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Xconomy: SMA Moment: Will Gene Therapy Shift Treatment …


This is a big moment for people diagnosed with spinal muscular atrophy, or SMA, a rare and potentially lethal genetic disorder that destroys muscles. For decades, there was no way to change the trajectory of their disease. They now have one marketed medicine, and this month, chances are theyll have another: a gene therapy that promises a long-lasting treatment, if not an outright cure, through a one-time dose.

This weekend at the annual American Academy of Neurology meeting, patients, their families, and doctors will gain more insight about the gene therapy, Zolgensma, which is owned by drug giant Novartis (NYSE: NVS), and how it might stack up against the approved medicine nusinersen (Spinraza), owned by Biogen (NASDAQ: BIIB). They can also look forward to the latest clinical data from an SMA drug, risdiplam, from Roche, that, if successful, would be the first that a patient could take orallya big deal, because the most severe cases of SMA are in newborns and infants, and Spinraza requires chronic spinal infusions.

Its ridiculously exciting, says Jahannaz Dastgir, a pediatric neurologist at Goryeb Childrens Hospital in Morristown, NJ. Its a great time to be a doctor.

All the new information comes amid anticipation that the FDA this month will approve Zolgensma. With the agencys green light, it would be the second approved gene therapy in the US, and one of just a handful around the world.

Zolgensma will also face something other gene therapies havent: competition. Approved in late 2016, Spinraza has already proven effective and, after early hiccups, has become a big seller for the beleaguered Biogen, with $1.7 billion in sales in 2018 and $518 million in the first quarter of 2019.

Novartis has high hopes for Zolgensma, too, having paid $8.7 billion to buy its developer AveXis in 2018. Its success or failure will be a bellwether for the economics of gene therapy. (Nationwide Childrens Hospital in Columbus, OH, where the therapy was developed, will be watching closely too.)

If both Spinraza and Zolgensma are available, doctors, payers, patients, and their families will face tough medical, logistical, and economic decisions. So far, Spinraza has far more data to support it. But it has a $750,000 first-year price tag and requires a few spinal infusions a year at a $375,000 annual cost thereafter, for life. Zolgensma could cost $1 million or more (Novartis has hinted much more) for a single dose, theoretically a bargain if it saves lives and negates downstream medical and social costs that SMA patients and their families would otherwise face.

A recent survey of 30 physicians in the US and Europe by the investment bank Jefferies suggested that a majority of newly diagnosed SMA patients, as well as those currently on Spinraza, will get Zolgensma. Jefferies predicts $2.6 billion in peak sales for Zolgensma.

Its possible that the best results could come from combination therapy, but that hasnt been tested and the costs would be exorbitant.

Alex Fay, a pediatric neurologist at UCSF Benioff Childrens Hospital in San Francisco, CA, says he would be hesitant to switch patients if Spinraza is well tolerated and working. Adding more complication, says Fay, is the fast progress of the disease. Those decisions are going to have to be made pretty quickly, says Fay.

Information revealed soon could make those decisions easier. Babies diagnosed with Type 1 SMA, the most common and deadly form of the disease, often die before the age of two. Type 2 patients may never be able to walk, while Type 3 patients can walk initially before losing strength later in life. In all types, it seems that the earlier the treatment, the more benefit.

Thus far, all public Zolgensma data have been in babies with Type 1. There will be more of that at AAN. Studies presented at the meeting this weekend will also, for the first time, reveal Zolgensmas effects on more moderate forms of SMA, and in patients who havent shown symptoms yet. Those data could help determine Zolgensmas eventual reach.

More than 7,500 patients across several SMA types have now received Spinraza, some as long as six years. Biogen recently used that experience to turn up the heat on Novartis.

Last week it published results in Neurology, the AANs medical journal, from a long-term study in later-onset patients, aged 5 to 19, who were likely to develop Type 2 or Type 3 SMA. Each group showed improvements on tests of motor function; historical data suggest they should get weaker. A couple patients with Type 3 SMA even regained the ability to walk during the trial, Biogen said.

Citing the study and other data supporting Spinraza, Biogen CEO Michel Vounatsos was adamant on an April 24 conference call that the drug will remain the standard of care for SMA for years to come.

The presentations this weekend will shed more light on the potential benefits and risks of the new world of SMA treatments, but there will plenty of questions left unanswered. Here we break down four key SMA topics that will be under intense discussion.

Fast Access: SMA is a battle against time. Neurons die and dont come back. Muscles waste away and are replaced by scar tissue and fat. The muscle-wasting is particularly fast for babies with Type 1. Time to treatment is of the essence. They may never Next Page

Ben Fidler is Xconomy's Deputy Editor, Biotechnology. You can e-mail him at

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What are the ethical issues surrounding gene therapy …

Because gene therapy involves making changes to the bodys set of basic instructions, it raises many unique ethical concerns. The ethical questions surrounding gene therapy include:

How can good and bad uses of gene therapy be distinguished?

Who decides which traits are normal and which constitute a disability or disorder?

Will the high costs of gene therapy make it available only to the wealthy?

Could the widespread use of gene therapy make society less accepting of people who are different?

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?

Current gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy cannot be passed to a persons children. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the inserted gene to be passed to future generations. This approach is known as germline gene therapy.

The idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they cant choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.

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CRISPR Therapeutics Provides Business Update and Reports …

-Enrollment ongoing in Phase 1/2 clinical trials of CTX001 for patients with severe hemoglobinopathies-

-IND and CTA approved for CTX110, wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies-

-On track to initiate Phase 1/2 clinical trial for CTX110 in 1H 2019-

-$437.5 million in cash as of March 31, 2019-

ZUG, Switzerland and CAMBRIDGE, Mass., April 29, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the first quarter ended March 31, 2019.

This past quarter, we began an important new period for CRISPR Therapeutics with the treatment of the first patient in our clinical trial for CTX001 in hemoglobinopathies, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. This is a significant landmark for the Company and we continue to enroll patients in our trials for both beta thalassemia and sickle cell disease. With the acceptance of our IND and CTA for CTX110, we look forward to the initiation of our clinical trials for our allogeneic CAR-T programs in the near-term and hope to bring other CAR-T programs to the clinic in the next six to twelve months.

Recent Highlights and Outlook

First Quarter 2019 Financial Results

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof and the timing of commencement of clinical trials), development timelines and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators; (ii) the number of patients that will be evaluated, the anticipated date by which enrollment will be completed and the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; (iii) the scope and timing of ongoing and potential future clinical trials; (iv) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties; (v) the sufficiency of CRISPR Therapeutics cash resources; and (vi) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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CRISPR Therapeutics AGCondensed Consolidated Statements of Operations(Unaudited, In thousands except share data and per share data)

CRISPR Therapeutics AGCondensed Consolidated Balance Sheets Data(Unaudited, in thousands)

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Media Contact:Jennifer PaganelliWCG on behalf of

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Genetic Testing FAQ | NHGRI

Benefits: Genetic testing may be beneficial whether the test identifies a mutation or not. For some people, test results serve as a relief, eliminating some of the uncertainty surrounding their health. These results may also help doctors make recommendations for treatment or monitoring, and give people more information for making decisions about their and their family's health, allowing them to take steps to lower his/her chance of developing a disease. For example, as the result of such a finding, someone could be screened earlier and more frequently for the disease and/or could make changes to health habits like diet and exercise. Such a genetic test result can lower a person's feelings of uncertainty, and this information can also help people to make informed choices about their future, such as whether to have a baby.

Drawbacks: Genetic testing has a generally low risk of negatively impacting your physical health. However, it can be difficult financially or emotionally to find out your results.

Emotional: Learning that you or someone in your family has or is at risk for a disease can be scary. Some people can also feel guilty, angry, anxious, or depressed when they find out their results.

Financial: Genetic testing can cost anywhere from less than $100 to more than $2,000. Health insurance companies may cover part or all of the cost of testing.

Many people are worried about discrimination based on their genetic test results. In 2008, Congress enacted the Genetic Information Nondiscrimination Act (GINA) to protect people from discrimination by their health insurance provider or employer. GINA does not apply to long-term care, disability, or life insurance providers. (For more information about genetic discrimination and GINA, see

Limitations of testing: Genetic testing cannot tell you everything about inherited diseases. For example, a positive result does not always mean you will develop a disease, and it is hard to predict how severe symptoms may be. Geneticists and genetic counselors can talk more specifically about what a particular test will or will not tell you, and can help you decide whether to undergo testing.

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CI MEMBER | Cryonics Institute


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.


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 "

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CI MEMBER | Cryonics Institute

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Vancouver Stem Cell Treatment Centre | Stem Cells

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).

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Gene therapy reverses rare immune disorder | National …

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.

Gene therapy reverses rare immune disorder | National ...

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Gene Therapy Market Emerging Trends, Growth and New …

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

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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 @

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.

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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.

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Menopause hormone therapy and your heart – Mayo Clinic

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.


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What Are Induced Pluripotent Stem Cells? – Stem Cell: The …

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.

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Stem Cell Basics VII. |

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

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Stem Cell Basics VII. |

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Why are Adult Stem Cells Important? Boston Children’s …

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.

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Creating Embryonic Stem Cells Without Embryo Destruction

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.

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Disease Prevention & Treatment 5th Edition: Life Extension …

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Frequently Asked Questions | Cryonics Institute

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 We're more than happy to help.

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International Cell and Gene therapy Conferences | Gene …

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.

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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.

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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.

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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.

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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.

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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.

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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

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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Stem Cell Key Terms | California’s Stem Cell Agency

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.

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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

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Stem Cell Key Terms | California's Stem Cell Agency

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Advance Stem Cell Therapy in India | Stem Cell Treatment …

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.


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

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NIH launches largest-ever study of breast cancer genetics …

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 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

NIHTurning Discovery Into Health

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


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Serious question about female genetics? | Yahoo Answers

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.

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Serious question about female genetics? | Yahoo Answers

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Gene therapy might be a cure for "bubble boy disease …

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.

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Highmark Health Blog | Gene Therapy Research: Dr. Passineau

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.

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Gene Therapy Questions | FAQs – Dana-Farber/Boston …

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

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Gene Therapy Questions | FAQs - Dana-Farber/Boston ...

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