courtesy of Memorial Park Funeral Home)

James Downing, CEO of St. Jude Children's Research Hospital, said it was the lifelong ambition of Sorrentino, a survivor of pediatric cancer, to develop a cure.

Were comfortable, I think, at this point stating this is a cure, Downing said. Only time will say this will be a durable, lifelong cure.

After the therapy, the babies received their standard vaccinations and are now living a normal life with fully functioning immune systems, St. Jude says. Ten infants have received the therapy so far.

Study co-author Stephen Gottschalk, chair of the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy, said the researchers hope the therapy will be a template for treating other blood disorders.

Newborns with bubble boy disease, caused by a mutation inside a specific gene,must be placed inprotective isolation because they lack a proper immune system. Contact with the outside world is a major infection risk.

Perhaps the most well-known person with the disease was David Vetter, who died in 1984 at 12 years old. He helped inspire the 1976 movie "The Boy in the Plastic Bubble."

David Vetter had to stay inside a bubble in Houston on Dec. 17, 1976. Vetter was born with a genetic disorder leaving him no natural immunity against disease. Vetter died in 1984.(Photo: AP)

Most with the disease die by age 2 without treatment.

This disease is called bubble boy disease because babies had to be kept in special plastic chambers to protect them from infections, said first and corresponding author Ewelina Mamcarzof the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy. We dont have these chambers now, we are more advanced, but we need to protect them from infections as simple as a common cold virus (that) can kill them.

The patients came to researchers between 2 and 14 months of age, Mamcarz said, with severe life-threatening infections.

The gene therapy works like this: A deactivated virus is inserted into the patients bone marrow, which deliversthe correct gene copy into blood stem cells, replacing the defective one. These cells are then frozen and undergo testing.

This virus is able to effectively deliver a healthy copy of the gene into a stem cell in a way that was not possible before, Mamcarz said.

The patient then receives two days of low-dose busulfan, a chemotherapy drug that makes space in the marrow for the stem cells to grow, and the cells are then infused back into the patient.

Dr. Ewelina Mamcarz, first and corresponding author of a study published in the New England Journal of Medicine about a therapy performed at St. Jude Children's Research Hospital oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1), a genetic condition called "bubble boy" disease.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

It takes about 10 days from the time the cells are taken outto when they are infused into the patient, Mamcarz said.

The proper immune cells were found within three months of the treatment in all but one patient, who needed a second dose of gene therapy, St. Jude says.

This novel approach has shown really outstanding results for the infants, Downing said. The treatment has fully restored the immune system in these patients, which wasnt possible before, and has no immediate side effects.

The gene therapy developed and produced at St. Jude differs from previous gene replacement efforts in part by not activating adjacent genes that could cause leukemia. The viruses are equipped with insulators to block that accidental activation.

Past gene therapy did not have insulators, which inadvertently caused leukemia, Gottschalk said.

Gael Jesus Pino Alva, 2, and his mother, Giannina Alva. Gael was treated with a new therapy designed to fight X-linked severe combined immunodeficiency (SCID-X1), a genetic condition known as "bubble boy" disease, at St. Jude Children's Research Hospital.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

Current treatments for bubble boydisease are limited. Bone marrow transplants from compatible sibling donors are the best bet, but most patientslack a properdonor.

Mamcarz said researchers would like to treat more patients and follow them for longer periods of time to see if the gene therapy performed in the clinicaltrial can truly be used as an upfront treatment, and it's still too early to determine costs.

But the results from the research are a first, and their approach could be used to eventually treatother disorders like sickle cell disease, she said.

The kids are cured because for the first time, we are able to restore all three types of cells that constitute a full immune system: T cells, B cells and NK cells, Mamcarz said. Our patients are able to generate a healthy, fully functioning immune system. That is the first for gene therapy.

Autoplay

Show Thumbnails

Show Captions

Downingsaid the search for a cure has been a journey spanning more than a decade. Early gene therapy studies with the viral vectorsled to leukemia, he said, causing the work to stall. But Sorrentino pushed on.

Brian Sorrentino decided we really needed to produce vectors we could trust in not inducing leukemia, Downingsaid.

The patients' quality of life following the treatmentshows theyindeed found a cure, Downing said.

The question will become, Will it be a durable cure? Will it last 10, 20, 50 years for these children? And only time will tell," he said.

Follow Max Garland on Twitter:@MaxGarlandTypes.

Autoplay

Show Thumbnails

Show Captions

Read or Share this story: https://www.usatoday.com/story/news/nation/2019/04/17/bubble-boy-disease-cured-st-jude-hospital/3502388002/

Excerpt from:
Bubble boy disease: Doctors successfully treat SCID-X1 ...