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Archive for the ‘Gene Therapy Research’ Category

Can Genetic Engineering Mitigate California's Drought?

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STANFORD, CA Water is in increasingly short supply in many parts of the United States. Here in California, where most of the state is experiencing extreme drought, 2013 was the driest year on record, and we have had no relief during what should be the height of the rainy season. Moreover, theres no end in sight: The Climate Prediction Center of the National Weather Serviceforecaststhat the drought will persist or intensify at least through April.

Reservoir levels are dropping, the snow pack is almost nonexistent, and many communities have already imposed restrictions on water usage. In the city of Santa Cruz, for example, restaurants can no longer serve drinking water unless diners specifically request it; Marin County residents have been asked not to clean their cars or to do so only at eco-friendly car washes; and there are limitations on watering lawns in towns in Mendocino County.

But it is the states premier industry farming that will feel the pinch most. In an average year, farmers use 80 percent of the water used by people and businesses, according to the Department of Water Resources.

During a January 19 press conference at which he declared a water emergency, Governor Jerry Brown said of the drought, This is not a partisan adversary. This is Mother Nature. We have to get on natures side and not abuse the resources that we have.

Drought may not be partisan, but it does raise critical issues of governance, public policyand how best to use the states natural resources. It also offers an example of the Law of Unintended Consequences: Ironically, Santa Cruz, Mendocino and Marin counties all of which boast politically correct, far-left politics are among the local jurisdictions that have banned a key technology that could conserve huge amounts of water.

The technology is genetic engineering performed with modern molecular techniques, sometimes referred to as genetic modification (GM) or gene-splicing, which enables plant breeders to make old crop plants do spectacular new things, including conserve water. In the United States and about 30 other countries, farmers are using genetically engineered crop varieties to produce higher yields, with lower inputs and reduced impact on the environment.

Even with R&D being hampered by resistance from activists and discouraged by governmental over-regulation, genetically engineered crop varieties are slowly but surely trickling out of the development pipeline in many parts of the world. Cumulatively, over 3.7 billion acres of them have been cultivated by more than 17 million farmers in 30 countries during the past 15 years without disrupting a single ecosystem or causing so much as a tummy ache in a consumer.

Most of these new varieties are designed to be resistant to herbicides, so that farmers can adopt more environment-friendly no-till farming practices and more benign herbicides; or to be resistant to pests and diseases that ravage crops. Others possess improved nutritional quality. But the greatest boon of all both to food security and to the environment in the long term will likely be the ability of new crop varieties to tolerate periods of drought and other water-related stresses. Where water is unavailable for irrigation, the development of crop varieties able to grow under conditions of low moisture or temporary drought could both boost yields and lengthen the time that farmland is productive.

Even where irrigation is feasible, plants that use water more efficiently are needed. Because irrigation for agriculture accounts for roughly 70 percent of the worlds fresh water consumption, the introduction of plants that grow with less water would allow much of it to be freed up for other uses. Especially during drought conditions, even a small percentage reduction in the use of water for irrigation could result in huge benefits.

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Can Genetic Engineering Mitigate California's Drought?

Articles from groundbreaking new Violence and Gender journal published

PUBLIC RELEASE DATE:

5-Feb-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, February 5, 2014Mary Ann Liebert, Inc., publishers has announced the publication of six articles from Violence and Gender, a new peer-reviewed journal dedicated to the understanding, prediction, and prevention of violence and spearheaded by Mary Ellen O'Toole, PhD, Forensic Behavioral Consultant and Senior FBI Profiler/Criminal Investigative Analyst (ret.). The Journal is the international forum for the critical examination of biological, genetic, behavioral, psychological, racial, ethnic, and cultural factors as they relate to the gender of perpetrators of violence, and explores the difficult issues that are vital to threat assessment and violence prevention. The articles are available free on the Violence and Gender website at http://www.liebertpub.com/vio.

Among the published articles is "Why Do Young Males Attack Schools? Seven Discipline Leaders Share Their Perspectives," which offers an unprecedented look into the reasons for the high incidence of school shootings in the U.S., and addresses the reasons we see mostly young males (15-29) committing these types of crimes. The article delves into what motivates these perpetrators, including the potential role of the copycat phenomenon in behavior. Sharing their perspectives are world-renowned experts Jorge Folino, MD, PhD, National University of La Plata (Buenos Aires Province, Argentina); James Garbarino, PhD, Loyola University Chicago (IL); Steven Gorelick, PhD, Hunter College, City University of New York (New York, NY); Helin Hkknen-Nyholm, PhD, PsyJuridica Ltd. (Espoo, Finland); J. Reid Meloy, PhD, University of California, San Diego (La Jolla, CA); Stanton Samenow, PhD, Alexandria, VA; and Yuki S. Nishimura, MD, PhD, Keio University (Yokohama City, Japan).

"Violence is complicated, and too often misunderstood, myth-based, and stereotyped; we are shocked when we see the 'nice guy' next door arrested for serial murder, or the quiet loner go on a shooting rampage," says Dr. O'Toole. "Many of us even default to using terms like 'monster' and 'evil' to explain such behavior and the people responsible. These archaic terms don't educate us or explain the violence but rather catapult us back in time. It's time for change in how we view violence."

Other published articles include an insightful roundtable discussion with Christopher Kilmartin, PhD, University of Mary Washington (Fredericksburg, VA), and Col. Jeffery M. Peterson, USMC (ret.) and Center for Naval Analyses (Alexandria, VA), entitled "Sexual Assault in the Military: A Discussion of the Current Status and Future Prevention;" the Review article "The Mission-Oriented Shooter: A New Type of Mass Killer," by Editor-in-Chief Mary Ellen O'Toole, PhD; and a Perspective entitled "Understanding Brain Health Can Prevent Another Sandy Hook Shooting," by Jeremy Richman, PhD, Founder and CEO of The Avielle Foundation (Sandy Hook, CT).

"The imperative for this journal is urgent," said publisher Mary Ann Liebert, "we must stem the tide, and the papers in Violence and Gender have a most important mandate."

###

The full inaugural issue of Violence and Gender will be published in Spring 2014.

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Articles from groundbreaking new Violence and Gender journal published

Is the male or female brain more vulnerable to triggers of violent behavior?

PUBLIC RELEASE DATE:

5-Feb-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, February 5, 2014Human behaviors such as violence depend on interactions in the brain between genetic and environmental factors. An individual may be more vulnerable to developing violent behaviors if they have predisposing factors and are then exposed to stress, abuse, or other triggers, especially early in life. The latest research on how differences between the male and female brain contribute to sex differences in violence is explored in Violence and Gender, a new peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Violence and Gender website at http://www.liebertpub.com/vio.

The article "Not Hardwired: The Complex Neurobiology of Sex Differences in Violence" describes the complex and flexible biological mechanisms in the brain that lead to the development of behaviors. These include interconnected neural networks, multiple genes, and chemical signals such as hormones and neurotransmitters, which can be modified by environmental factors. Brain structure, function, and connectivity can all differ between men and women, affecting how they may change on exposure to stressful or abusive triggers.

"Neurobiologist Dr. Debra Niehoff explains the amazing interaction of how our brains, genetics, and environmental influences can interact and serve as the genesis for violent behavior," says Editor-in-Chief of Violence and Gender Mary Ellen O'Toole, PhD, Forensic Behavioral Consultant, and Senior FBI Profiler/Criminal Investigator Analyst (ret.). "This holistic view of the origin of violence means that reducing violence will not be a simple fix because it does not have a single origin or cause. The temptation to delineate a male and female brain must be resisted because there is overlap between the two. With more research will come greater insight and knowledge about the biological and environmental causes of violence. With more knowledge will come answers; answers will lead to solutions, and with solutions will come prevention."

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About the Journal

Violence and Gender is the only peer-reviewed journal focusing on the understanding, prediction, and prevention of acts of violence. Through research papers, roundtable discussions, case studies, and other original content, the Journal critically examines biological, genetic, behavioral, psychological, racial, ethnic, and cultural factors as they relate to the gender of perpetrators of violence. Led by Editor-in-Chief Mary Ellen O'Toole, PhD, Forensic Behavioral Consultant and Senior FBI Profiler/Criminal Investigative Analyst (ret.), Violence and Gender explores the difficult issues that are vital to threat assessment and prevention of the epidemic of violence. Violence and Gender is published quarterly online with Open Access options and in print, and is the official journal of The Avielle Foundation.

About the Publisher

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Is the male or female brain more vulnerable to triggers of violent behavior?

JANUS Varispan – Video


JANUS Varispan

By: Center for Genetic Medicine

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JANUS Varispan - Video

Researchers discover genetic mutations that cause rare and …

A team of researchers, led by physicians and scientists at Intermountain Healthcare's Intermountain Medical Center and ARUP Laboratories, has made a medical breakthrough by discovering genetic mutations that cause a rare and deadly lung disease.

The disease, pulmonary capillary hemangiomatosis or PCH, is a rare cause of pulmonary hypertension, which occurs predominantly in young adults. PCH affects less than one in a million people, and has been extremely difficult and expensive to diagnose, as well as challenging to treat.

This genetic discovery offers new hope.

"This is a significant finding. This discovery should advance our understanding of this rare pulmonary vascular disorder and other related disorders," said Greg Elliott, MD, MACP, senior investigator of the study and medical director of the Pulmonary Hypertension Center at Intermountain Medical Center in Murray, Utah, and professor of medicine at the University of Utah School of Medicine.

Results of the study will be published in the February issue of the journal Chest, the official publication of the American College of Chest Physicians.

Dr. Elliott and his team at Intermountain Medical Center and the University of Utah School of Medicine collaborated with researchers from Columbia University, Vanderbilt University and Mayo Clinic-Scottsdale.

To find the genetic mutation, the research team used a relatively new technology whole exome sequencing performed at ARUP Laboratories in Salt Lake City to test DNA samples. They discovered the genetic mutations in Eukaryotic Translation Initiation Factor 2 Alpha Kinase 4. EIF2AK4 is a protein responsible for down-regulating protein synthesis when cells are exposed to stress.

Researchers found that in patients with the genetic mutations, their bodies don't properly regulate blood vessels in the lung. As a result, the capillaries in the lungs proliferate and the patient develops pulmonary hypertension.

D. Hunter Best, PhD, and Kelli L. Sumner, BS, two scientists on the research team, conducted the exome sequencing and analyzed the data in collaboration with colleagues at Columbia University and Vanderbilt University.

"Whole exome sequencing is breakthrough technology that allows us to test accurately and cost effectively for rare genetic disorders," said Dr. Elliott. "Without Dr. Best's expertise and the work done by Kelly Sumner in the laboratory, this discovery would not have occurred."

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Researchers discover genetic mutations that cause rare and ...

Regents push for change in genetic medicine

Regents push for change in genetic medicine

BY KEVIN SVEC | FEBRUARY 06, 2014 5:00 AM

What was once confined only to science-fiction movies is now the subject of boardroom meetings. The University of Iowa Carver College of Medicine, the home to the Iowa Institute of Human Genetics, plans to start a revolution in modern medicine. Today, most of the medication prescribed is based on the weight and body surface area of the patient. The institutes goal is to promote an alternative, which will be known as personal genomic medicine.

Such medicine would cater to each patients specific needs. The medication prescribed would be based on the genetic makeup of a patient rather than her or his body index.

Using a genetic test, scientists would be able to evaluate each patients needs, allowing health-care providers to personalize each drug treatment.

The medicine will work with each individual patient based on her or his personal health risks. By personalizing the medicine, the doctors could increase the likelihood that the drug would have the best possible effect on each patient.

Richard Smith, the director of the Institute of Human Genetics, noted several advantages of genomic medicine.

Newborns would be able to have screenings done to determine what medication would work best from the beginning, said Smith.

According to the Jackson Laboratory website, any prescription drug now on the market only works for half of the people who take it. Antidepressants are effective for only 63 percent of those who take it. The percentage rate of effectiveness jumps to 75 percent among cancer patients. Genetic testing can change that.

Part of the process has started already. For those willing to pay, they can have their genes tested. The cost for the test is $296 through the UI Hospitals and Clinics, $256 through the institute. However, Medicare is willing to pay for $295 of the costs.

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Penn study reveals genetics impact risk of early menopause among some female smokers

PUBLIC RELEASE DATE:

5-Feb-2014

Contact: Katie Delach katie.delach@uphs.upenn.edu 215-349-5964 University of Pennsylvania School of Medicine

PHILADELPHA - New research is lighting up yet another reason for women to quit smoking. In a study published online in the journal Menopause, researchers from the Perelman School of Medicine at the University of Pennsylvania report the first evidence showing that smoking causes earlier signs of menopause in the case of heavy smokers, up to nine years earlier than average in white women with certain genetic variations.

Though previous studies have shown that smoking hastens menopause by approximately one to two years regardless of race or genetic background, this study is the first of its kind to demonstrate that genetic background is significantly associated with a further increased risk of menopause in some white women who smoke. No statistically significant relationships between smoking, the gene variants under investigation and earlier menopause were observed in African American women.

While symptoms of menopause such as hot flashes, anxiety and insomnia can result in discomfort, embarrassment, and irritability, the onset of menopause is also associated with risks of coronary artery disease, osteoporosis, and death from all causes. On average, women enter menopause at around 50 years of age. However, the research team now reports that menopause may begin at an earlier age in white female smokers who are carriers of two different gene variants. While the genes themselves do not result in early onset menopause, variations of the genes CYP3A4*1B and CYP1B1*3 were found to increase the risk of entering menopause at an earlier age in white smokers. The genetic variants were present in seven and 62 percent of white women in the study population, respectively.

"This study could shed new light on how we think about the reproductive risks of smoking in women. We already know that smoking causes early menopause in women of all races, but these new results show that if you are a white smoker with these specific genetic variants, your risk of entering menopause at any given time increases dramatically," said the study's lead author Samantha F. Butts, MD, MSCE, assistant professor of Obstetrics and Gynecology at Penn Medicine.

Results of the study, which enrolled over 400 women aged 35 to 47 from the Penn Ovarian Aging Study, found that in carriers of the CYP3A4*1B variation, the average time-to-menopause after entering the study in heavy smokers, light smokers, and nonsmokers was 5.09 years, 11.36 years, and 13.91 years, respectively. This means that for heavily smoking white females with this genetic background, the average time-to-menopause was approximately nine years earlier than in nonsmoking carriers.

In white carriers of the CYP1B1*3 variation, the average time-to-menopause in heavy smokers, light smokers, and nonsmokers was 10.41 years, 10.42 years, and 11.08 years, respectively -- a statistically significant difference although not as stark as the findings for the CYP3A4*1B variant.

The Penn study did not examine why no statistically significant relationships between smoking, the gene variants under investigation, and earlier menopause were observed in African Americans.

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Penn study reveals genetics impact risk of early menopause among some female smokers

National Poll Shows Public Divided on Genetic Testing to Predict Cancer Risk

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Newswise Salt Lake City, UT A national poll from the University of Utahs Huntsman Cancer Institute shows 34 percent of respondents would not seek genetic testing to predict their likelihood of developing a hereditary cancer even if the cost of the testing was not an issue. Concerns about employment and insurability were cited as the primary reason, even though current laws prohibit such discrimination.

The poll also shows only 35 percent of respondents would be extremely or very likely to seek aggressive prophylactic or preventive treatment, such as a mastectomy, if they had a family history of cancer and genetic testing indicated a genetic pre-disposition to cancer.

I see patients every week who could have taken steps to reduce their risk if theyd known theyd had a predisposition for a certain type of cancer. The best treatment for cancer is prevention, of which genetic testing plays an integral role, said Saundra Buys, M.D., co-director of the Family Cancer Assessment Clinic and medical director of the High Risk Cancer Research at Huntsman Cancer Institute (HCI), and professor of medicine at the University of Utah. In addition to educating the public about the important role genetic testing plays in both prevention and treatment of cancer, we must also work to eliminate perceived false barriers to testing, such as concerns about insurability and employment.

Nearly 40 percent of those who said they wouldnt seek testing reported being somewhat or extremely concerned that the results would impact opportunities for employment, while 69 percent of that same group reported being somewhat or extremely concerned that the results would have an adverse impact on their ability to get insurance.

Inherited mutations play a major role in the development of approximately 5 percent of all cancers. Genetic mutations associated with more than 50 hereditary cancer syndromes including those discovered at the University of Utah for melanoma, colon and breast cancer have been identified.

Buys says the survey demonstrates that even with increased media attention to genetic testing in recent months more work is needed to educate the public about the type of information genetic testing provides and who should seek it. She says family and personal health history are the most important factors in determining whether a person should consider genetic testing.

She warns, however, that genetic testing is only as good as the genetic counseling that accompanies it. There are many genetic tests being ordered in physician offices around the country without the benefit of genetic counseling. The results of these tests are complex, and without appropriate counseling, can cause confusion and unneeded anxiety for patients, said Buys.

Other findings from the poll:

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National Poll Shows Public Divided on Genetic Testing to Predict Cancer Risk

Study Reveals Genetics Impact Risk of Early Menopause Among Some Female Smokers

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Newswise PHILADELPHA - New research is lighting up yet another reason for women to quit smoking. In a study published online in the journal Menopause, researchers from the Perelman School of Medicine at the University of Pennsylvania report the first evidence showing that smoking causes earlier signs of menopause in the case of heavy smokers, up to nine years earlier than average in white women with certain genetic variations.

Though previous studies have shown that smoking hastens menopause by approximately one to two years regardless of race or genetic background, this study is the first of its kind to demonstrate that genetic background is significantly associated with a further increased risk of menopause in some white women who smoke. No statistically significant relationships between smoking, the gene variants under investigation and earlier menopause were observed in African American women.

While symptoms of menopause such as hot flashes, anxiety and insomnia can result in discomfort, embarrassment, and irritability, the onset of menopause is also associated with risks of coronary artery disease, osteoporosis, and death from all causes. On average, women enter menopause at around 50 years of age. However, the research team now reports that menopause may begin at an earlier age in white female smokers who are carriers of two different gene variants. While the genes themselves do not result in early onset menopause, variations of the genes CYP3A4*1B and CYP1B1*3 were found to increase the risk of entering menopause at an earlier age in white smokers. The genetic variants were present in seven and 62 percent of white women in the study population, respectively.

This study could shed new light on how we think about the reproductive risks of smoking in women. We already know that smoking causes early menopause in women of all races, but these new results show that if you are a white smoker with these specific genetic variants, your risk of entering menopause at any given time increases dramatically, said the studys lead author Samantha F. Butts, MD, MSCE, assistant professor of Obstetrics and Gynecology at Penn Medicine.

Results of the study, which enrolled over 400 women aged 35 to 47 from the Penn Ovarian Aging Study, found that in carriers of the CYP3A4*1B variation, the average time-to-menopause after entering the study in heavy smokers, light smokers, and nonsmokers was 5.09 years, 11.36 years, and 13.91 years, respectively. This means that for heavily smoking white females with this genetic background, the average time-to-menopause was approximately nine years earlier than in nonsmoking carriers.

In white carriers of the CYP1B1*3 variation, the average time-to-menopause in heavy smokers, light smokers, and nonsmokers was 10.41 years, 10.42 years, and 11.08 years, respectively -- a statistically significant difference although not as stark as the findings for the CYP3A4*1B variant.

The Penn study did not examine why no statistically significant relationships between smoking, the gene variants under investigation, and earlier menopause were observed in African Americans.

It is possible that uniform relationships among white and African American women were not found due to other factors associated with race that modify the interaction between smoking and genes, said Butts. It is well known that race affects multiple features of menopause, and this could be another. Further investigation is needed to clarify this question.

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Study Reveals Genetics Impact Risk of Early Menopause Among Some Female Smokers

TV Gessulli – Choice Genetics – Video


TV Gessulli - Choice Genetics
Yves Naveau, diretor-geral da Choice Genetics, fala sobre os resultados da empresa em 2013 comentando a nova identidade assumida no ano passado.

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Bio 8.6 Non Mendelian Genetics – Multiple Alleles & Polygenic Traits – Video


Bio 8.6 Non Mendelian Genetics - Multiple Alleles Polygenic Traits

By: Heather Newman

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Bio 8.6 Non Mendelian Genetics - Multiple Alleles & Polygenic Traits - Video

Genetics 2-1-14 opening for – Video


Genetics 2-1-14 opening for
Stir Fried w/ Michael Kang Quixotes Denver CO.

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Genetics – The Whale Song – Video


Genetics - The Whale Song
Opening for Stir Fried wsg Michael Kang of the String Cheese Incident. 02-01-14 Quixotes Denver CO.

By: katerhake

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Genetics - The Whale Song - Video

Trinomial expansion applied to Genetics – Video


Trinomial expansion applied to Genetics
How to Expand (a+b+c)^2 ? (a + b + c)^2 = (a + b + c)(a + b + c) = a(a + b + c) + b(a + b + c) + c(a + b + c) = a^2 + ab + ac + ba + b^2 + bc + ca + cb + c^2...

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Trinomial expansion applied to Genetics - Video

Quantitative Genetics with Mice – Video


Quantitative Genetics with Mice
Go here for the full lesson on TED Ed:http://ed.ted.com/on/r5q7oxwk Real footage of a cross between an albino and agouti mouse. Stay tuned for a video on the...

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Quantitative Genetics with Mice - Video

Myriad Genetics to expand with acquisition of disease monitoring company

Myriad Genetics, seen here in this June 2013 file photo, is acquiring global diagnostics company, Crescendo Biosciences in a $270 million cash deal.

Laura Seitz, Deseret News

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SALT LAKE CITY A local genetics testing giant is acquiring a global diagnostics company that is leading out in autoimmune and inflammatory diseases common to the American public and throughout the world.

Officials at Salt Lake City-based Myriad Genetics hope that by expanding its base and incorporating novel protein-based diagnostics for monitoring disease activity, it can impact more lives.

In a Tuesday announcement, Myriad officials said it is planning to pay $270 million for California-based Crescendo Biosciences, a molecular diagnostics company dedicated to developing and commercializing quantitative blood tests for rheumatoid arthritis and other autoimmune diseases.

"Crescendo Bioscience fits well into our diagnostic portfolio that is focused on saving lives and improving the quality of life of patients across major diseases," said Peter Meldrum, Myriad president and CEO. He said the products from both companies pair well and promise a great future for the company.

Meldrum said Crescendo's core product, a quantitative protein-based test called Vectra DA, "represents a $3 billion global market opportunity for Myriad," which already markets its genetics testing products in the United States and abroad.

The tests help find gene mutations, of which Myriad can classify as harmful or benign. The company promises a less than two-week turnaround time for its tests, which is "essential where the technical results may guide surgical decisions," Meldrum said.

"Gene mutation classification is a numbers game and since we test hundreds of thousands of patients every year, we believe we will dramatically expand our mutation classification advantage and further widen the gap between Myriad and its competition," he said.

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Myriad Genetics to expand with acquisition of disease monitoring company

Gene therapy pioneer uniQure raises $82 million in U.S. IPO

(Reuters) - A small Dutch company behind the Western world's first approved gene therapy priced its shares above the expected range in a U.S. stock offering on Wednesday, showing the current investor appetite for biotechnology.

Amsterdam-based uniQure said it would sell 5.4 million shares at an initial public offering price of $17.00 each, netting it $81.9 million after expenses. It had previously indicated a price range of $13.00 to $15.00.

The stock was trading just below the issue price at $16.80 by 1600 GMT.

UniQure won approval in November 2012 to sell its drug Glybera in Europe and intends to start selling it as a treatment for the ultra-rare disease lipoprotein lipase deficiency (LPLD) with partner Chiesi in the first half of 2014.

The drug is likely to break new ground as the world's most expensive medicine, with a potential price tag of more than $1 million. A high price is needed because a single dose could last a lifetime, giving uniQure just one shot at recouping its investment.

Chief Executive Jorn Aldag said in 2012 that Glybera could sell for around 250,000 euros a year for five years, implying a total price of 1.25 million euros ($1.6 million).

The company has since said that no decision has been taken on price and its IPO prospectus also said it now believed that a one-time price, rather than an annuity-based system, was the most likely pricing model.

Glybera is a modified virus that delivers the correct version of a gene into people afflicted with LPLD, a hereditary disorder that raises the risk of potentially lethal inflammation of the pancreas.

Rare or so-called orphan diseases are winning increased attention from drug developers and several products from companies including Sanofi, Shire and Alexion already cost hundreds of thousands of dollars a year.

UniQure is not the first gene therapy firm to float on Nasdaq. Bluebird Bio made its debut last June but the Cambridge, Mass.-based company has yet to win a regulatory green light for its products.

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Gene therapy pioneer uniQure raises $82 million in U.S. IPO

Emerging Medical Devices for Minimally Invasive Cell Therapy – Video


Emerging Medical Devices for Minimally Invasive Cell Therapy
Dr. Eoin O #39;Cearbhaill, a lecturer in biomedical engineering at the School of Mechanical and Materials Engineering, University of Dublin, in Dublin, Ireland, ...

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TiGenix : reaches major cell therapy milestone with 1000th.

REGULATED INFORMATION FEBRUARY 4, 2014

TiGenix reaches major cell therapy milestone with 1000th implant of ChondroCelect

Leuven (BELGIUM) - February 4, 2014 - TiGenix (NYSE Euronext: TIG), a leader in the field of cell therapy, announced today that it reached a major milestone with the performance of the 1000th ChondroCelect implantation for cartilage repair in the knee. ChondroCelect is the first cell therapy that was granted approval by the European Medicines Agency (EMA) as an Advanced Therapy Medicinal Product (ATMP). Today it is routinely used in orthopedic centers of excellence across several European countries.

"A 1000 patients have already benefited from this innovative therapy, further supporting its efficacy and safety profile," said Eduardo Bravo, CEO of TiGenix. "A milestone such as today's is a clear demonstration of how far the cell therapy field has progressed over recent years, and I have no doubt that it is on its way to become a mainstay in clinical practice. We will continue to work towards turning our ChondroCelect franchise into a cash flow positive asset, and to push the clinical development of our pipeline of stem cell programs to a successful conclusion."

About ChondroCelect An innovative treatment, ChondroCelect has been shown to result in long-term durable clinical benefits in patients with recent cartilage lesions. Five-year follow-up data confirm that the therapeutic effect and the clinical benefit of ChondroCelect gained over baseline is maintained up to at least five years after the cartilage repair intervention. In addition, the data confirm that early treatment with ChondroCelect results in a superior clinical benefit over microfracture, and a lower failure rate.

Cartilage lesions of the knee are a frequent cause of disability in the active population. Caused by repetitive microtraumata, or due to sports or traffic accidents, cartilage lesions rarely heal spontaneously. When untreated, they predispose to osteoarthritis, which causes disability and represents a major socioeconomic burden. A treatment that allows symptom relief and functional recovery is key. To meet this important medical need, TiGenix developed ChondroCelect, the first cell therapy that was granted approval by the EMA as an ATMP.

ChondroCelect is administered to patients in an autologous chondrocyte implantation procedure known as Characterized Chondrocyte Implantation. TiGenix has designed a sophisticated manufacturing process to preserve the cells' characteristics and biological activity, and to maintain their ability to produce high quality cartilage. This process meets the highest quality standards and has been approved by the EMA.

For more information: Eduardo Bravo Chief Executive Officer eduardo.bravo@tigenix.com

Claudia D'Augusta Chief Financial Officer claudia.daugusta@tigenix.com

About TiGenix

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TiGenix : reaches major cell therapy milestone with 1000th.

Stem cell treatment: Controversial for humans, but not for pets

PHOENIX, Ariz. -

Stem cell research and therapy on humans has traveled a long and often politically troubled path.

Not so for pets, where stem cell treatment has been used for nearly 10 years and now it is so routine, and so successful, it can be done in a day.

Ava is a 90 pound, 2-year-old Akita, who is about to undergo stem cell surgery. A little IV, a little anesthesia and Ava is out.

"It is used for arthritis mostly," said Dr. Velvet Edwards.

Ava is just beginning her day at Pecan Grove Veterinary Hospital in Tempe. Dr. Edwards oversees the stem cell procedure.

"Stem cells are healing cells, so they seek out area of injury damage or destruction," explained Edwards. "They accelerate healing and help the animal, the patient, the pet just use their own natural abilities to get better."

Veterinary stem cells are harvested from the animal's own fat cells. They are separated and processed by machinery right inside the vet's office and then injected back into the dog's trouble spots.

Thanks to new technology developed by Meti Vet, the process is completed in just a day.

"The pet comes in the morning, it's anesthetized and I collect about two to four grams of fat usually behind the shoulder blade," said Edwards. "Then I hand that fat over to my technicians to run it through a series of steps.. basically to dissolve the fat and get down to a little stem cell pellet... Then we take that pellet and we reconstitute it and make it injectable. I will put it back into the animal's body wherever I need it later that day."

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Stem cell treatment: Controversial for humans, but not for pets

New Hope As Researchers Discover Genetic Mutations That Cause Rare and Deadly Lung Disease

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Newswise SALT LAKE CITY A team of researchers, led by physicians and scientists at Intermountain Medical Center and ARUP Laboratories in Salt Lake City, has made a medical breakthrough by discovering genetic mutations that cause a rare and deadly lung disease.

The disease, pulmonary capillary hemangiomatosis or PCH, is a rare cause of pulmonary hypertension, which occurs predominantly in young adults. PCH affects less than one in a million people, and has been extremely difficult and expensive to diagnose, as well as challenging to treat.

This genetic discovery offers new hope.

This is a significant finding. This discovery should advance our understanding of this rare pulmonary vascular disorder and other related disorders, said Greg Elliott, MD, MACP, senior investigator of the study and medical director of the Pulmonary Hypertension Center at Intermountain Medical Center in Murray, Utah, and professor of medicine at the University of Utah School of Medicine.

Results of the study will be published in the February issue of the journal CHEST, the official publication of the American College of Chest Physicians. The study is embargoed by CHEST until Feb 4 at 6am, EST.

Dr. Elliott and his team at Intermountain Medical Center and the University of Utah School of Medicine collaborated with researchers from Columbia University, Vanderbilt University and Mayo Clinic-Scottsdale.

To find the genetic mutation, the research team used a relatively new technology whole exome sequencing performed at ARUP Laboratories in Salt Lake City to test DNA samples. They discovered the genetic mutations in Eukaryotic Translation Initiation Factor 2 Alpha Kinase 4. EIF2AK4 is a protein responsible for down-regulating protein synthesis when cells are exposed to stress.

Researchers found that in patients with the genetic mutations, their bodies don't properly regulate blood vessels in the lung. As a result, the capillaries in the lungs proliferate and the patient develops pulmonary hypertension.

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New Hope As Researchers Discover Genetic Mutations That Cause Rare and Deadly Lung Disease

New hope: Researchers discover genetic mutations that cause rare and deadly lung disease

PUBLIC RELEASE DATE:

4-Feb-2014

Contact: Jess C. Gomez jess.gomez@imail.org 801-718-8495 Intermountain Medical Center

SALT LAKE CITY A team of researchers, led by physicians and scientists at Intermountain Healthcare's Intermountain Medical Center and ARUP Laboratories, has made a medical breakthrough by discovering genetic mutations that cause a rare and deadly lung disease.

The disease, pulmonary capillary hemangiomatosis or PCH, is a rare cause of pulmonary hypertension, which occurs predominantly in young adults. PCH affects less than one in a million people, and has been extremely difficult and expensive to diagnose, as well as challenging to treat.

This genetic discovery offers new hope.

"This is a significant finding. This discovery should advance our understanding of this rare pulmonary vascular disorder and other related disorders," said Greg Elliott, MD, MACP, senior investigator of the study and medical director of the Pulmonary Hypertension Center at Intermountain Medical Center in Murray, Utah, and professor of medicine at the University of Utah School of Medicine.

Results of the study will be published in the February issue of the journal CHEST, the official publication of the American College of Chest Physicians. The study is embargoed by CHEST until Feb 4 at 6am, EST.

Dr. Elliott and his team at Intermountain Medical Center and the University of Utah School of Medicine collaborated with researchers from Columbia University, Vanderbilt University and Mayo Clinic-Scottsdale.

To find the genetic mutation, the research team used a relatively new technology whole exome sequencing performed at ARUP Laboratories in Salt Lake City to test DNA samples. They discovered the genetic mutations in Eukaryotic Translation Initiation Factor 2 Alpha Kinase 4. EIF2AK4 is a protein responsible for down-regulating protein synthesis when cells are exposed to stress.

Read the original post:
New hope: Researchers discover genetic mutations that cause rare and deadly lung disease

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