Archive for July, 2017

Advances in medical imaging enable bespoke tissues and organs to be developed for transplant or engraftment with remarkable resolution and definition using 3D bioprinting. The incorporation of stem cell therapies into these 3D tissue constructs is incredibly promising for the delivery of pioneering stem cell regenerative therapies. Typically, 3D bioprinting requires use of a biomaterial to aid with deposition, which can cause negative host responses. To avoid such problems, US researchers have developed a biomaterial-free cardiac patch (Scientific Reports 7 4566).

Heart disease affects thousands of people every year and effective repair of cardiac tissue would reduce a large medical health care burden. Researchers from the Narutoshi Hibino lab at Johns Hopkins Hospital and Johns Hopkins University have devised a 3D-bioprinting procedure that allows for the biofabrication of cardiac tissue patches to deliver regenerative stem cells, without using biomaterials. The process utilises aggregated balls of cardiac cells (cardiospheroids), which are directly printed into a cardiac patch construct. The cardiospheroids are identified, picked up by a vacuum and bioprinted directly onto a needle microarray (a video of the 3D-bioprinting process used is available from JOVE). This novel method allows the patch to be constructed with cells alone and will avoid detrimental effects induced by biomaterial grafts.

Stem cell techniques for tissue regeneration typically rely on biomaterial scaffolds to provide structure and support for cells during grafting. The grafting or introduction of biomaterials to a patient induces an immune response, or can create scar tissue from the graft, potentially damaging the region of tissue intended to be repaired. Through developing a biomaterial-free graft, it is possible to avoid these detrimental factors. And by using a patient's own stem cells it is possible to create native tissue that is fully biocompatible.

3D bioprinting was crucial to the development of effective cardiac patches, with specific spatial distribution being crucial to mechanical integrity. Cardiospheres without specific placement to overlap with other cardiospheres disintegrated after removal from the needle array; although partially disintegrated regions were able to fuse back together eventually. This effect removed the structural definition of the patch, negating the advantages of using bioprinting for developing a cardiac patch of specified dimensions.

The researchers grafted patches onto rat hearts and after a week saw signs of blood vessel formation, with viable cells and red blood cells present in the cardiac patch. Tissue protein stains showed that collagen was present in the patch, indicating the deposition of a native extracellular matrix from the cells, crucial to cell integration. Further staining showed the presence of human nucleic acid in rat tissue, implying that the human cell derived patch had successfully grafted with the rat tissue.

This biomaterial-free cardiac patch was developed using pluripotent cardiomyocyte stem cells, cardiac fibroblasts and human umbilical vein endothelial cells (HUVECs), which were aggregated into cardiospheroids for bioprinting. Cardiospheroids were able to develop a functional phenotype after 48 hours, with spontaneous beating and electrical conductivity a week after bioprinting. Cardiomyocytes alone were not able to reproduce this functional phenotype.

This process demonstrates a novel approach to eliminating biomaterial-induced damage. Further development of this 3D bioprinting technique in conjunction with stem cell therapies could progress biomaterial-free cardiac patches into the popular domain.

3D printers help build a better cranial nerve4D bioprinting: adding dynamic actuationThe first laser-printed 3D cellular tubes3D-printed polymer stents evolve

Geoffrey Potjewyd is a PhD Student contributor to medicalphysicsweb, working in the Division of Neuroscience and Experimental Psychology, as part of the CDT in Regenerative Medicine at The University of Manchester. He is studying the neurovascular unit in relation to vascular dementia and Alzheimer's disease, using biofabrication, biomaterials and stem cell based techniques.

See more here:
3D bioprinted cardiac patches are biomaterial free - Medical Physics Web (subscription)

Cellular Dynamics International, the stem cell company founded by UW-Madison stem cell pioneer James Thomson, is backing off on moving its headquarters to a big, new building in Verona and will stay in Madison, at least for now, as it prepares to push forward with its first potential stem cell-based treatment in early 2018.

CDI president Kaz Hirao said Thursday the company is shelving plans to shift operations to a $40 million, 133,700-square-foot building that was to be built for CDI on Kettle Moraine Trail in Verona. The building was expected to house 280 employees, with so-called clean rooms, quality-control labs, processing rooms and offices.

Instead, CDIs main offices and labs will remain at 525 Science Drive in University Research Park and the company will remodel an existing building whose site has not yet been determined to house several clean rooms that will meet government standards for manufacturing stem cells for use in clinical drug trials.

Fujifilm (CDIs parent company) has a very strong commitment and wants to see (the) Madison (site) grow in the future. Strategy-wise, that has not changed, Hirao said. Madison has a great ecosystem for our businesses.

He said the National Eye Institute plans to submit an application to the U.S. Food and Drug Administration in January 2018 for a retinal cell therapy it has been developing with CDI for age-related macular degeneration, an eye disease that can lead to blindness. The National Eye Institute has conducted animal studies on the drug, Hirao said.

It is the first of a series of stem cell-based drugs the company is working on. CDI expects to file investigational new drug applications for treating Parkinsons disease and for cardiac disease in 2019, he said.

In order to make stem cells that meet government standards for use in human clinical trials, Hirao said the company will establish clean rooms that meet regulations for current good manufacturing practices. He said he expects to designate a location in the next month or two, within about a 15-minute drive of CDI headquarters, to handle the companys stem cell manufacturing needs for the immediate future.

Next year, CDI will review its plans again, Hirao said, and will again consider a move to a larger, consolidated building. If it decides to go ahead with that, Verona would be one of the preferred options, he said.

CDI had obtained up to $6 million in financial incentives from the city of Verona for the building that was to be built and owned by developer John K. Livesey.

Verona planning and development director Adam Sayre called CDIs decision to pull back on the plans unfortunate, but said city officials will keep in contact with Cellular Dynamics over the coming months.

The city would continue to welcome them with open arms, Sayre said. Well see what the next year brings.

At University Research Park, CDI occupies about 55,000 square feet, director Aaron Olver said. Weve recently provided CDI with some additional space to help them grow, he said.

CDI is one of the true gems among companies powered by UW-Madison research, and we would certainly do anything we could to help them find clean room space to continue their work, Olver said.

Founded in 2004, CDI was acquired by Fujifilm Holdings Corp. for $307 million in April 2015.

The company has 165 employees, including about 125 in Madison. Hirao said he expects to add employees, but said its too soon to estimate how many, or how quickly the company will grow.

Excerpt from:
CDI ditches move to Verona - Madison.com

Marie Grim of Langley is looking for 100 people between the ages of 17 and 35 who are willing to take a few moments to have their cheeks swabbed.

Theres no pain, no fuss, she said.

And you could save someones life, anywhere in the world.

You could match with someone in Japan.

The campaign to sign up more potential stem cell donors, people who are willing to allow DNA samples to taken using cotton swabs, was inspired by the experience of her sister-in-law.

Cloverdale resident Tania Grim, a mother of four was diagnosed with leukemia in January.

She had to wait several months before a compatible donor was found whose stem cells will be used to replace bone marrow and abnormal white blood cells eradicated by a combination of chemotherapy and radiation.

We have been on quite the journey, Marie said.

I have sat with her at appointments and heard others get news of their donor while she had not.

Now that Tania has her donor, Marie would like to improve the odds for other families.

She already has a location and tentative date to collect the swabs September 8 at Immanuel Christian Reformed Church in Langley if she can round up enough donors.

Tania, who is preparing for her stem cell procedure in September, urged prospective stem cell contributors to sign up.

I am so grateful that the word is being spread about the huge need for donors, Tania said.

It is a very simple thing to do that can save a life.

If you are the right age to be a donor, you can contact Marie at 604-530-1326 or by email at mariegrim@hotmail.com.

Interested donors can also contact Canadian Blood Services directly at https://blood.ca/en.

More than 390,000 Canadians have joined the OneMatch Stem Cell and Marrow Network registry maintained by Canadian Blood Services, volunteering to be stem cell donors for any patient in need of a transplant, anywhere in the world.

Right now, the agency says about 70 per cent of eligible donors on the registry are Caucasian, which means the odds of finding match for other ethnicities, such as Canadians with indigenous, Asian or African heritage, are not good.

The Canadian registry connects to an international network established by the World Marrow Donor Association (WMDA) that has access to over 28 million donors in over 53 countries.

Not everyone who registers will be matched to a patient and asked to donate, but each registrant provides hope for those waiting, a message posted to the agency website states.

A person could be a match within a few months of registering, a year later or even seven years later.

If a volunteer donor is found to be a match, they face a relatively minor surgical procedure and can expect to make a quick recovery.

The agency says over 80 diseases and disorders can be treated with a stem cell transplant.

There are hundreds of patients in Canada waiting for a match, but only half of them find a match.

Patients are more likely to find a matching donor from within their own ethnic group.

The odds of family members matching is extremely slight, the agency said, which is why it does not support donor drives targeting relatives.

RELATED STORY: Surrey teen rallies stem cell donors to help with desperate need for South Asians

dan.ferguson@langleytimes.com

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Drive for stem cell donors in Langley - Surrey Now-Leader

Illustration incorporating gene-expression maps and cell images from the new research. Credit: University of Michigan

Every day, 17 million HIV-infected people around the world swallow pills that keep the virus inside them at bay.

That is, as long as they swallow those pills every day for the rest of their life.

But no matter how many drugs they take, they'll always have the virus in them, lurking in their white blood cells like a fugitive from justice.

And if they ever stop, HIV will come out of hiding and bring down their immune system from the inside out, causing the disease known as AIDS and potentially spreading to others before killing them.

Now, new research into HIV's hiding places reveals new clues about exactly how it persists in the body for years. The discovery could speed the search for drugs that can flush HIV out of its long-term hideouts and cure an infection for good.

In a new paper in PLoS Pathogens, a team led by University of Michigan researcher Kathleen Collins, M.D., Ph.D. reports that HIV hides in more types of bone marrow cells than previously thought - and that when these cells divide, they can pass the virus's genetic material down to their "daughter" cells intact.

This keeps the infection going for years, without tipping off the armed guards of the immune system.

Collins and her colleagues made the discovery in bone marrow samples donated by dozens of long-term HIV patients treated at U-M's academic medical center, Michigan Medicine, and at Henry Ford Health System in Detroit.

Using funding from the National Institutes of Health, they found that HIV can hide in hematopoietic progenitor cells (HPCs), which also serve as the parents of new blood cells that replace worn-out ones on a regular basis. HIV tricks the cells into incorporating the virus's genetic material into the cells' own DNA.

"Looking for the cells that harbor functional HIV is like searching for a needle in a haystack. Our new results expand our understanding of the type of cells that can do it," says Collins, a professor of Microbiology and Immunology and of Infectious Disease at the U-M Medical School. "It's like a cancer biology problem, only the 'mutation' in the cells is the inserted viral genome."

HPCs are made by hematopoietic stem cells, the "master cells" of blood production found in the marrow. Previous research had shown that HIV can hide for years in the bone marrow.

But it was not known whether the virus persisted only in stem cells or whether the reservoir could include more differentiated progenitor cells. Demonstrating that progenitor cells form a long-lived reservoir of virus expands the number of cell types that need to be targeted.

By demonstrating that HIV genetic material can lurk in blood progenitor cells, the researchers extend other recent studies indicating that such cells can live for years, says Collins, whose lab team included lead author Nadia Sebastian, a U-M M.D./Ph.D. student.

She notes that from the point of view of the virus, finding a harbor in this kind of cell means it can hedge its bets, giving it a chance at survival and eventual reproduction if its host's defenses weaken. The virus that causes chicken pox - varicella - also does this, hiding out in nerve cells just under the skin for years until it awakens and causes the painful condition called shingles.

Knowing exactly what cells harbor HIV over the long-term is crucial to battling persistent infections. Other research has focused on the T cells that carry out key immune system functions.

"Having established this, now we're poised to ask if we can treat HIV infection by targeting hematopoietic progenitor cells," she explains. The team is evaluating potential drugs that could kill just these cells.

The research team on the new paper also includes former U-M stem cell researcher Sean Morrison, Ph.D., who now leads a research center at the University of Texas Southwestern Medical Center. Morrison's lab uses mice as a model to study stem and progenitor cells.

They find in the new paper that in order for HIV to infect a progenitor cell, that cell must have a type of receptor on its surface, called CD4, that the virus can attach to. Additionally, the researchers show that two subtypes of HIV can infect these cells: those that use the CXCR4 co-receptor to enter cells as well as those that use CCR5, which expands the types of HIVs that can potentially cause reservoirs.

Finding those progenitor cells in the marrow of the human patients who agreed to undergo a biopsy for the sake of pure research was tricky, Collins says. But thanks to them, researchers are a step closer to a day when HIV infection is no longer a life sentence for millions of people around the world.

"Moving from the state we're in, where patients will always have to be on these drugs, to a better form of therapy where they can stop, would have a huge effect," she says. "Today's medications have side effects, as well as financial costs. To get to the next step, we need to target the types of cells that form a latent infection, including these progenitor cells."

Explore further: Scientists find that persistent infections in mice exhaust progenitors of all blood cells

More information: Nadia T. Sebastian et al, CD4 is expressed on a heterogeneous subset of hematopoietic progenitors, which persistently harbor CXCR4 and CCR5-tropic HIV proviral genomes in vivo, PLOS Pathogens (2017). DOI: 10.1371/journal.ppat.1006509

View original post here:
Cells that stand in the way of HIV cure: Discovery expands understanding of marrow's role - Medical Xpress

Its the fourth most common cancer for women in the U.S., but doctors say 80 percent of the time endometrial cancer is caught in stage one.

Dr. Fadi Abu Shahin, a gynecologic oncologist on the medical staff of Lee Health, says endometrial cancer can be caused by age, genetics, or medication. Endometrial cancer is cancer of the endometrium, which is the lining of the uterus on the inside. The most common is what we call unopposed estrogen where the womans body is producing estrogen only and not producing progesterone to counter act the effects of estrogen in the uterus.

That can lead to overgrowth of the lining of the uterus and can become cancerous over time. Women who are at risk for endometrial cancer may have a genetic predisposition or polycystic ovarian syndrome, where they dont ovulate regularly. In both cases doctors may prescribe birth control or hormone therapy as a preventative. The best treatment for those women is to put them on birth control pills or some form of hormonal therapy to prevent and protect the uterus from exposure to estrogen, said Dr. Abu Shahin.

Theres no regular screening for endometrial cancer, but doctors say if women experience symptoms they need to call their physician. In women after menopause any bleeding is abnormal. In women before menopause any irregularity in their periods or bleeding outside their period is abnormal, said Dr. Abu Shahin.

Endometrial cancer is the most common gynecologic cancer in women. If women are at risk, doctors may use genetic screening, biopsies, or ultrasounds to look for precancerous changes in the uterus. 70 to 80 percent of all women with endometrial cancer are diagnosed at stage one, which means the cancer was contained to the uterus, which means its highly curable, said Dr. Abu Shahin.

Patients may undergo a hysterectomy or choose to have only the high risk lymph nodes removed in a shorter, less invasive procedure.

Original post:
Health Matters: Detecting and Treating Endometrial Cancer - NBC2 News

Jennifer Brea, who has Chronic fatigue syndrome/myalgic encephalomyelitis,reveals her story in her film, Unrest.

By Giorgia GuglielmiJul. 31, 2017 , 3:00 PM

After a 5-month road trip across Asia in 2010, 22-year-old college graduate Matthew Lazell-Fairman started feeling constantly tired, his muscles sore and head aching. A doctor recommended getting a gym membership, but after the first training session, Lazell-Fairmans body crashed: He was so exhausted he couldnt go to work as a paralegal for the Federal Trade Commission in Washington, D.C., for days. Lazell-Fairman has never fully recovered. He can now do a few hours of light activitycooking, for exampleper day but has to spend the rest of his time lying flat in bed.

Lazell-Fairman is among the estimated 17 million people worldwide with chronic fatigue syndrome (CFS), a disease whose trigger is unknown and for which there are neither standard diagnostic tools nor effective treatments. In the largest study of its kind, researchers have now found that the blood levels of immune molecules that cause flulike symptoms such as fever and fatigue track the severity of symptoms in people who have received a diagnosis of CFS. The results may provide insight into the cause of the mysterious illness, or at least provide a way of gauging its progress and evaluating treatments.

This work is another strong piece of evidence that there is a biologic dysfunction at the root of the disease, says Mady Hornig, a physician scientist at Columbia University whose research has also identified potential biomarkers for CFS.

People with CFSmany patients and advocates prefer to call the condition myalgic encephalomyelitis (ME) because of the complex set of symptoms that are not limited to fatigueexperience prolonged, extreme exhaustion that doesnt improve with rest. The fatigue may worsen with physical or mental activity and often comes with brain fog, a feeling of mental clouding, and sensitivity to noise, light, or other stimuli like taste and smell. Patients may also have memory impairment, muscle pain, and gut problems such as diarrhea, bloating, and nausea.

For years, CFS had been dismissed as a psychological disorder, and some physicians advocated treating it primarily with psychotherapy or a gradual increase in physical activity. But many researchers, and funding agencies such as the National Institutes of Health, increasingly view it as a physiological problem. Its a medical mystery, says Mark Davis, an immunologist at Stanford University in Palo Alto, California.

For this reason, Davis, together with Stanford immunologist Jose Montoya and their teams, recently evaluated whether an imbalance of the immune system may trigger CFS/ME, as previous studies have suggested. By analyzing the blood of 192 people who had met one of the established criteria for CFS/ME diagnosis and 392 healthy individuals, the team found that the levels of 17 cytokines, substances produced by immune cells in response to infection, correlated with disease severity. They were higher in patients with the severest symptoms than in patients with milder symptoms or healthy people. In patients with the mildest symptoms, the levels of those same cytokines were lower than in healthy people, and in patients with moderate symptoms they were comparable to individuals with no disease. Of these 17 immune molecules, the vast majority is known to stimulate inflammation and produce flulike symptoms, the researchers report today in the Proceedings of the National Academy of Sciences.

I found surprising that so many cytokines are altered in the patients, Davis says. It seems like the disease is leaving no cytokine untouched. The work adds to our understanding that there are complex alterations of the immune system, Hornig says.

A few immune molecules drew particular attention because their levels didnt correlate with disease severity. The researchers have found that the blood level of TGF-, a cytokine that is involved in myriad biological processes, such as inflammation and cancer, was higher in CFS/ME patients, regardless of the severity of their symptoms, than in healthy people. And the blood levels of resistin, a hormone produced by immune cells, were lower in patients compared with individuals with no disease.

These molecules might drive the disorder, scientists speculate, but they could also simply reflect the patients immune system fighting back against an inflammation of different origin. These are deliberately agnostic assays, Davis says. First you look for correlation and then you try to understand why these things correlate with the disease.

Nonetheless, the new study is a tremendous step forward, says Gordon Broderick, a systems biologist at Rochester General Hospital in New York. Being able to examine so many CFS/ME patients and identify which immune molecules are associated with more intense symptoms is a big deal, he says.

Although it is important to rule out that altered levels of cytokines arent linked to factors such as allergies or sex hormones, which can also influence the immune system, the large number of patients showing an imbalance in these immune molecules suggests that it is either a cause or result of CFS/ME, Broderick says.

In the future, the scientists hope to use these immune molecules as diagnostic tools for the puzzling condition, and also figure out the role of cytokines and the immune system in the disease. I hope to see more research along those lines, Hornig says. Theres a lot of patients waiting for some answers.

See more here:
More clues link immune system imbalance with chronic fatigue syndrome - Science Magazine

1. Cross-sex hormone therapy (CSHT) may be associated with worsening cardiovascular risk factors in transgender men.

2. CSHT may not result in increases in cardiovascular morbidity or mortality.

Evidence Rating Level: 2 (Good)

Study Rundown: Understanding the unique health care needs of the transgender population is highly important; approximately 1.4 million persons identify as transgender in the United States. One important area of study involves the role of sex hormonesincluding hormone blockers and cross-sex hormone therapyand cardiovascular disease (CVD). Sex hormones may be used to help transgender persons fully realize their gender identity. Despite the importance of this area of research, one of the barriers to providing appropriate care to transgender persons is the lack of physician training and clinical guidelines within this area. Additionally, there is limited health care research within this population. The authors of this study, therefore, conducted a systematic review that highlights research on the association between hormone therapy and CVD in cisgender adults, in addition to summarizing the association between CSHT and CVD in transgender adults. This study has several limitations. First, many studies included in this review focus on younger persons, limiting the generalizability of CSHT in older transgender adults. Additionally, there are few randomized controlled trials with respect to CSHT, which limits knowledge of any associations between CSHT and CVD.

Click to read the study, published in the Annals of Internal Medicine

Relevant Reading: Hormone therapy for transgender patients

In-Depth [narrative review]: The authors of this study systematically searched PubMed and EMBASE databases, of which 13 met inclusion and exclusion criteria. Of the articles, 3 reported findings only in transgender women, 3 only in transgender men, and 7 looked at both populations. Based on the articles reviewed, the authors observed that CSHT is associated with improved psychological functioning of transgender persons. While studies looking at CSHT and cardiovascular risk factors in transgender men are limited, several studies, including an early prospective study and an observational longitudinal study, suggest that CSHT increases CVD risk factors. These risk factors include blood pressure values, lipid levels, and insulin resistance. Furthermore, in transgender women, CSHT may increase thromboembolic risk. However, due to the lack of randomized controlled trials and prospective cohort studies found in this review, it is clear that further research is required to determine the cardiovascular effects of long-term CSHT for transgender adults.

Image: PD

20172 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.

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More research needed to determine cardiovascular effects of long-term hormone therapy for transgender adults - 2 Minute Medicine

There's no shortage of weird tips and tricks on the Internet for everything from making your kids eat vegetables to getting your newborn to stop crying,but the latest trend in DIY is truly something else. Apparently, women are putting urine in toothpaste based on claims in a viral YouTube video that you can use this "natural" method as an alternative way to tell if you're pregnant.

The toothpaste pregnancy test works like this: First, you squeeze a small amount of plain white toothpaste into a cup, then you add a few drops of urine, mix it up, and wait three minutes. If the toothpaste starts to froth or turn a bluish color, that means the "test" is positive.

More from CafeMom: If You're Pregnant & Your Toilet Seat Turned Blue, You Are Not Alone

The video has more than 2 million views and Us Weekly notes it's a mainstay on Google Trends. As the websitebabyprepping.com explains, the toothpaste pregnancy test came about "decades ago when pregnancy tests weren't easily found in stores, especially for women living in rural settings." Now, they're mostly used as a cheap alternative to store-bought pregnancy tests.

Of course, not everyone is sold. The comments on YouTube range from grateful and enthusiastic to people saying this is total BS to men claiming they tried it and got a positive result (LOL).

So, is the toothpaste pregnancy test legit?

Sort of, says Dr. Napoleon Maminta, a primary care physician at Naptown Priority Healthin Indiana. If done correctly, this test may give a woman "reason to believe that she is pregnant," he says. But that positive result is not a guarantee.

More from CafeMom:How This Weird Purple Line on Your Booty Might Reveal When Your Baby's Coming

"While over-the-counter tests are difficult to compromise and taint, this homemade option can be easier to compromise if a person fails to take certain precautions," Dr. Maminta tells CafeMom.

If you're just seeing this for the first time and simplyhave to try it, Dr. Maminta offered some tips to get the best results.First, he says, it's vital to use clean cups so that no contaminants come into contact with the toothpaste. Second, women should use their first morning urine rather than using urine later in the day.

"First morning urine in pregnant women has higher concentrations of the pregnancy hormone hCG, which reacts with the chemicals in the paste to cause a color change," he explains.

Lastly, the toothpaste used must be naturally white and not one that is striped or artificially colored. "The natural white paste has the correct chemical composition to react with the hCG in the urine to cause a change in the paste's color and structure," Dr. Maminta explains. "Paste that is dyed will not react in the right way to give enough of a reading for a woman to know if she could be pregnant."

Even with all the precautions in place, Dr. Maminta still cautions that this should be viewed as an "initial test" and pregnancy should be confirmed via traditional, well-tested methods.

Long story short? Toothpaste pregnancy tests are not the most reliable method of finding out if you're pregnant. But, if you're trying to conceive and looking for some preliminary results before you shell out for another test? It might be worth a shot. As with all things related to pregnancy and our bodies, take the "alternative method" with a grain of salt -- and make sure you see a doctor!

Originally posted here:
Toothpaste Pregnancy Tests Are a Thing -- but Do They Work? - The Stir

iPSCells Represent a Superior Approach

iPS cell-derived cardiomyocyte patch demonstrates spontaneous and synchronized contractions after 4 days in culture.

One of the greatest promises of human stem cells is to transform these early-stage cells into treatments for devastating diseases. Stem cells can potentially be used to repair damaged human tissues and to bioengineer transplantable human organs using various technologies, such as 3D printing. Using stem cells derived from another person (allogeneic transplantation) or from the patient (autologous transplantation), research efforts are underway to develop new therapies for historically difficult to treat conditions. In the past, adult stem and progenitor cells were used, but the differentiation of these cell types has proven to be difficult to control. Initial clinical trials using induced pluripotent stem (iPS) cells indicate that they are far superior for cellular therapy applications because they are better suited to scientific manipulation.

CDIs iPS cell-derived iCell and MyCell products are integral to the development of a range ofcell therapyapplications. A study using iCell Cardiomyocytesas part of a cardiac patch designed to treat heart failure is now underway. This tissue-engineered implantable patch mayemerge as apotential myocardial regeneration treatment.

Another study done with iPS cell-derived cells and kidney structures has marked an important first step towards regenerating, and eventually transplanting, a functioning human organ. In this work, iCell Endothelial Cellswere used to help to recapitulatethe blood supply of a laboratory-generated kidney scaffold. This type of outcome will be crucial for circulation and nutrient distribution in any rebuilt organ.

iCell Endothelial Cells revascularize kidney tissue. (Data courtesy of Dr. Jason Wertheim, Northwestern University)

CDI and its partners are leveraging iPS cell-derived human retinal pigment epithelial (RPE) cells to develop and manufacture autologous treatments for dry age-related macular degeneration (AMD). The mature RPE cells will be derivedfrom the patients own blood cells using CDIs MyCell process. Ifapproved by the FDA, this autologous cellular therapy wouldbe one of the first of its kind in the U.S.

Learn more about the technologybehind the development of these iPScell-derived cellular therapies.

Excerpt from:
Cellular Therapy - The World Leader in Stem Cell Technology

By TIM TROGLEN Staff Writer Published: July 31, 2017 12:57 PM

AKRON The Akron Zoo announced today that Roscoe, a 14-year old male snow leopard, was euthanized on July 26 after being diagnosed with a fast-growing cancer that severely affected the bone in his lower jaw.

According to Doug Piekarz, Akron Zoo president CEO, Roscoe has been a beloved member of our family since he arrived from San Antonio. He will be missed by all of us. I want to thank our professional animal care staff who cared for him attentively every day, and our veterinary care staff who so diligently treated him during his illness to ensure his welfare,

According to the Snow Leopard Trust, at least one snow leopard is killed each day in the wild. With only a few thousand left on earth, we recognize the importance of the work we are doing to ensure the Snow Leopards survival. Roscoe will continue to play a critical role with his genetics preserved to help create a more genetically diverse future generation of snow leopards.

According to zoo officials, the median life expectancy of a snow leopard is 14 years old.

Roscoe was a resident of the Akron Zoo since 2004 and sired seven cubs while in Akron, three of which are still at the zoo.

Roscoe will be missed deeply by the zoo staff, volunteers and community, said Dr. Kim Cook, Akron Zoo director of animal health and conservation. He was a laid-back cat who had a great bond with Shanti. In fact they were able to be together at all times, which is rare for snow leopards, which are typically solitary animals.

Officials noticed Roscoe was not feeling well a few weeks ago through a change in his appetite and behavior.

The vet staff at the zoo performed an exam, which led to the diagnosis of squamous cell carcinoma bone cancer in Roscoes jaw. His appetite and activity began to rapidly decrease and the decision was made to humanely euthanize Roscoe. His annual preventive medicine exam last summer showed no signs of the cancer.

Snow leopards are an endangered species, and the zoo participates in the Association of Zoos and Aquariums Snow Leopard Species Survival Plan, which includes a total of 167 snow leopards. Participation in the program has led to three successful litters with Roscoe and the zoos female snow leopard Shanti. Two cubs were born in 2012 and 2014, and three cubs were born in 2016 and are still at the Akron Zoo, according to officials. The other four cubs are at other AZA-accredited zoos in the U.S.

In 2010 and 2012, the Akron Zoo worked with researchers to freeze Roscoes sperm to potentially use in the future for artificial insemination. With the advances in veterinary medicine, Roscoes legacy could continue for many generations and help prevent the extinction of snow leopards.

Roscoe was born May 18, 2003, at the San Antonio Zoo and came to the Akron Zoo Dec. 15, 2004.

Read the original here:
Akron Zoo mourns death of male snow leopard - Ravenna Record Courier

A team of scientists in South Australia are providing a powerful new line of defence against one of horticulture's most damaging pests.

The National Sterile Insect Facility in Port Augusta, is set to produce 50 million sterile male Queensland fruit flies a week by 2019.

The ambitious three-year project is all in a bid to safeguard the multi-billion dollar horticulture industry across South Australia and Victoria.

The Sterile Insect Technology (SIT) is the first insect pest control method that uses genetics, or a widescale form of insect birth control.

The technique involves breeding fruit flies and sterilising the males with an x-ray before they are released into an area with a wild population.

These sterile male flies are then released over infested areas, where they mate with wild females who eventually become outnumbered and die out.

The $45 million SITPlus initiative, led by Horticulture Innovation Australia, complements the state-of-the-art facility in Port Augusta, approximately 350 kilometres from Adelaide.

Program director Dan Ryan took SA Country Hour for an exclusive tour through the $3.8 million factory.

He said the whole process began in the egg collection room where the team was breeding up to 50 million flies per week.

"We've got males and females in here; [they] are in there to have sex, so this is the exciting room for the flies," he said.

"What we've got is a surface that the females can overposit or lay eggs into and then we collect those eggs, we put them on a larval tray where the eggs can hatch into larvae.

"Then the larvae mature and they go to a pupal rearing room, [where] we raise the pupae. They mature inside the pupae which is just a little shell."

Flies in the SIT facility are marked with a bright pink dye so they can be identified in the wild.

(ABC Rural: Courtney Fowler)

Flies in the SIT facility are marked with a bright pink dye so they can be identified in the wild.

ABC Rural: Courtney Fowler

Mr Ryan said once the flies leave the pupal rearing room, they were covered with a distinctive dye so they could be easily identified in the wild.

"We mark the pupae because we need to know when we get out in the wild; is this a fly that's come from a factory and is sterile or is this a wild fly," he said.

"So what we do is cover them with a bright iridescent dye on the pupae, then the fly comes out of the pupae and gets covered in dye.

"We do have back-up processes to identify them; in the very rare occurrence that there is no dye on a fly we have two tests which will verify it."

Mr Ryan said from the dye-marking room, the flies are x-rayed and ready for release into the wild.

"We all think of x-rays with broken arms but these flies are getting x-rays to become sterile," he said.

"It's a very low dose of x-ray because as you can imagine, it doesn't take much to make a fly sterile but we have a 100 per cent success rate.

"[On release] these sterile flies go out and trick the wild females and the population crashes, it's a horrible trick on the wild female flies but is all part of protecting horticulture in Australia."

The larvae mature inside the pupae inside a little shell in the pupal rearing room.

(ABC Rural: Courtney Fowler)

The larvae mature inside the pupae inside a little shell in the pupal rearing room.

ABC Rural: Courtney Fowler

Queensland fruit fly destroys an estimated $300 million of fruit and vegetable crops every year.

Mr Ryan said the SIT program was a 'game-changer' for management of the Queensland fruit fly across south eastern Australia.

Queensland fruit flies destroys an estimated $300 million dollars of fruit and vegetable crops every year.

(ABC: Laurissa Smith)

Queensland fruit flies destroys an estimated $300 million dollars of fruit and vegetable crops every year.

He said the facility would not only ensure South Australia's remains fruit fly-free but would also help reduce populations across the country.

"South Australia markets horticulture overseas based on freedom from Queensland fruit fly, that's worth a lot of money to the industry," he said.

"If they lost that market access it would really impact the value of their businesses, so this is all about protecting those businesses.

"Another use is for areas where you have large isolated farms, so a good example of that might be Hillston NSW, where you've got a collection of large citrus farms and a cherry farms.

"It's a great place to put that pressure down and perhaps establish longer term another pest-free area.

"The third use is helping farmers in endemic areas manage the flies; one of the problems growers have is they can manage the fly on their farm but they're always getting reinvasion from off the farm."

SIT program director Dan Ryan says the facility aims to breed 50 million flies per week by 2019.

(ABC Rural: Courtney Fowler)

SIT program director Dan Ryan says the facility aims to breed 50 million flies per week by 2019.

ABC Rural: Courtney Fowler

Biosecurity SA executive director Will Zacharin said the facility in Port Augusta was putting South Australia on the world map in Sterile Insect Technology.

A close up picture of a Queensland fruit fly.

(Clint Jasper)

A close up picture of a Queensland fruit fly.

"It's the first purpose-built fruit-fly facility in Australia, to make sure that we could significantly ramp up the number of flies that we could get out of the factory," he said.

"This [will enable us] to provide flies right across south eastern Australia for those areas that need it.

"This is moving from a solution in a can and just trying to use chemicals to control a problem, to using new innovative technology in terms of sterile insects.

"It's going to be more long-term, it's going to be good for the producer, it's going to be good for the environment [and] its going to be good for consumers."

Mr Zacharin said he was confident the program could help other major horticultural regions across southern NSW and Victoria strive towards becoming fruit fly-free.

"We've looked at facilities overseas that produce up to a billion flies a week, so on world terms this is a small facility," he said.

"But it's about demonstrating to industries and communities that releasing sterile flies is a better way to go in the long-term.

"If we can prove this works very well in the Australian environment, there's no reason why we can't push fruit fly freedom from where we are in SA, right across the Murray corridor, into southern NSW and Victoria.

"That will increase productivity for growers and will also give them better access to international markets."

South Australia is the only mainland State which remains fruit fly free in the country.

(ABC: Damien Hooper)

South Australia is the only mainland State which remains fruit fly free in the country.

The SITPlus program is led by Horticulture Innovation Australia, in partnership with Primary Industries and Regions SA, South Australian Research and Development Institute, Victorian Department of Economic Development, Jobs, Transport and Resources, CSIRO, Plant and Food Research Australia, NSW Department of Primary Industries and Macquarie University.

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How Sterile Insect Technology could combat one of horticulture's most damaging pests - ABC Online

Kristin Comella, Chief Science Officer

Chronic obstructive pulmonary disease (COPD) is the fourth leading cause of death in the United States, and is projected to be the third by 2020. COPD is associated with an exaggerated chronic inflammatory response causing airway abnormalities. Patients typically undergo a progression of declining lung function, characterized by an increase of cough, shortness of breath, and mucus production. Extra-pulmonary manifestations of COPD include osteoporosis, cardiovascular disease, skeletal muscle abnormalities, and depression. There is currently no cure and the manifestations can only be treated symptomatically. It afflicts more than 5% of the population in many countries and accounts for more than 600 billion in health care costs, morbidity, and mortality.

Adult stem cells are found in every part of the body and their primary role is to heal and maintain the tissue in which they reside. Stem cells are unspecialized cells capable of renewing themselves by cell division. In addition, they have the ability to differentiate into specialized cell types. Adult stem cells can be harvested from a patients own tissue, such as adipose (fat) tissue, muscle, teeth, skin or bone marrow. One of the most plentiful sources of stem cells in the body is the fat tissue. In fact, approximately 500 times more stem cells can be obtained from fat than bone marrow. Stem cells derived from a patients own fat are referred to as adipose-derived stem cells (ADSCs). Adipose derived stem cells have been explored with respect to their activity in diseases involving significant inflammatory or degenerative components. More recently, adult stem cells have been identified as having the potential to reverse the effects of diseases like COPD.

The mixed population of cells that can be obtained from fat is called a stromal vascular fraction (SVF). The SVF can easily be isolated from fat tissue in approximately 30-90 minutes in a clinic setting (under local anesthesia) using a mini-lipoaspirate technique. The SVF contains all cellular elements of fat, excluding adipocytes. Tens to hundreds of millions of ADSCs can be obtained in the context of the SVF acquired from 20-200 ml of adipose tissue during this out-patient procedure. This sets the stage for their practical use at the point-of-care, in which a preparation of ASC can be provided for infusion or injection after the mini-liposuction. COPD patients who have undergone stem cell therapies often express the willingness to receive additional cell infusions if possible, due to a feeling of well-being associated with the injection. There is early evidence of feasibility and safety of infusions into the patients with COPD. In relevant studies, intravenous infusion of cultured adipose stem cells has been demonstrated to remarkably improve the onset and progression of smoke exposure-induced emphysema in rodents.

Stem cells possess enormous regenerative potential. The potential applications are virtually limitless. Patients can receive cutting edge treatments that are safe, compliant, and effective. Our team has successfully treated over 7000 patients with very few safety concerns reported. One day, stem cell treatments will be the gold standard of care for the treatment of most degenerative diseases. We are extremely encouraged by the positive patient results we are seeing from our physician-based treatments. Our hope is that stem cell therapy will provide relief and an improved quality of life for many patients. The future of medicine is here!

For additional information on Stem Cell Centers of Excellences South Miami clinic, visit http://www.stemcellcoe.com.

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Stem Cells Offer New Solutions for Lung Disease - Miami's Community Newspapers

Scientists in Australia have used a 3D printer to create nerve cells found in the brain using a special bio-ink made from stem cells.

The research takes us a step closer to making replacement brain tissue derived from a patients own skin or blood cells to help treat conditions such as brain injury, Parkinsons disease, epilepsy and schizophrenia.

The bio-ink is made of human induced pluripotent stem cells (iPSC), which have the same power as embryonic stem cells to turn into any cell in the body, and possibly form replacement body tissues and even whole organs.

3D printing with bio-ink (ABC News)

[Jeremy Crookfrom the University of Wollongong stated]many neuropsychiatric disorders result from an imbalance of key chemicals called neurotransmittersFor example, he said, defective serotonin and GABA-producing nerve cells are implicated in schizophrenia and epilepsy[Thus] the team used 3D printing to make neurones involved in producing GABA and serotonin.

Apart from providing customized transplants, 3D printed tissue could be useful for medical research.

For example, tissue from a patient with epilepsy or schizophrenia could be created, specifically to study their particular version of the condition.

You can compare how neuronal networks form differently compared to healthy patient, said Dr Crook.

[Read the full study here]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Scientists create 3D-printed brain-like tissue from stem cells

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3D printed brain-like tissue made from stem cells offers hope to address neurological disorders - Genetic Literacy Project

By Kevin Orland on 7/31/2017

CALGARY (Bloomberg) -- TransCanada Corp. said it still expects commercial support for its controversial Keystone XL oil pipeline, tamping down speculation that it was having trouble finding customers for thelong-delayed line.

Keystone XL, which was rejected by the Obama administration before being revived by President Donald Trump this year,would boost TransCanadas dividend growth, the company said in a statement Friday. Media reports in recent weeks said that the company was having trouble signing up customers for the pipeline, conceived to help move crude from Albertas oil sands to refineries on the U.S. Gulf Coast.

TransCanada said earlier this year that it was working to sign new shippers following years of delays. Given the time it took to gain federal approval, TransCanada said it expected some shippers to reduce their volume commitments and that other new customers would be introduced. The company said on Thursday that its soliciting additional commitments to ship oil on Keystone XL.

Weve had good support from our legacy shippers, which gives us a good base to launch this open season, Paul Miller, TransCanadas president of liquids pipelines, said on a conference call.

The open season closes on Sept. 28, with the results of the process expected to be finalized in late November, Miller said. The company should also receive its regulatory decisions from Nebraska around that time and will weigh both of those factors in determining whether to proceed with the line, he said. If TransCanada decides to move ahead on Keystone XL, it would need six to nine months to prepare for construction and about two years to build it, he said.

The shares were up 0.2% at C$63.66 as of 1:44 p.m. in Toronto. Calgary-based TransCanada gained 5% this year through Thursday.

Dividend growth

Success in advancing Keystone XL or other growth initiatives such as the Bruce Power life extension may augment or extend the companys dividend growth outlook, CEO Russ Girling said in the statement. The company plans to increase its annual dividend at the upper end of an 8% to 10% range through 2020.

Keystone won votes of confidence from the chief executive officers of Canadian oil producers Cenovus Energy Inc. and Suncor Energy Inc. this week. The CEOs both said they support Keystone and that the Canadian energy industry needs more pipeline capacity. Suncor confirmed that it plans to ship its products on Keystone.

Albertas oil producers have long warned that a lack of pipeline space was hurting their prospects. That pipeline pinch may start to hit the industry later this year as Suncors massive Fort Hills oil-sands project starts to produce oil and Canadian Natural Resources Ltd. completes another phase of expansion at its Horizon mine.

Beyond Keystone

Looking beyond Keystone, TransCanada is spending C$2 billion ($1.6 billion) to expand its natural gas pipeline network in Western Canada. The upgrades to the Nova Gas system will include 171 mi (275 km) of new pipeline, additional compression and new metering stations.

The company said on Friday that it was applying to the National Energy Board to expand capacity on its Canadian Mainline, which carries natural gas from producers in Alberta to markets in the nations east. The company would spend about C$160 million on the project, which is underpinned by 15-year contracts.

TransCanadas second-quarter profit was 76 Canadian cents a share, excluding some items. Theaverage estimate of analysts surveyed by Bloomberg was 68 cents.

Continued here:
TransCanada still sees producer support for Keystone XL - WorldOil (subscription)

Global Hormone Replacement Therapy Market: Overview

The medical treatment where the patients receive hormones to substitute the naturally occurring hormones with the other hormones or to add naturally occurring hormones that are absent is known as hormone replacement therapy. In the females that are at the stage of menopause, hormone replacement therapy is used to restore female hormone levels, so that the body functions normally.

Request Free Sample Report @ https://www.zionmarketresearch.com/sample/hormone-replacement-therapy-market

Global Hormone Replacement Therapy Market: Segmentation

The global hormone replacement therapy is fragmented into therapy type, distribution channel, and application. On the basis of a therapy type, the global market is segregated into estrogen replacement therapy, thyroid hormone replacement, and growth hormone replacement. The thyroid hormone replacement segment is further sub-segmented into tablets, injections, and capsules. The growth hormone replacement segment is sub-categorized into somatostatin analogs and dopamine agonist. On the basis of the distribution channel, the market is categorized into e-commerce, retail pharmacies and drugstores, hospital pharmacies, compounding pharmacies, and others. On the basis of application, the market is divided into hypothyroidism, menopause, cancer, hypopituitarism, and others.

Global Hormone Replacement Therapy Market: Growth Factors

The key factors that are driving the hormone replacement therapy market are enlarged demand for the regenerative medicines which include reproductive-cycle boosting and anti-aging. The market is being positively impacted due to the increasing demand from other therapeutic areas which include thyroid hormone therapy and growth hormone therapy as the hormone replacement therapy is comparatively safe and efficient method and is cost effective. The other benefits that are associated with the hormone replacement therapy include minimum risk incidence of cardiovascular disease, osteoporosis, and vasomotor symptoms are also reduced thus expecting to fuel the growth of hormone replacement therapy market. The limitations of the hormone replacement therapy market include the side effects that are involved in this therapy such as fluid retention, indigestion, headache, and depression thus hindering the popularity of the therapeutic area.

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Global Hormone Replacement Therapy Market: Regional Analysis

Regional diversification of the hormone replacement therapy market is given as follows Asia Pacific, Latin America, the Middle East & Africa, Western Europe, Eastern Europe, and North America. The region that is dominating the hormone replacement therapy market is North America, which is due to the fact that the U.S has the largest market owing to the popularity of the therapy among the patients that are aged 35 years and above. The factors that are contributing to the market growth in this region are increasing disposable income, early aging, and the availability of compounded drugs. In the coming years, the hormone replacement therapy market will grow speedily in Asia Pacific region owing to the increasing awareness among the people. The emerging nations such as India, Japan, and China will contribute largely to the market growth.

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Global Hormone Replacement Therapy Market: Competitive Players

The key market players that are involved in the hormone replacement therapy market include Pfizer, BioSante Pharmaceuticals and Amgen, Noven Pharmaceuticals, Bayer AG, Merck & co., and QuatRx Pharmaceuticals.

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Hormone Replacement Therapy Market: Industry Players to Show High Growth Rate by 2024 - Monotone Critic

Xconomy Boston

A leading genome-editing researcher is urging extra caution as drug companies race to turn the landmark technology he helped create into human medicine.

In a paper published today in Nature Medicine, Feng Zhang of the Broad Institute of MIT and Harvard and colleague David Scott argue that researchers should analyze the DNA of patients before giving them experimental medicines that alter their genes with the breakthrough technology CRISPR. The suggestion, among others in the paper, stems from a deeper look at the wide array of subtle differences in human DNA.

Zhang is a key inventor of CRISPR-Cas9, which describes a two-part biological system that slips into the nucleus of cells and irreversibly alters DNA. One part is an enzyme, natures molecular scissors, which cuts DNA. The second part is a string of ribonucleic acid (RNA) that guides the enzyme to the proper spot. In five years since its invention, CRISPR-Cas9 has become a mainstay of biological research, and researchers including Zhang (pictured above) have moved quickly to improve upon its components. His work is at the center of a long-running patent battle to determine ownership of the technology.

Zhang and Scotts recommendation taps into a long-running debate in the gene-editing field about off-target effectsthe fear of misplaced cuts causing unintended harm. Most recently, the FDA took up a similar issue at a meeting to assess a type of cell therapy, known as CAR-T, for kids with leukemia. The FDA highlighted the risk that the cells, which have certain genes edited to make them better cancer fighters, may cause secondary cancers long after a patients leukemia has been cured. (FDA advisors unanimously endorsed the therapys approval nonetheless.)

Some researchers say there should be near certainty that gene altering techniques wont go awry before testing in humans, caution that stems in part from gene therapy experiments in the U.S. and Europe nearly 20 years ago that killed an American teenager and triggered leukemia in several European boys.

While no medicine is risk-free, other researchers say the tools to gauge risk have improved.

Andy May, senior director of genome engineering at the Chan Zuckerberg Biohub in San Francisco, calls Zhang and Scotts recommendation for patient prescreening a good discussion point, but the danger is someone will pick up on this and say you cant push forward [with a CRISPR drug] until everyone is sequenced.

Its an extremely conservative path to take, says May, who until recently was the chief scientific officer at Caribou Biosciences, a Berkeley, CA-based firm in charge of turning the discoveries of UC Berkeleys Jennifer Doudna and her colleagues into commercial technology. (May was also a board member of Cambridge, MA-based Intellia Therapeutics (NASDAQ: NTLA), which has exclusive license to use Caribous technology in human therapeutics.)

Berkeley is leading the challenge to Zhangs CRISPR patents and last week filed the first details in its appeal of a recent court decision in favor of Zhang and the Broad Institute.

Zhang sees prescreening as a form of companion diagnostic, which drug companies frequently use to identify the right patients for a study. A whole genome sequencewhich costs about $1,000could filter out patients unlikely to benefit from a treatment or at higher risk of unintended consequences, such as cancer. In the long run, it could also encourage developers to create more variations of a treatment to make genome-editing based therapeutics as broadly available as possible, said Zhang.

Its well known that human genetic variation is a hurdle in the quest to treat genetic diseases either by knocking out disease-causing genes or replacing them with healthy versions. But Zhang and Scott use newly available genetic information to deepen that understanding. In one Broad Institute database with genetic information from more than 60,000 people, they find one genetic variation for every eight letters, or nucleotides, in the exomethat is, the sections of DNA that contain instructions to make proteins. (There are 6 billion nucleotides in each of our cells.) The wide menu of differences is, in effect, an open door to misplaced cuts that CRISPRs enzymes might be prone to.

Zhang and others are working on many kinds of enzymes, from variations on the workhorse Cas9, to new ones entirely. He and Scott found that the deep pool of genetic variation makes some forms of the Cas enzyme more likely than others to go awry, depending on the three-nucleotide sequence they lock onto in the targeted DNA.

Zhang and Scott write that CRISPR drug developers should avoid trying to edit DNA strings that are likely to have high variation. In their paper, they examine 12 disease-causing genes. While more common diseases, such as those related to high cholesterol, will contain higher genetic variation because of the broader affected population, every gene, common or not, contains regions of high and low variation. Zhang and Scott say developers can build strategies around the gene regions they are targeting.

For example, going after a more common disease might require a wider variety of product candidates, akin to a plumber bringing an extra-large set of wrenches, with finer gradations between each wrench, to a job site with an unpredictable range of pipe sizes.

CRISPR companies say they are doing just that. We have always made specificity a fundamental part of our program, says Editas Medicine CEO Katrine Bosley. Zhang is a founder of Editas (NASDAQ: EDIT), which has exclusive license to the Broads Next Page

Alex Lash is Xconomy's National Biotech Editor. He is based in San Francisco.

Continued here:
CRISPR Pioneer Zhang Preaches Extra Caution In Human Gene ... - Xconomy

Theres still a way to go from editing single-cell embryos to a full-term designer baby. Credit: ZEISS Microscopy, CC BY-SA

The announcement by researchers in Portland, Oregon that they've successfully modified the genetic material of a human embryo took some people by surprise.

With headlines referring to "groundbreaking" research and "designer babies," you might wonder what the scientists actually accomplished. This was a big step forward, but hardly unexpected. As this kind of work proceeds, it continues to raise questions about ethical issues and how we should we react.

What did researchers actually do?

For a number of years now we have had the ability to alter genetic material in a cell, using a technique called CRISPR.

The DNA that makes up our genome comprises long sequences of base pairs, each base indicated by one of four letters. These letters form a genetic alphabet, and the "words" or "sentences" created from a particular order of letters are the genes that determine our characteristics.

Sometimes words can be "misspelled" or sentences slightly garbled, resulting in a disease or disorder. Genetic engineering is designed to correct those mistakes. CRISPR is a tool that enables scientists to target a specific area of a gene, working like the search-and-replace function in Microsoft Word, to remove a section and insert the "correct" sequence.

In the last decade, CRISPR has been the primary tool for those seeking to modify genes human and otherwise. Among other things, it has been used in experiments to make mosquitoes resistant to malaria, genetically modify plants to be resistant to disease, explore the possibility of engineered pets and livestock, and potentially treat some human diseases (including HIV, hemophilia and leukemia).

Up until recently, the focus in humans has been on changing the cells of a single individual, and not changing eggs, sperm and early embryos what are called the "germline" cells that pass traits along to offspring. The theory is that focusing on non-germline cells would limit any unexpected long-term impact of genetic changes on descendants. At the same time, this limitation means that we would have to use the technique in every generation, which affects its potential therapeutic benefit.

Earlier this year, an international committee convened by the National Academy of Sciences issued a report that, while highlighting the concerns with human germline genetic engineering, laid out a series of safeguards and recommended oversight. The report was widely regarded as opening the door to embryo-editing research.

That is exactly what happened in Oregon. Although this is the first study reported in the United States, similar research has been conducted in China. This new study, however, apparently avoided previous errors we've seen with CRISPR such as changes in other, untargeted parts of the genome, or the desired change not occurring in all cells. Both of these problems had made scientists wary of using CRISPR to make changes in embryos that might eventually be used in a human pregnancy. Evidence of more successful (and thus safer) CRISPR use may lead to additional studies involving human embryos.

What didn't happen in Oregon?

First, this study did not entail the creation of "designer babies," despite some news headlines. The research involved only early stage embryos, outside the womb, none of which was allowed to develop beyond a few days.

In fact, there are a number of existing limits both policy-based and scientific that will create barriers to implanting an edited embryo to achieve the birth of a child. There is a federal ban on funding gene editing research in embryos; in some states, there are also total bans on embryo research, regardless of how funded. In addition, the implantation of an edited human embryos would be regulated under the federal human research regulations, the Food, Drug and Cosmetic Act and potentially the federal rules regarding clinical laboratory testing.

Beyond the regulatory barriers, we are a long way from having the scientific knowledge necessary to design our children. While the Oregon experiment focused on a single gene correction to inherited diseases, there are few human traits that are controlled by one gene. Anything that involves multiple genes or a gene/environment interaction will be less amenable to this type of engineering. Most characteristics we might be interested in designing such as intelligence, personality, athletic or artistic or musical ability are much more complex.

Second, while this is a significant step forward in the science regarding the use of the CRISPR technique, it is only one step. There is a long way to go between this and a cure for various disease and disorders. This is not to say that there aren't concerns. But we have some time to consider the issues before the use of the technique becomes a mainstream medical practice.

So what should we be concerned about?

Taking into account the cautions above, we do need to decide when and how we should use this technique.

Should there be limits on the types of things you can edit in an embryo? If so, what should they entail? These questions also involve deciding who gets to set the limits and control access to the technology.

We may also be concerned about who gets to control the subsequent research using this technology. Should there be state or federal oversight? Keep in mind that we cannot control what happens in other countries. Even in this country it can be difficult to craft guidelines that restrict only the research someone finds objectionable, while allowing other important research to continue. Additionally, the use of assisted reproductive technologies (IVF, for example) is largely unregulated in the U.S., and the decision to put in place restrictions will certainly raise objections from both potential parents and IVF providers.

Moreover, there are important questions about cost and access. Right now most assisted reproductive technologies are available only to higher-income individuals. A handful of states mandate infertility treatment coverage, but it is very limited. How should we regulate access to embryo editing for serious diseases? We are in the midst of a widespread debate about health care, access and cost. If it becomes established and safe, should this technique be part of a basic package of health care services when used to help create a child who does not suffer from a specific genetic problem? What about editing for nonhealth issues or less serious problems are there fairness concerns if only people with sufficient wealth can access?

So far the promise of genetic engineering for disease eradication has not lived up to its hype. Nor have many other milestones, like the 1996 cloning of Dolly the sheep, resulted in the feared apocalypse. The announcement of the Oregon study is only the next step in a long line of research. Nonetheless, it is sure to bring many of the issues about embryos, stem cell research, genetic engineering and reproductive technologies back into the spotlight. Now is the time to figure out how we want to see this gene-editing path unfold.

Explore further: In US first, scientists edit genes of human embryos (Update)

This article was originally published on The Conversation. Read the original article.

Read more:
Editing human embryos with CRISPR is moving ahead now's the ... - Phys.Org

JoAnne Viviano The Columbus Dispatch @JoAnneViviano

Dennis Matko was headed for a knee replacement when he discovered a new therapy that would instead inject his own stem cells and plasma into the joint to help prevent degradation.

The 69-year-old Clintonville resident said he had been pretty active in his 50s, leading to problems with the right knee. He eventually had his meniscus removed. He had been through physical therapy, cortisone shots and gel injections, but the pain persisted.

The therapy, he said, was a no-brainer. He was sold because the procedure involved putting his own fluids into his body with no foreign objects and no drugs.

Dr. Joe Ruane, the orthopedic doctor who treated Matko, introduced the therapy at OhioHealth, but there are a number of places using the therapy around the state and country.

It's used to treat people with osteoarthritis, the type of arthritis caused by wear and tear.

Ruane said that the need for total knee replacements in the U.S. is expected to climb by 600 percent in the next 20 years, and there is concern that there might not be enough surgeons to perform the procedures.

We need an alternative, and patients are looking for alternatives, and given the choice between a knee replacement and an injection, many patients would choose an injection, he said.

The treatment involved removing Matkos bone marrow from the back of his pelvic bone, a process done in the office under general anesthesia. The marrow was then processed to form a concentrate of stem cells and other growth factors.

Matko also had blood drawn to create platelet-rich plasma, which acts as a signaling system to get the stem cells to respond.

Ruane injected both components into the knee, delivering more than 100 stimulating and growth factors to the joint.

Ruane said the process inhibits irritating chemicals that contribute to inflammation, decreases the activity of enzymes that break down cartilage, and helps the knee to make some of its own joint fluid again.

And, to a small degree, it does help regrow some of the tissue in the knee that has been destroyed by the arthritis, Ruane said.

The procedures are most effective in young patients with early arthritis, said Dr. Adolph Lombardi of Joint Implant Surgeons in New Albany, where stem-cell and platelet-rich plasma injections are offered as separate therapies. It won't help with bone-on-bone disease, he said.

While other injections might offer short-term pain relief, platelet-rich plasma has been shown to offer a full year of relief, said Lombardi, who works with the Mount Carmel Health System. The idea is that bone-marrow stem cells, when injected into a hip or knee, can differentiate into cartilage cells and help with regeneration.

"All of this is very new but it seems to be extremely promising," Lombardi said. "This is using their own bodies' healing potential to maintain cartilage and relieve pain."

Dr. Michael Baria performs the procedure at Wexner Medical Center at Ohio State University, where the bone-marrow and platelet-rich plasma injections also are offered as separate treatments. He agreed that the hope with the bone-marrow injections is that the stem cells turn into cartilage cells, improving or halting the osteoarthritis disease.

But in his experience, the treatment is helpful for patients with advanced disease.

"The most common patient we see for this is going to be in late-stage arthritis, so kind of at the end of their rope," Baria said. "Platelet-rich plasma is usually not as good for bone-on-bone arthritis. Bone marrow doesnt seem to be limited by bone on bone."

The body has trouble healing arthritis because cartilage doesnt get enough blood supply, Ruane said. Injecting the stem cells boosts the bodys own process.

While platelet-rich plasma has been shown to decrease inflammation, stem-cell use is newer and has yet to be proven effective, Baria noted.

OhioHealth andJoint Implant Surgeons are currently in the midst of controlled randomized trials, hoping to prove the effectiveness of the procedures and obtain approval from the U.S. Food and Drug Administration.

Unless that happens, the procedure will be considered experimental, and insurance doesnt cover costs. Matko paid $2,800 for the injections at OhioHealth.

Before the treatment, Matko was having trouble with mundane things like going up and down stairs and with other activities, such as taking hikes or walks with his wife or working out. A retired police officer, he now works as a business consultant and spends a lot of time on his feet, so he was looking for better mobility there as well.

Matko said the injections have helped his knee, which is getting progressively better over time. He said hes been able to increase his activity, getting back to the gym and taking hikes and walks. He has minimal pain climbing stairs and hes more comfortable in his work.

Im not saying its all better but its much better, Matko said. Its headed in the right direction.

He realizes the treatment is not a cure.

Im not looking for a miracle, he said. I just want to forestall problems as long as possible.

.

.

jviviano@dispatch.com

@JoAnneViviano

Read more:
Patients' plasma, stem cells help knee problems - The Columbus Dispatch

BrainStorm Cell Therapeutics of Petah Tikva is recruiting American patients for a Phase 3 clinical study of its NurOwn stem-cell treatment intended to halt progression of amyotrophic lateral sclerosis (ALS).

The announcement was made in a patient webinar last week.

The NurOwn platform grew out of a technique developed at Tel Aviv University for growing and enhancing stem cells harvested from patients own bone marrow. The enhanced cells, injected via lumbar puncture, secrete elevated levels of nerve-growth factors believed to protect existing motor neurons, promote motor neuron growth and reestablish nerve-muscle interaction.

A 24-week Phase 2 safety study was concluded in 2016 on 48 participants (36 treated, 12 placebo) with possible, probable and definite ALS. This study was done at the University of Massachusetts Medical School, Massachusetts General Hospital and the Mayo Clinic.

The Phase 3 double-blind, placebo-controlled study, to begin enrollment in August, will look at efficacy and safety of repeated doses. The California Institute for Regenerative Medicine has awarded Brainstorm a $16 million grant to support the pivotal trial.

This study will accept 200 randomized study participants between the ages of 18 and 60 (half getting the treatment and half a placebo) at the three previous centers as well as California Pacific Medical Center in San Francisco, UC-Irvine near Los Angeles and another site not announced.

Potential participants must live within about 100 miles of one of the centers for ease of follow-up. They will receive three doses over a 16-week treatment phase and then undergo 28 weeks of follow-up.

BrainStorm President and CEO Chaim Lebovits said he hopes to get approval by the end of the year for a hospital exemption program in Israel an accelerated regulatory pathway that would clear the way for a first batch of 50 patients to receive NurOwn at Tel Aviv Sourasky Medical Center. However, there will be no compassionate treatment using NurOwn in Israel or elsewhere.

The NurOwn platform technology also has potential applications in any neurodegenerative disease, such as multiple sclerosis and Parkinsons.

For more information, click here.

Go here to read the rest:
ALS treatment to begin Phase 3 clinical trials in US - ISRAEL21c

A stem cell-based method created by University of California, Irvine scientists can selectively target and kill cancerous tissue while preventing some of the toxic side effects of chemotherapy by treating the disease in a more localized way.

Weian Zhao, associate professor of pharmaceutical sciences, and colleagues have programmed human bone marrow stem cells to identify the unique physical properties of cancerous tissue. They added a piece of code to their engineered cells so that they can detect distinctively stiff cancerous tissue, lock into it and activate therapeutics.

In a study appearing inScience Translational Medicine, the researchers report they have effectively and safely employed this stem cell-targeting system in mice to treat metastatic breast cancer that had spread to the lung. They first transplanted the engineered stem cells to let them find and settle into the tumor site where they secreted enzymes called cytosine deaminase. The mice were then administered an inactive chemotherapy called prodrug 5-flurocytosine, which was triggered into action by the tumor site enzymes.

Zhao said his team specifically focused on metastatic cancer, which comes when the disease spreads to other parts of the body. Metastatic tumors are particularly deadly and the cause of 90 percent of cancer deaths.

This is a new paradigm for cancer therapy, Zhao said. We are going in a direction that few have explored before, and we hope to offer an alternative and potentially more effective cancer treatment.

Zhao added that this stem cell-targeting approach can provide an alternative to many forms of chemotherapy, which has a number of bad side effects. While this widely used method is powerful enough to kill rapidly growing cancer cells, it also can harm healthy ones.

Our new type of treatment only targets metastatic tissue, which enables us to avoid some of conventional chemotherapys unwanted side effects, said Zhao, who is a member of the Chao Family Comprehensive Cancer Center and the Sue & Bill Gross Stem Cell Research Center at UCI.

This published work is focused on breast cancer metastases in the lungs, he added. However, the technology will be applicable to other metastases as well, because many solid tumors have the hallmark of being stiffer than normal tissue. This is why our system is innovative and powerful, as we dont have to spend the time to identify and develop a new genetic or protein marker for every kind of cancer.

So far, the Zhao team has done preclinical animal studies to demonstrate that the treatment works and is safe, and they hope to transition to human studies in the near future. They are currently expanding to include other type of cells, including cancer tissue-sensing, engineered immune-system T cells (called CAR-T) to treat metastasizing breast and colon cancers. They also plan to transform the technology for other diseases such as fibrosis and diabetes, which result in stiffening of otherwise healthy tissue.

Along with Zhao, UCI doctoral students Linan Liu and Shirley Zhang, are co-leading authors of the study. The National Institutes of Health, the Department of Defense, the American Cancer Society and the California Institute for Regenerative Medicine provided support.

Read more from the original source:
Stem Cell Therapy Attacks Cancer by Targeting Unique Tissue ... - R & D Magazine

BALTIMORE Although there are medications to treat depression, many scientists arent sure why theyre effective and why they dont work for everyone.

Researchers at the University of Maryland School of Medicine believe they may have found a key to the puzzle of major depression that could lead to therapies for those who dont respond to medications already on the market.

A study by the researchers has identified the central role a gene known as Slc6a15 plays in either protecting from stress or contributing to depression, depending on its level of activity in a part of the brain associated with motivation, pleasure and reward seeking.

Published in the Journal of Neuroscience in July, the study is the first to illuminate in detail how the gene works in a kind of neuron that plays a key role in depression, the according to the medical school.

Specifically, the researchers found that mice with depression had reduced levels of the genes activity, while those with high levels of the genes activity handled chronic stress better.

Though senior researcher Mary Kay Lobos primary studies were done with mice, she also examined the brains of people who had committed suicide and found reduced levels of the genes activity, confirming a likely link.

She hopes now that drugs could be developed that would encourage the genes activity.

I thought it was fascinating we had this system in place that allows us to go after things or be motivated or have pleasure and I was interested in how it becomes dysfunctional in certain diseases like depression, Lobo said. I hope that we can identify molecules that could potentially be therapeutically treated or targeted to treat depression.

Lobo and her colleagues have been examining the gene for years. In 2006, they discovered that it was more common among specific neurons in the brain that they later learned were related to depression. Five years later, other researchers learned that the gene played a role in depression and Lobo and her research colleagues decided to investigate what that role is in those specific neurons.

About 15 million adults, or 6.7 percent of all U.S. adults, experience major depression in a given year, according to the Anxiety and Depression Association of America. It is the leading cause of disability for Americans ages 15 to 44. It is more prevalent in women and can develop at any age, but the median age of onset is 32.5.

David Dietz, an associate professor in the Department of Pharmacology and Toxicology at the State University of New York at Buffalo, said little was known previously about the biological basis of depression in the brain. Many drugs used to treat depression were discovered serendipitously, he said, and it wasnt clear why they worked.

Were starting to really get an idea of what does the depressed brain look like, Dietz said. When you put the whole puzzle together, you see where the problem is. For too long weve been throwing things at individual pieces. Its so complex and we have so little information that it was almost bound to be that way. For the first time this is one of those bigger pieces you can slide into the jigsaw puzzle.

Lobo said its not clear yet how Slc6a15 works in the brain, but she believes it may be transporting three types of amino acids into a subset of neurons called D2 neurons in a part of the brain called the nucleus accumbens. The nucleus accumbens and D2 neurons are known to play a role in pleasure, activating when one eats a delicious meal, has sex or drinks alcohol.

The amino acids would then be synthesized into neurotransmitters. Depression previously has been linked to imbalances of the neurotransmitters serotonin, norepinephrine and dopamine.

So even though people may have proper levels of amino acids in their bodies, the neurons in their brains that need them may not be getting enough if the transporter is not working as it should.

This gene is critical for putting very specific amino acids in the right place so that neurotransmitters can be synthesized, said A.J. Robison, an assistant professor in the Department of Physiology at Michigan State University. Its the location, location, location idea. Its not the amino acids, its where theyre at and in which cells.

Robison said Lobos next step would be discovering more about how the transporter gene works.

The fact that this transporter seems to be important is what the paper shows and how it does it is not shown, and thats a challenge for her, he said. Figuring out the how of it is the next step and Dr. Lobo is particularly positioned to do it.

Lobos team was able to use gene therapy, a form of therapy in the early stages of being studied in humans, in the mice to boost the genes activity. The mice were exposed to larger, more aggressive mice, which usually causes depressive symptoms. But the gene therapy helped protect the mice against the stress, the team found. When the team reduced the genes activity in the mice, just one day of exposure to the aggressive mice was enough to cause symptoms of depression.

Gene therapy is starting to be used in the treatment of some types of cancers, but Lobo said science had not yet advanced to the point where it can be used for treating neurological issues in human patients. A more likely treatment would be a drug that targets the genes activity directly, she said.

I think this is a major step toward our understanding of the precise maladaptive changes that occur in response to stress, said Vanna Zachariou, an associate professor in the Department of Neuroscience at the Icahn School of Medicine at Mount Sinai. It can be a more efficient way to target depression because its not simply targeting monoamine receptors or dopamine but targeting molecular adaptations that occur. It doesnt act necessarily as the drugs we have available, so it might create an alternative avenue to treat depression.

Lobo said she wouldnt refer to Slc6a15 as a depression gene, saying the disease was complex and could have many factors.

I wouldnt say theres one depression gene she said. A number of things play a role, and also theres no depression neuron, theres multiple depression neurons.

There also may be different types of depression with different symptoms, she said. With the disease, some sufferers sleep a lot, while others sleep a lot less, for example.

With all these complex diseases, its hard to link it to something, she said. Like Huntingtons disease, we know theres a specific gene that causes Huntingtons disease. For depression we dont have that.

See more here:
Maryland scientists research gene linked to depression | The ... - The Spokesman-Review

The idea of treating Duchenne muscular dystrophy by replacing defective dystrophin genes with normal ones is not new, but previous approaches have been limited by the gene's size. A University of Missouri-led team has developed a new gene transfer method to solve this problem.

Duchenne is caused by mutations in the dystrophin gene, which codes for a protein of the same name. Without the stabilizing dystrophin protein, muscle fibers, including those in the heart, eventually weaken and die.

Gene therapy seeks to treat DMD by restoring dystrophin production. Adeno-associated viruses (AAV) are usedto deliver the gene, as they do not cause disease in humans. But because the dystrophin gene is too large for the virus to carry, researchers had to developmodified versions of the gene, dubbed mini- or microdystrophin, for gene therapy.

Problem is, editing the gene can leave out a binding site for the enzyme nNos, which is important for blood flow during muscle contraction, the researchers said. So the team, which also includes scientists from the University of Washington, developed a new AAV microdystrophin vector that has an nNos binding site and a component that promotes dystrophin expression in muscle cells.

RELATED: Shortened telomeres linked to heart damage in Duchenne muscular dystrophy

They injected the vector into mouse models that resemble DMD.Fifteen weeks later, they foundall 10 of the treated mice had high levels of the microdystrophin protein in all of their skeletal muscles. The treatment reduced inflammation, scarring and hardening in the mices muscles and restored their muscle strength.

The research, published in Molecular TherapyMethods & Clinical Development, is encouraging, but a new treatment is a few years off.

"There is still a lot to learn about the dystrophin gene, the dystrophin protein, Duchenne muscular dystrophy disease mechanisms, and gene transfer vectors," said senior author Dongsheng Duan, of the University of Missouri, in a press release. "Future studies will hopefully allow us to develop a more effective therapy to treat Duchenne muscular dystrophy in the coming years."

Most of the recent attention in DMD has gone to Sarepta, which gotits controversial Duchenne drug past the FDAafter much debate. Now the company is chasing other treatments for the disease. In January, Sarepta penned a microdystrophin research deal with Nationwide Childrens Hospital and in June, it inked a deal that gave it the option to co-develop France-based Genethons microdystrophin program.

Sareptas drug, Exondys 51, only works in patients with a particular mutation, about 13% of the total DMD patient population. Its new research partnerships could yield gene therapies that would treat many morepatients.

Continued here:
U. of Missouri-led scientists improve gene transfer in Duchenne therapy - FierceBiotech

Although medications exist to treat depression, many scientists arent sure why theyre effective and why they dont work for everyone.

Researchers at the University of Maryland School of Medicine believe they may have found a key to the puzzle of major depression that could lead to therapies for those who dont respond to medications already on the market.

A new study by the researchers has identified the central role a gene known as Slc6a15 plays in either protecting from stress or contributing to depression, depending on its level of activity in a part of the brain associated with motivation, pleasure and reward seeking.

Published in the Journal of Neuroscience in July, the study is the first to illuminate in detail how the gene works in a kind of neuron that plays a key role in depression, according to the University of Maryland School of Medicine.

Specifically, the researchers found that mice with depression had reduced levels of the genes activity, while those with high levels of the genes activity handled chronic stress better.

Though senior researcher Mary Kay Lobos primary studies were done with mice, she also examined the brains of people who had committed suicide and found reduced levels of the genes activity, confirming a likely link.

She hopes now that drugs could be developed that would encourage the genes activity.

I thought it was fascinating we had this system in place that allows us to go after things or be motivated or have pleasure and I was interested in how it becomes dysfunctional in certain diseases like depression, Lobo said. I hope that we can identify molecules that could potentially be therapeutically treated or targeted to treat depression.

Lobo and her colleagues have been examining the gene for years. In 2006, they discovered that it was more common among specific neurons in the brain that they later learned were related to depression. Five years later, other researchers learned the gene played a role in depression and Lobo and her research colleagues decided to investigate what that role is in those specific neurons.

About 15 million adults, or 6.7 percent of all U.S. adults, experience major depression in a given year, according to the Anxiety and Depression Association of America. It is the leading cause of disability for Americans aged 15 to 44. It is more prevalent in women and can develop at any age, but the median age of onset is 32.5.

David Dietz, an associate professor in the Department of Pharmacology and Toxicology at the State University of New York at Buffalo, said little was known previously about the biological basis of depression in the brain. Many drugs used to treat depression were discovered serendipitously, he said, and it wasnt clear why they worked.

Were starting to really get an idea of what does the depressed brain look like, Dietz said. When you put the whole puzzle together, you see where the problem is. For too long weve been throwing things at individual pieces. Its so complex and we have so little information that it was almost bound to be that way. For the first time this is one of those bigger pieces you can slide into the jigsaw puzzle.

Lobo said its not clear yet how Slc6a15 works in the brain, but she believes it may be transporting three types of amino acids into a subset of neurons called D2 neurons in a part of the brain called the nucleus accumbens. The nucleus accumbens and D2 neurons are known to play a role in pleasure, activating when one eats a delicious meal, has sex or drinks alcohol.

The amino acids would then be synthesized into neurotransmitters. Depression previously has been linked to imbalances of the neurotransmitters serotonin, norepinephrine and dopamine.

So even though people may have proper levels of amino acids in their bodies, the neurons in their brains that need them may not be getting enough if the transporter is not working as it should.

This gene is critical for putting very specific amino acids in the right place so that neurotransmitters can be synthesized, said A.J. Robison, an assistant professor in the Department of Physiology at Michigan State University. Its the location, location, location idea. Its not the amino acids, its where theyre at and in which cells.

Robison said Lobos next step would be discovering more about how the transporter gene works.

The fact that this transporter seems to be important is what the paper shows and how it does it is not shown, and thats a challenge for her, he said. Figuring out the how of it is the next step and Dr. Lobo is particularly positioned to do it.

Lobos team was able to use gene therapy, a form of therapy in the early stages of being studied in humans, in the mice to boost the genes activity. The mice were exposed to larger, more aggressive mice, which usually causes depressive symptoms. But the gene therapy helped protect the mice against the stress, the team found. When the team reduced the genes activity in the mice, just one day of exposure to the aggressive mice was enough to cause symptoms of depression.

Gene therapy is starting to be used in the treatment of some types of cancers, but Lobo said science had not yet advanced to the point where it can be used for treating neurological issues in human patients. A more likely treatment would be a drug that targets the genes activity directly, she said.

I think this is a major step toward our understanding of the precise maladaptive changes that occur in response to stress, said Vanna Zachariou, an associate professor in the Department of Neuroscience at the Icahn School of Medicine at Mount Sinai. It can be a more efficient way to target depression because its not simply targeting monoamine receptors or dopamine but targeting molecular adaptations that occur. It doesnt act necessarily as the drugs we have available, so it might create an alternative avenue to treat depression.

Lobo said she wouldnt refer to Slc6a15 as a depression gene, saying the disease was complex and could have many factors.

I wouldnt say theres one depression gene she said. A number of things play a role, and also theres no depression neuron, theres multiple depression neurons.

There also may be different types of depression with different symptoms, she said. With the disease, some sufferers sleep a lot, while others sleep a lot less, for example.

With all these complex diseases, its hard to link it to something, she said. Like Huntingtons disease, we know theres a specific gene that causes Huntingtons disease. For depression we dont have that.

cwells@baltsun.com

See the rest here:
University of Maryland scientists research gene linked to depression - Baltimore Sun

Global Cancer Gene Therapy Market Research Report 2017 to 2022presents an in-depth assessment of the Cancer Gene Therapy including enabling technologies, key trends, market drivers, challenges, standardization, regulatory landscape, deployment models, operator case studies, opportunities, future roadmap, value chain, ecosystem player profiles and strategies. The report also presents forecasts for Cancer Gene Therapy investments from 2017 till 2022.

This study answers several questions for stakeholders, primarily which market segments they should focus upon during the next five years to prioritize their efforts and investments. The Cancer Gene Therapy markets are highly fragmented due to the presence of numerous small and large vendors. Most of the international pharmaceutical companies are facing challenges such as price pressure, regulatory constraints, and competition from local and other international pharmaceutical companies in the Cancer Gene Therapy markets. The competitive environment in the market is expected to intensify with an increase in product extensions, technological innovations, and increase in mergers and acquisitions.

These stakeholders include Cancer Gene Therapy manufacturers such as : Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies, Introgen Therapeutics, ZIOPHARM Oncology, MultiVir, Shenzhen SiBiono GeneTech.

Inquire for sample of report @:

https://www.marketinsightsreports.com/reports/07318362/global-cancer-gene-therapy-market-professional-survey-report-2017/inquiry

Primary sources are mainly industry experts from core and related industries, and suppliers, manufacturers, distributors, service providers, and organizations related to all segments of the industrys supply chain. The bottom-up approach was used to estimate the global market size of Cancer Gene Therapy based on end-use industry and region, in terms of value. With the data triangulation procedure and validation of data through primary interviews, the exact values of the overall parent market, and individual market sizes were determined and confirmed in this study.

Secondly the study, besides estimating the Cancer Gene Therapy market potential till 2022, analyzes on who can be the market leaders and what partnerships would help them to capture the market share. The report gives an overview about the dynamics of the market, by discussing various aspects such as drivers, restraints, Porters 5 forces, value chain, customer acceptance and investment scenario.

TheGlobal Cancer Gene Therapy marketconsists of different international, regional, and local vendors. The market competition is foreseen to grow higher with the rise in technological innovation and M&A activities in the future. Moreover, many local and regional vendors are offering specific application products for varied end-users. The new vendor entrants in the market are finding it hard to compete with the international vendors based on quality, reliability, and innovations in technology.

The research report presents a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It also contains projections using a suitable set of assumptions and methodologies. The research report provides analysis and information according to categories such as market segments, geographies, types, technology and applications.

Global Cancer Gene Therapy Sales (K Units) and Revenue (Million USD) Market Split by Product Type:

Global Cancer Gene Therapy Sales (K Units) by Application (2016-2022)

by Application

(2016-2022)

The research provides answers to the following key questions:

This independent 109 page report guarantees you will remain better informed than your competition. With over 150 tables and figures examining the Cancer Gene Therapy market, the report gives you a visual, one-stop breakdown of the leading products, submarkets and market leaders market revenue forecasts as well as analysis to 2022.

Browse Full Report @:

https://www.marketinsightsreports.com/reports/07318362/global-cancer-gene-therapy-market-professional-survey-report-2017

Geographically, this report is segmented into several key Regions, with production, consumption, revenue (million USD), and market share and growth rate of Storage Area Network Switch in these regions, from 2012 to 2022 (forecast), covering

by Regions

The report provides a basic overview of the Cancer Gene Therapy industry including definitions, classifications, applications and industry chain structure. And development policies and plans are discussed as well as manufacturing processes and cost structures.

Then, the report focuses on global major leading industry players with information such as company profiles, product picture and specifications, sales, market share and contact information. Whats more, the Cancer Gene Therapy industry development trends and marketing channels are analyzed.

The report segments this market based on products, portability, methods, applications, and end users. Among various end users, the hospitals segment is expected to account for the largest share of the market and is expected to grow at the highest CAGR from 2014 to 2019. The high growth in this segment can be attributed to the rising rate of maternal mortality and fetal birth defects, and increasing number of initiatives taken by government organizations for improving prenatal care.

The research includes historic data from 2012 to 2016 and forecasts until 2022 which makes the reports an invaluable resource for industry executives, marketing, sales and product managers, consultants, analysts, and other people looking for key industry data in readily accessible documents with clearly presented tables and graphs. The report will make detailed analysis mainly on above questions and in-depth research on the development environment, market size, development trend, operation situation and future development trend of Cancer Gene Therapy on the basis of stating current situation of the industry in 2017 so as to make comprehensive organization and judgment on the competition situation and development trend of Cancer Gene Therapy Market and assist manufacturers and investment organization to better grasp the development course of Cancer Gene Therapy Market.

The study was conducted using an objective combination of primary and secondary information including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to a SWOT analysis of the key vendors.

The report is a compilation of first-hand information, qualitative and quantitative assessment by industry analysts, inputs from industry experts and industry participants across the value chain. The report provides in-depth analysis of parent market trends, macro-economic indicators and governing factors along with market attractiveness as per segments. The report also maps the qualitative impact of various market factors on market segments and geographies.

There are 15 Chapters to deeply display the global Cancer Gene Therapy market.

Chapter 1, to describe Cancer Gene Therapy Introduction, product scope, market overview, market opportunities, market risk, market driving force;

Chapter 2, to analyze the top manufacturers of Grain and Seed Cleaning Equipment, with sales, revenue, and price of Grain and Seed Cleaning Equipment, in 2016 and 2017;

Chapter 3, to display the competitive situation among the top manufacturers, with sales, revenue and market share in 2016 and 2017;

Chapter 4, to show the global market by regions, with sales, revenue and market share of Grain and Seed Cleaning Equipment, for each region, from 2012 to 2017;

Chapter 5, 6, 7,8and 9, to analyze the key regions, with sales, revenue and market share by key countries in these regions;

Chapter 10and 11, to show the market by type and application, with sales market share and growth rate by type, application, from 2012 to 2017;

Chapter 12, Cancer Gene Therapy market forecast, by regions, type and application, with sales and revenue, from 2017 to 2022;

Chapter 13, 14 and 15, to describe Cancer Gene Therapy sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

Link:
Cancer Gene Therapy Market Analysis And Review By Experts 2017 - Equity Insider (press release)

LONDON (Reuters) - GlaxoSmithKline is swimming against the tide by getting out of treatments for rare diseases at a time when rivals like Sanofi and Shire see the field as a rich seam for profits.

Successful medicines for rare conditions are potentially very lucrative, since prices frequently run into hundreds of thousands of dollars, but patient numbers can be extremely low.

New GSK Chief Executive Emma Walmsley announced the strategic review and potential divestment of rare diseases on Wednesday as part of a wide-ranging drive to streamline pharmaceutical operations.

It follows a less than impressive experience for GSK in the field, including the fact that its pioneering gene therapy Strimvelis only secured its first commercial patient in March, 10 months after it was approved for sale in Europe in May 2016.

Since then a second patient has also been treated and two more are lined up to receive the therapy commercially, a spokesman said.

Strimvelis, which GSK developed with Italian scientists, is designed for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID). SCID is sometimes known as "bubble baby" disease, since those born with it have immune systems so weak they must live in germ-free environments.

The new treatment became the first life-saving gene therapy for children when it was approved last year, marking a step forward for the emerging technology to fix faulty genes.

Walmsley said GSK was not giving up on gene and cell therapy entirely. Research will be focused in future in areas with larger potential patient numbers, including oncology.

Reporting by Ben Hirschler; Editing by Adrian Croft

Original post:
GSK gives up on rare diseases as gene therapy gets two customers - Reuters