Archive for the ‘Gene Therapy Research’ Category

Public release date: 21-May-2013 [ | E-mail | Share ]

Contact: Mark Couch mark.couch@ucdenver.edu 303-724-5377 University of Colorado Denver

AURORA, Colo. (May 21, 2013) Research into genetic features of pulmonary fibrosis by physicians and scientists at the University of Colorado School of Medicine may lead to improved treatment of this deadly lung disease, according to a paper published online by JAMA.

The study found that a particular genetic variation helps predict survival in some cases of pulmonary fibrosis, a chronic progressive disease with a median survival of three years. As a result of the study, researchers may want to consider including genetic testing in future clinical trials.

The researchers looked at whether the MUC5B promoter polymorphism, which previously was found to be associated with developing the disease, is also associated with survival time for individuals with pulmonary fibrosis. MUC5B is a protein that is a component of the mucous produced by the bronchial tubes. Pulmonary fibrosis is a condition where lung tissue becomes thickened, stiff and scarred.

The investigators found that genetic changes in the MUC5B gene are associated with marked improvement in survival among patients with pulmonary fibrosis. This represents the first genetic test that can be used to prognosticate in pulmonary fibrosis and may prove useful taking care of patients with this disease. Currently in the United States, there are no drugs approved for use in cases of the disease. This paper indicates that future drug trials should consider including this genetic test to determine the benefit of their intervention.

The research was supported by funding from the National Heart, Lung and Blood Institute and the Dorothy P. and Richard P. Simmons Endowed Chair for Pulmonary Research at the University of Pittsburgh School of Medicine. Twenty-two authors shared credit for the paper, including researchers from the University of Chicago, the University of Illinois at Chicago, the University of Pittsburgh, Vanderbilt University and InterMune from Brisbane, Calif.

Anna L. Peljto, DrPH, from the Colorado School of Public Health was the first author and David A. Schwartz, MD, chairman of the School of Medicine's Department of Medicine, was the senior author of the paper. Additional authors from Colorado institutions were Tasha E. Fingerlin, PhD, from the Colorado School of Public Health; Lori J. Silveira, PhD, Max A. Seibold, PhD, Kevin K. Brown, MD, and Janet L. Talbert, MS, from National Jewish Health; and Elissa Murphy, MS and Marvin I. Schwarz, MD, from the University of Colorado School of Medicine.

David Schwartz this month gave the American Thoracic Society's Amberson Lecture, an honor bestowed annually on an individual with a career of major lifetime contributions to clinical or basic pulmonary research and/or clinical practice. The JAMA study was released online to coincide with its presentation at the American Thoracic Society's international conference.

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Researchers find genetic tie to improved survival time for pulmonary fibrosis

Public release date: 21-May-2013 [ | E-mail | Share ]

Contact: Mark Couch mark.couch@ucdenver.edu 303-724-5377 University of Colorado Denver

AURORA, Colo. (May 21, 2013) A paper recently published in the New England Journal of Medicine and co-written by physicians and scientists at the University of Colorado School of Medicine finds that an important genetic risk factor for pulmonary fibrosis can be used to identify individuals at risk for this deadly lung disease.

Researchers looked at a fairly common variant of the gene for mucin-5B, a protein that is a component of the mucous produced by the bronchial tubes. While this variant of the MUC5B gene is fairly common, pulmonary fibrosis is an uncommonly reported disease.

In a review of CT scans of more than 2,600 adults who did not have a clinical diagnosis of pulmonary fibrosis, researchers found imaging evidence of lung inflammation and scarring in about 9 percent of those over age 50. In this age group, these abnormal findings on CT scans were significantly more common among the 21 percent people with the MUC5B genetic variant.

Importantly, definite lung fibrosis seen on CT scan was strongly associated with the MUC5B genetic variant. While these abnormalities do not necessarily indicate a disease that will progress, the presence of these abnormalities were associated with more shortness of breath and cough as well as smaller lung sizes and ability to transfer oxygen.

The findings suggest that pulmonary fibrosis, which is a condition where lung tissue becomes thickened, stiff and scarred, may be a part of a much more common, but likely less severe, syndrome and could potentially be predicted on the basis of the MUC5B genetic variant.

David A. Schwartz, MD, chairman of the School of Medicine's Department of Medicine, was a corresponding and senior author of the paper. Gary M. Hunninghake, MD, MPH, from the pulmonary and critical care division of the Department of Medicine at Brigham and Women's Hospital in Boston was a corresponding and first author of the paper. The research is supported by grants from the National Institutes of Health, the National Heart, Lung and Blood Institute and the U.S. Veterans Administration.

Twenty-one authors shared credit for the paper, including researchers from Brigham and Women's Hospital and Boston University. Joining Schwartz from Colorado were Elissa Murphy, MS, and Marvin I. Schwarz, MD, from the School of Medicine and Tasha E. Fingerlin, PhD, from the Colorado School of Public Health.

David Schwartz this month gave the American Thoracic Society's Amberson Lecture, an honor bestowed annually on an individual with a career of major lifetime contributions to clinical or basic pulmonary research and/or clinical practice.

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Researchers find genetic risk factor for pulmonary fibrosis

Genomics Forum 2013 - Joan Scott, The National Coalition for Health Education in Genetics
Managing Incidental Findings: Context Matters DATE: Friday, May 3, 2013 HOSTED BY: Genome BC.

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Hemp 101: Feral Hemp Genetics, A Viable Energy Solution
Hemp entrepreneur and inventor Agua Das explains how feral hemp genetics can be a viable resource to restore industrial hemp-seed development to a scale that...

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Let #39;s Play The Sims 3 - Perfect Genetics - Episode 8 - Parents of the Year
Stefan and Heather continue to work on raising their two children and planning for their third, to Stefan #39;s dismay. A few moments of selfish neglect raise qu...

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Let's Play The Sims 3 - Perfect Genetics - Episode 8 - Parents of the Year - Video

Biology Genetics Sophia and Madison
7th period, Booker T. Washington Judah.

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Biology Genetics Sophia and Madison - Video

Let #39;s Play The Sims 3 - Perfect Genetics - Episode 9 - New View in Riverview
The family braces for Bailey #39;s upcoming birthday to discover if he is indeed the perfect genetic heir. In addition, a new arrival is imminent and it pushes t...

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Kenneth Miller Evolution Creation Genetics Christian Atheist The Collapse of Intelligent Design 4

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Kenneth Miller Evolution Creation Genetics Christian Atheist The Collapse of Intelligent Design 4 - Video

SEATTLE, WA--(Marketwired - May 21, 2013) - Atossa Genetics, Inc. (NASDAQ: ATOS), The Breast Health Company, is sponsoring two screenings of the new film Decoding Annie Parker at the Seattle International Film Festival on June 6 and 8, 2013.

Based on true events, Decoding Annie Parker follows a 15-year war waged on both scientific and emotional fronts by a pair of women demonstrating extreme bravery under pressure. Annie Parker (Samantha Morton) is on intimate terms with breast cancer, having watched both her mother and sister succumb to it. When she herself is diagnosed with the disease, she struggles to hold her family together, displaying a force of spirit that belies the odds. Elsewhere, geneticist Mary-Claire King (Helen Hunt) is researching the idea of an undiscovered link between DNA and cancer, a process that finds her scrambling for both funding and the support of her disbelieving colleagues. How the paths of these two women intersect is funny, irreverent and heartwarming. Director Steven Bernstein's feature debut deftly balances the seriousness of the situation with the all-too-human response, finding unpredictable grace notes of beauty and wit during even the darkest of times. Featuring stellar performances by Morton, Hunt and a first-rate supporting cast, Bernstein's film pays ample tribute to one of the most important scientific discoveries of the 20th century as well as the people forever changed in its wake.

Dr. Steven Quay, chairman, CEO & president of Atossa Genetics, stated, "We are honored to sponsor Decoding Annie Parker, a work that celebrates Dr. Mary-Claire King's laboratory at the University of Washington and its mission to further understand the role of genetics in breast cancer. It is a mission we all applaud at Atossa Genetics, which is focused on assessing breast cancer risk with our recently launched ForeCYTE Breast Health Test. We are also dedicated to the prevention of breast cancer through the development and commercialization of an investigative intraductal therapy approach to treat precancerous abnormalities, including ductal carcinoma in situ."

"The Seattle International Film Festival is proud to partner with Atossa Genetics in honoring Dr. King's discovery of the link between DNA and breast cancer," said Mary Bacarella, Managing Director, Seattle International Film Festival. "Decoding Annie Parker is a film that pays tribute to one of the most important scientific discoveries of the 20th century. Dr. King's BRCA test and Atossa Genetics' ForeCYTE Breast Health Test are changing the future of breast cancer detection and prevention. SIFF is extremely proud to play a role in raising public awareness of this incredible work."

More information about Decoding Annie Parker is available at http://www.siff.net/festival-2013/decoding-annie-parker and http://www.decodingannieparkerfilm.com/.

About the Seattle International Film Festival

Founded in 1976, the Seattle International Film Festival (SIFF) creates experiences that bring people together to discover extraordinary films from around the world with the Seattle International Film Festival, SIFF Cinema and FutureWave Education.

Recognized as one of the top film festivals in North America, the Seattle International Film Festival is the largest, most highly attended film festival in the United States reaching more than 150,000 annually. The 25-day festival is renowned for its wide-ranging and eclectic programming presenting over 250 features and 150 short films from over 70 countries each year.

About Atossa Genetics, Inc.

Atossa Genetics, Inc. (NASDAQ: ATOS), The Breast Health Company, is based in Seattle, WA, and is focused on preventing breast cancer through the commercialization of patented diagnostic medical devices and patented laboratory developed tests (LDT) that can detect precursors to breast cancer up to eight years before mammography, and through research and development that will permit it to commercialize treatments for pre-cancerous lesions.

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Atossa Genetics to Sponsor Screenings of Decoding Annie Parker at the Seattle International Film Festival on June 6 ...

RUTHERFORD, N.J.--(BUSINESS WIRE)--

Cancer Genetics, Inc. (CGIX) ("CGI" or the "Company"), a leader in oncology-focused personalized medicine, will attend for the first time the 2013 American Society of Clinical Oncology (ASCO) Annual Meeting on May 31-June 4, 2013 at McCormick Place in Chicago. CGI will be at stand number 19120 during the event.

The ASCO Annual Meeting brings together more than 25,000 oncology professionals from a broad range of specialties to discuss challenges and developments in cancer care and research.

Fitting with the theme of this years meeting, Building Bridges to Conquer Cancer, CGI has established research collaborations with premier institutions including Memorial Sloan-Kettering Cancer Center, National Cancer Institute, Cleveland Clinic and Mayo Clinic. These and other efforts have resulted in the Companys commercial launch of multiple proprietary tests for the diagnosis and treatment management of hematological and urogenital cancers.

CGI diagnostic tests include MatBA, based on proven genomic data for the clinical management of hematological cancers, and UroGenRA, designed for kidney cancer diagnosis and subtyping.

In addition to presenting these proprietary tests to healthcare professionals, CGI plans to meet with biopharmas as well. CGIs Select One program, launched in early 2012 to serve the clinical trial needs of biopharmas, has shown constant growth since its inception. The Company looks forward to creating new opportunities at the ASCO meeting.

To learn more about the 2013 ASCO Annual Meeting, visit http://chicago2013.asco.org.

About Cancer Genetics, Inc.

Cancer Genetics, Inc. (CGI) is an emerging leader in DNA-based cancer diagnostics and servicessome of the most prestigious medical institutions in the world. Our tests target cancers that are difficult to diagnose and predict treatment outcomes. These cancers include hematological, urogenital and HPV-associated cancers.

We also provide a comprehensive range of non-proprietary oncology-focused tests and laboratory services that provide critical genomic information to healthcare professionals as well as biopharma and biotech. Our state-of-the-art reference lab is focused entirely on maintaining clinical excellence and is both CLIA certified and CAP accredited and has licensure from several states including New York State.

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Cancer Genetics to Attend ASCO Annual Meeting

RUTHERFORD, N.J.--(BUSINESS WIRE)--

Cancer Genetics, Inc. (CGIX) ("CGI" or the "Company"), a leader in oncology-focused personalized medicine, has entered into an agreement with MultiPlan, Inc. (MultiPlan), the industrys most comprehensive provider of healthcare cost management solutions.

As a participating provider in the PHCS and MultiPlan networks, CGI will be able to offer providers with its cutting-edge proprietary tests and testing services in hematological and urogenital cancers. MultiPlan contracts with 900,000 healthcare providers across the United States, and has an estimated 57 million consumers accessing MultiPlan network products.

Dr. Anthony Sposato, MultiPlan Vice President & Corporate Medical Director, stated, The inclusion of CGIs oncology-focused testing services and proprietary tests are a welcome addition to our provider networks, allowing for efficient incorporation of genomic information in patient care.

This is a tremendous opportunity for us and further validates our position in cancer diagnostics, stated Panna Sharma, president and CEO of CGI. We expect the MultiPlan agreement to increase access to our oncology-focused services for patients and clinicians while providing the potential to streamline reimbursements.

About Cancer Genetics

Cancer Genetics, Inc. (CGI) is an emerging leader in DNA-based cancer diagnostics and servicessome of the most prestigious medical institutions in the world. Our tests target cancers that are difficult to diagnose and predict treatment outcomes. These cancers include hematological, urogenital and HPV-associated cancers.

We also offer a comprehensive range of non-proprietary oncology-focused tests and laboratory services that provide critical genomic information to healthcare professionals as well as biopharma and biotech. Our state-of-the-art reference lab is focused entirely on maintaining clinical excellence and is both CLIA certified and CAP accredited and has licensure from several states including New York State.

CGI has established strong research collaborations with major cancer centers such as Memorial Sloan-Kettering, The Cleveland Clinic and the National Cancer Institute. For further information, please seewww.cancergenetics.com.

About MultiPlan

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Cancer Genetics Signs Agreement with MultiPlan

Public release date: 20-May-2013 [ | E-mail | Share ]

Contact: Jim Kelly jpkelly@utmb.edu 409-772-8791 University of Texas Medical Branch at Galveston

University of Texas Medical Branch at Galveston researchers have been awarded a five-year, $1.8 million grant by the National Institute of Neurological Disorders and Stroke to apply the techniques of gene therapy to the problem of neuropathic pain that is, pain that arises from a malfunction in the nervous system.

Neuropathic pain is a daily reality for millions of Americans, manifesting itself in a variety of life-impairing ways. Someone suffering from neuropathic pain might feel intense discomfort in response to a light touch, for example, or suddenly feel as though he or she were freezing in response to a small decrease in temperature. Caused by either accidental or disease-induced nerve damage, this kind of pain has proven very difficult to treat.

"Patients in neuropathic pain are willing to do almost anything to get relief," said Dr. Volker Neugebauer, the co-principal investigator on the grant. "They're in torment, often in really desperate situations."

To make matters worse, long-term neuropathic pain often causes depression, acting through emotional mechanisms in the brain meant to underscore the importance of pain signals. Depression further increases the perception of pain, creating a vicious cycle of increasing pain and depression. And while conventional pain medicines can block the pain signal, they are usually successful for only a limited time only; eventually the pain returns when the nervous system compensates for the blockade.

Neugebauer and his UTMB colleague and co-principal investigator Thomas Green believe that a better anti-neuropathic pain strategy is to target higher brain regions and prevent the abnormal generation of persistent emotions. They focus on the amygdala, a structure best known for its role in emotional responses, including anxiety and depression and in Neugebauer's previous work for its connection to pain regulation. Neugebauer and Green hypothesize that stopping abnormal activity in the amygdala by a particular type of receptor for the neurotransmitter serotonin will enable the successful treatment of neuropathic pain.

Although increased serotonin activity in the brain is generally thought of as a good thing it's the mechanism used by many antidepressant drugs activation of the serotonin 2C receptor in the amygdala can cause problems, according to Neugebauer. "In neuropathic pain we see that this receptor is activated on cells that regulate output from the amygdala to brain areas where responses to potentially harmful situations are generated," Neugebauer said. "This activity should be turned off when such response is no longer needed or useful, but these serotonin 2C receptors continue to drive amygdala output, creating a chronic pain state."

In experiments with laboratory rats in which neuropathic pain behavior has been induced by nerve damage, Neugebauer and Green plan to investigate the possibility of "re-normalizing" the amygdala by injecting it with specially designed viruses containing genetic material that blocks cells' generation of serotonin 2C receptors.

"The viruses that we're using are adeno-associated viruses, very common vectors that about 80 percent of the people in our society have been exposed to," Green said. "We've modified them so that they can't replicate, and inserted a gene that instructs the amygdala cells to make small pieces of RNA that interfere with the production of serotonin 2C receptors."

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Pain, pain, go away

SAN DIEGO, May 21, 2013 /PRNewswire/ -- Ceregene, Inc. today announced additional efficacy data from a secondary analysis of its double-blind, randomized, controlled Phase 2b clinical study of CERE-120 (AAV-neurturin). CERE-120 is a gene therapy product designed to deliver the neurotrophic factor neurturin, for Parkinson's disease. This exploratory analysis identified a more robust response to CERE-120 in Parkinson's patients diagnosed within 5 years prior to treatment relative to those diagnosed 10 years or more (p<.005 as measured by change from baseline on the unified parkinson disease rating scale or motor-off primary endpoint for this study. previously ceregene reported that was not met overall study population although one of prescribed secondary endpoints off did demonstrate statistical significance and no safety issues were identified. in addition to cere-120 favorably impacting updrs patients treated within years diagnosis additional exploratory analyses revealed similar improvement same other important measurements including pdq-39 measure quality life patient daily diaries hours without troubling dyskinesias>

Raymond T. Bartus, Ph.D., executive vice president and chief scientific officer of Ceregene stated: "The concept that earlier-stage patients may respond better to neurotrophic factor therapies such as CERE-120 is consistent with the field's long-standing appreciation for their mechanism of action, as well as more recent information derived from autopsy brain tissue donated by Parkinson's disease patients. While a number of practical considerations will have to be addressed in order to design and execute clinical trials that enroll only early-stage patients, this additional exploratory analysis adds further empirical support that the concept deserves serious consideration."

These and other efficacy and safety data related to the CERE-120 program were presented, by invitation, at a symposium at the American Society for Gene and Cell Therapy annual meeting in Salt Lake City on May 18, 2013. The title of Dr. Bartus' talk was: "CERE-120 (AAV2-neurturin) for the treatment of Parkinson's disease: Experience from 4 clinical trials and human autopsy data".

During its 12-year history, Ceregene has established leadership positions in the fields of gene therapy and neurotrophic factors for the treatment of neurodegenerative diseases. In addition to publishing 24 peer-reviewed scientific publications describing novel nonclinical and clinical findings in those fields, the company has safely dosed a total of over 100 patients in two clinical programs: CERE-120 (AAV-NRTN) for Parkinson's disease and CERE-110 (AAV-NGF) for Alzheimer's disease. A randomized, controlled Phase 2 study of CERE-110 for Alzheimer's is continuing. It is fully enrolled and financially supported in large part by a grant from the NIH, with top line data expected by late 2014. In addition to the Parkinson's and Alzheimer's programs, Ceregene has conducted extensive preclinical work with CERE-120 for Huntington's disease, as well as another gene therapy/neurotrophic factor product (AAV-NT4) for blinding ocular diseases (such as Retinitis Pigmentosa, macular degeneration, diabetic retinopathy and glaucoma) and yet another (AAV-IGF1) for Lou Gehrig's disease (ALS). Cergene is currently evaluating strategic alternatives to advance its AAV gene therapy and neurotrophic factor platforms. This study was partially funded by a grant from The Michael J. Fox Foundation for Parkinson's Disease Research.

About CERE-120 and its Application to Treating Parkinson's DiseaseCERE-120 is composed of a harmless adeno-associated virus (AAV) vector carrying the gene for neurturin, a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and restoring function. Neurturin is a member of the same protein family as glial cell line-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties and both have been shown to benefit the midbrain dopamine neurons that degenerate in Parkinson's disease. Degeneration of these neurons is responsible for the major motor impairments of Parkinson's disease. CERE-120 is delivered by stereotactic injection to the terminal fields (i.e., the ends of the degenerating neurons), located in an area of the brain called the putamen, as well as the cell bodies for these same neurons, located in a different area of the brain, called the substantia nigra. Once CERE-120 is delivered to the brain, it provides stable, controlled and highly targeted neurturin expression for years following a single injection, confirmed in both animal and human studies.

About Parkinson's DiseaseParkinson's disease is a progressive movement disorder that affects a million people in the United States. Its main symptoms, stiffness, tremors and slowed movements and gait, are caused by a loss of dopamine-containing nerve cells in the substantia nigra, which project their axons to the putamen. Dopamine is a neurotransmitter involved in controlling movement and coordination, so Parkinson's patients exhibit a progressive inability to initiate and control physical movements. There is currently no treatment that can reverse the degeneration of these neurons, let alone cure Parkinson's disease.

About CeregeneCeregene, Inc. is a San Diego-based biotechnology company focused on the development of nervous system growth factors (neurotrophic factors) as treatments for neurodegenerative and retinal disorders using gene transfer for their delivery. The company has established a leadership position in the fields of gene therapy and neurotrophic factors, having launched 6 separate clinical trials in Parkinson's and Alzheimer's disease, enrolling a total of nearly 200 patients, over 100 whom have been administered the gene therapy products, some several years ago, with no safety serious issues. Ceregene's clinical program for Alzheimer's disease involves CERE-110, an AAV2-based vector expressing nerve growth factor (NGF). A fully enrolled multi-center, controlled Phase 2 study with CERE-110 is ongoing, conducted in collaboration with the Alzheimer's Disease Cooperative Study and partially funded by a grant from the National Institutes of Health (NIH). Ceregene was launched in January 2001. The company's investors include Alta Partners, MPM Capital, Hamilton BioVentures, Investor Growth Capital, California Technology Partners and BioSante Pharmaceuticals.

About The Michael J. Fox Foundation for Parkinson's ResearchAs the world's largest private funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors, and volunteers. In addition to funding more than $325 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of Thousands of Team Fox members around the world.

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Ceregene Reports Additional Efficacy Data From Parkinson's Disease Phase 2b Study

AMSTERDAM, The Netherlands, May 21, 2013 /PRNewswire/ --

uniQure B.V., a leader in the field of human gene therapy, today announced that with its consortium partners it is to receive a EUR 2.5 million Eurostars grant to develop an RNA interference (RNAi) gene therapy for Huntington's disease. The consortium is a pan-European collaboration consisting of uniQure as the coordinator, Lausanne University Hospital, Switzerland, University Medical Center Gttingen, Germany, and Maria Curie-Skodowska University, Poland.

(Logo: http://photos.prnewswire.com/prnh/20130220/595152 )

The program's aim is to develop a gene therapy for the treatment of Huntington's disease (HD), a rare and devastating neurodegenerative disease caused by mutations in the Huntingtin (Htt) gene. As a result of the defective gene, mutated proteins accumulate in the brain and destroy neurons, leading at first to involuntary, random body movements, but eventually to progressive cognitive decline and finally dementia. Onset of the disease usually manifests itself around 35 to 44 years of age, while life expectancy after diagnosis is on average 20 years. The program will start on June 1, 2013 and run for three years.

"Our Huntington's disease program is part of our strategic effort to demonstrate the potential of our AAV-delivery platform in RNAi," says Jrn Aldag, CEO of uniQure. "The RNAi field has great promise to become a new and important treatment modality. However, the field has been held back by the lack of effective delivery mechanisms. We believe that our AAV technology is ideally suited to deliver RNAi compounds with high accuracy and efficacy. In addition, the development with the consortium of a gene expression system for GDNF will not only benefit the HD program, but also holds great promise for our Parkinson's disease program, and potentially other CNS disorders."

About the program

The consortium's goals are to develop a regulated gene expression system for glial cell derived neurotrophic factor (GDNF) to improve the maintenance and survival of neurons as a HD gene therapy, and to develop regulated expression of artificial miRNA to conditionally silence the Htt gene. The main outcome of the program is a robust pre-clinical assessment of the first regulated gene therapy vector suitable for optimized treatment of HD patients. The ability to regulate gene expression would additionally represent an exciting innovation in the field of gene therapy, creating new opportunities to tackle challenging diseases where gene expression is only required at certain times. The consortium expects that the program's results should lead to clinical safety trials within two years after the conclusion of the project.

About uniQure

uniQure is a world leader in the development ofhuman gene based therapies.uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure's product pipeline of gene therapy products in development comprise hemophilia B, acute intermittent porphyria, Parkinson's disease and Sanfilippo B. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform.uniQure's largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at http://www.uniqure.com.

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uniQure and Consortium to Receive EUR 2.5 Million Eurostars Grant to Develop RNAi Gene Therapy for Huntington's Disease

Businessman had an increased risk of cancer through the BRCA2 gene He entered a trial at Institute of Cancer Research as it ran in his family BRAC1 and BRAC2 genes are linked to an aggressive form of cancer Angeline Jolie had a double mastectomy after testing positive for BRAC1

By Sophie Borland

PUBLISHED: 19:21 EST, 19 May 2013 | UPDATED: 02:01 EST, 20 May 2013

A businessman has become the first man to have his prostate removed after discovering he was carrying a faulty gene that raises the risk of cancer.

The 53-year-old was told he had the rogue BRCA2 gene after taking part in a clinical trial at the Institute of Cancer Research in London.

Last week Angelina Jolie revealed that she had had a double mastectomy after discovering that the BRCA1 gene gave her an 87 per cent risk of developing breast cancer.

Scientists believe that other men who know they are carriers will choose to go down the same route (stock image)

Both the BRCA2 and BRCA1 genes have long been known to increase the risk of breast and ovarian forms of the disease but recent research has also linked them to the prostate.

The businessman, who has not been identified but is married with children and lives in London, has other family members who have suffered breast or prostate cancers.

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British businessman has his prostate removed over cancer risk from 'Jolie gene'

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/vrrrm8/growth) has announced the addition of the "Growth Opportunities in Global Steam Turbine Market in Power Generation 2012-2017: Trends, Forecasts and Opportunity Analysis" report to their offering.

The global steam turbine market stands on the threshold of strong growth. In 2011, the market topped $12.1 billion, growing at a Compounded Annual Growth Rate (CAGR) of 2.4%. The Asia Pacific (APAC) region captured the largest market share with approximately $9.3 billion, representing 77% of the market. According to market forecasts, the global steam turbine market is poised for growth at 3% CAGR over the next five years (2012-2017) to reach approximately $15.3 billion in 2017.

The author discusses the challenges and opportunities faced by the global steam turbine market. The steam turbine market is affected by renewable sources of energy such as wind and solar power. The prices of other sources of energy such as natural gas are another factor affecting this market. The demand for steam turbines, however, will remain solid with emerging, large economies, such as India and China, generating high demand.

The study encompasses the market's major drivers. APAC accounts for the largest market share of steam turbines. North America and Europe represent larger markets than the Rest of the World market.

This report highlights the aspects of the global steam turbine market. Due diligence has been given to the current market scenario, as well as the technological and financial benefits of installing steam turbines in power generation plants globally. China has emerged as the leading player in the manufacturing and installation of steam turbines.

For more information visit http://www.researchandmarkets.com/research/vrrrm8/growth

About Research and Markets

Research and Markets is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Research and Markets: Growth Opportunities in Global Steam Turbine Market in Power Generation 2012-2017 Report Lets ...

Public release date: 20-May-2013 [ | E-mail | Share ]

Contact: Evan Lerner elerner@upenn.edu 215-573-6604 University of Pennsylvania

The allure of personalized medicine has made new, more efficient ways of sequencing genes a top research priority. One promising technique involves reading DNA bases using changes in electrical current as they are threaded through a nanoscopic hole.

Now, a team led by University of Pennsylvania physicists has used solid-state nanopores to differentiate single-stranded DNA molecules containing sequences of a single repeating base.

The study was led by Marija Drndi, an associate professor in the Department of Physics and Astronomy in the School of Arts and Sciences, along with graduate students Kimberly Venta and Matthew Puster and post-doctoral researchers Gabriel Shemer, Julio A. Rodriguez-Manzo and Adrian Balan. They collaborated with assistant professor Jacob K. Rosenstein of Brown University and professor Kenneth L. Shepard of Columbia University.

Their results were published in the journal ACS Nano.

In this technique, known as DNA translocation measurements, strands of DNA in a salt solution are driven through an opening in a membrane by an applied electric field. As each base of the strand passes through the pore, it blocks some ions from passing through at the same time; amplifiers attached to the nanopore chip can register the resulting drop in electrical current. Because each base has a different size, researchers hope to use this data to infer the order of the bases as the strand passes through. The differences in base sizes are so small, however, that the proportions of both the nanopores and membranes need to be close those of the DNA strands themselves a major challenge.

The nanopore devices closest to being a commercially viable option for sequencing are made out of protein pores and lipid bilayers. Such protein pores have desirable proportions, but the lipid bilayer membranes in which they are inserted are akin to a film of soap, which leaves much to be desired in terms of durability and robustness.

Solid-state nanopore devices, which are made of thin solid-state membranes, offer advantages over their biological counterparts they can be more easily shipped and integrated with other electronics but the basic demonstrations of proof-of-principle sensitivity to different DNA bases have been slower.

"While biological nanopores have shown the ability to resolve single nucleotides, solid-state alternatives have lagged due to two challenges of actually manufacturing the right-sized pores and achieving high-signal, low-noise and high-bandwidth measurements," Drndi said. "We're attacking those two challenges here."

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Penn research makes advance in nanotech gene sequencing technique

Britain launched a research program on Monday that should eventually allow all cancer patients to have access to the kind of genetic analysis that led Hollywood star Angelina Jolie to decide to undergo a double mastectomy.

The project, involving the Institute of Cancer Research (ICR) in London, the U.S. gene sequencing firm Illumina, geneticists and cancer doctors, aims to find a way to allow more cancer genes be tested in more people.

Researchers announcing the 2.7 million pound ($4 million) project, funded by the Wellcome Trust medical charity, stressed this was not a response to reports last week of Jolie's decision to undergo surgery to reduce her breast cancer risk.

"What we're trying to do here is develop processes that will allow comprehensive and systematic use of genetic information in cancer medicine so that (more people) will be able to benefit from the types of information and situations we were hearing about last week (with the Jolie story)," said Nazneen Rahman, head of genetics at the ICR and a leader on the new project.

Mutations in some genes, known as cancer predisposition genes, greatly increase the risk that a person will get cancer.

Jolie tested positive for a high risk gene mutation that made her about five times more likely to develop breast cancer than women who do not carry this mutation, according to the U.S. National Cancer Institute.

There are nearly 100 other known cancer predisposition genes, but in Britain - where most healthcare is part of the taxpayer-funded National Health Service - testing for them is currently very restricted.

Yet recent advances in reading the genetic code, known as gene sequencing, mean that looking for gene mutations is now faster and cheaper than ever - paving the way for gene testing eventually to become routine for all cancer patients.

"It is very important to know if a mutation in a person's genetic blueprint has caused their cancer," Rahman told reporters at a briefing in London.

"It allows more personalized treatment, so for example such people are often at risk of getting another cancer and may choose to have more comprehensive surgery, or may need different medicines, or extra monitoring."

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UK aims to make gene testing more accessible for cancer patients

By Kate Kelland

LONDON (Reuters) - Britain launched a research program on Monday that should eventually allow all cancer patients to have access to the kind of genetic analysis that led Hollywood star Angelina Jolie to decide to undergo a double mastectomy.

The project, involving the Institute of Cancer Research (ICR) in London, the U.S. gene sequencing firm Illumina, geneticists and cancer doctors, aims to find a way to allow more cancer genes be tested in more people.

Researchers announcing the 2.7 million pound ($4 million) project, funded by the Wellcome Trust medical charity, stressed this was not a response to reports last week of Jolie's decision to undergo surgery to reduce her breast cancer risk.

"What we're trying to do here is develop processes that will allow comprehensive and systematic use of genetic information in cancer medicine so that (more people) will be able to benefit from the types of information and situations we were hearing about last week (with the Jolie story)," said Nazneen Rahman, head of genetics at the ICR and a leader on the new project.

Mutations in some genes, known as cancer predisposition genes, greatly increase the risk that a person will get cancer.

Jolie tested positive for a high risk gene mutation that made her about five times more likely to develop breast cancer than women who do not carry this mutation, according to the U.S. National Cancer Institute.

There are nearly 100 other known cancer predisposition genes, but in Britain - where most healthcare is part of the taxpayer-funded National Health Service - testing for them is currently very restricted.

Yet recent advances in reading the genetic code, known as gene sequencing, mean that looking for gene mutations is now faster and cheaper than ever - paving the way for gene testing eventually to become routine for all cancer patients.

"It is very important to know if a mutation in a person's genetic blueprint has caused their cancer," Rahman told reporters at a briefing in London.

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UK tries out new model for gene testing in cancer patients

Repeal Cannabis-Hemp-Marijuana Prohibition
Activist, Advocate, Minister, Patient, Survivor,... I, Mary Thomas-Spears aka Mary Thomas aka Rev. Mary,... Founder of Diverse Sanctuary Community Network,.....

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OMD Genetic Engineering (Official Video)
From the album "Dazzle Ships" (1983)

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OMD Genetic Engineering (Official Video) - Video

Public release date: 20-May-2013 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 ext. 2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, May 20, 2013National guidelines recommend that at-risk women be screened for elevated cholesterol levels to reduce their chances of developing cardiovascular disease. But who is 'at risk?' The results of a study by investigators at the Centers for Disease Control and Prevention (CDC) to estimate the proportion of women young and old who have cholesterol levels that meet the definition of being at-risk are reported in an article in Journal of Women's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women's Health website at http://www.liebertpub.com/jwh.

In "Cholesterol Screening for Women: Who is 'At Risk?'" Cheryl Robbins, Patricia Dietz, Shanna Cox, and Elena Kuklina, from the CDC, Atlanta, GA, analyzed data for a representative sample of 1,781 U.S. women not previously diagnosed with elevated cholesterol.

More than half (55%) of younger women (ages 20-44 years) and 74.2% of older women (>45 years) were at-risk for high cholesterol as defined by U.S. Preventive Services Task Force guidelines. Nearly all of the women in both age groups had at least one risk factor that would make them candidates for cholesterol screening according to the American Heart Association risk definition. The authors suggest the need for future research to determine whether screening and treatment of young women with high cholesterol will help to decrease subsequent deaths due to cardiovascular disease.

"The high prevalence of dyslipidemia reported in this study even among younger women is striking and supports the need for increased education about the risks for cardiovascular disease in women," says Susan G. Kornstein, MD, Editor-in-Chief of Journal of Women's Health, Executive Director of the Virginia Commonwealth University Institute for Women's Health, Richmond, VA, and President of the Academy of Women's Health.

###

About the Journal

Journal of Women's Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women's healthcare issues. Complete tables of content and a sample issue may be viewed on the Journal of Women's Health website at http://www.liebertpub.com/jwh. Journal of Women's Health is the Official Journal of the Academy of Women's Health and the Society for Women's Health Research.

About the Academy

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Which women should be screened for high cholesterol?

CARLSBAD, Calif., May 20, 2013 /CNW/ - Life Technologies Corporation (LIFE) today announced that its Applied Biosystems 3500 Dx /3500 xL Dx Genetic Analyzers CS2 have been approved by Health Canada for diagnostic use. The development marks a major extension of Life Technologies' capabilities to serve the clinical end market in North America with Sanger-based solutions.

The Applied Biosystems 3500 Dx Series Genetic Analyzers deliver high quality performance, higher throughput and increased productivity for clinical laboratories around the world. The 3500xL Dx is an automated 24 capillary-based Sanger Sequencer; the 3500 Dx is an eight capillary instrument. Together, the offering of both 3500 Dx and 3500xL Dx provides hospitals of all sizes the flexibility they need to meet their unique throughput demands designed for a wide range of sequencing applications. In addition to diagnostic use, the instruments are appropriate for a wide range of research applications, including de novo sequencing and mutational profiling.

"The current approval of the 3500 Dx Series Genetic Analyzers by Health Canada emphasizes Life Technologies' success in pursuing regulatory pathways for our diagnostics laboratory instruments, as well as our vision to becoming a global leader in the molecular diagnostics industry," said Ronnie Andrews , president of Medical Sciences at Life Technologies.

Applied Biosystems Sanger Sequencers supplied the technology that powered the Human Genome Project, and Sanger instruments remain the sequencing "gold-standard" for accuracy, reliability and ease of use. The participation of the sequencing technologies in clinical diagnostics is reshaping the disease treatment paradigm worldwide.

In February 2013 , the 3500 Dx/3500xL Dx Genetic Analyzers CS2 Series received U.S. Food and Drug Administration (FDA) 510(k) clearance for use with the company's SeCore HLA typing kits. Additional products offered by Life Technologies for the molecular diagnostics lab market in Canada include the Applied Biosystems 7500 Fast Dx Real-time PCR instrument and the QuantStudio Dx Real-time PCR Instrument, both available as Class I devices.

About Life Technologies Life Technologies Corporation (LIFE) is a global biotechnology company that is committed to providing the most innovative products and services to leading customers in the fields of scientific research, genetic analysis and applied sciences. With a presence in more than 180 countries, the company's portfolio of 50,000 end-to-end solutions is secured by more than 5,000 patents and licenses that span the entire biological spectrum -- scientific exploration, molecular diagnostics, 21st century forensics, regenerative medicine and agricultural research. Life Technologies has approximately 10,000 employees and had sales of $3.8 billion in 2012.

Life Technologies' Safe Harbor StatementThis press release includes forward-looking statements about Life Technologies' anticipated results that involve risks and uncertainties. Some of the information contained in this press release, including, but not limited to, statements as to industry trends and Life Technologies' plans, objectives, expectations and strategy for its business, contains forward-looking statements that are subject to risks and uncertainties that could cause actual results or events to differ materially from those expressed or implied by such forward-looking statements. Any statements that are not statements of historical fact are forward-looking statements. When used, the words "believe," "plan," "intend," "anticipate," "target," "estimate," "expect" and the like, and/or future tense or conditional constructions ("will," "may," "could," "should," etc.), or similar expressions, identify certain of these forward-looking statements. Important factors which could cause actual results to differ materially from those in the forward-looking statements are detailed in filings made by Life Technologies with the Securities and Exchange Commission. Life Technologies undertakes no obligation to update or revise any such forward-looking statements to reflect subsequent events or circumstances.

(Logo: http://photos.prnewswire.com/prnh/20110216/MM49339LOGO)

Life Technologies Contact Suzanne Clancy 760-602-4545 858-205-4235 (mobile) suzanne.clancy@lifetech.com

SOURCE: Life Technologies Corporation

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Life Technologies Announces Regulatory Approval of its 3500 Dx Series Genetic Analyzers in Canada

Scientists say they have formed the most complete picture yet of how the body controls the production of proteins needed to stay healthy.

Researchers at the University of Edinburgh are tracking the complex interactions between genes that enable proteins to be produced for use in cells.

They are studying tiny strands of genetic code - known as microRNAs - that switch larger genes on and off to regulate the production process.

Four hundred of these strands have been observed in action for the first time using a new tracking technique developed at the university. Researchers say they have been able to identify which genes are controlled by each microRNA.

Dr Grzegorz Kudla, of the university's Institute of Genetics and Molecular Medicine, said the study had uncovered a wealth of information about the way microRNAs interact with other genetic material in the body.

He said: "Imagine you are in a small town. You know everybody's name because you have access to a phonebook, but you also want to know who interacts with whom.

"You can guess - perhaps people living on the same street interact, or you can spend time asking people about their friends. Our experiment amounts to screening footage from the town's CCTV system to identify pairs of people talking to each other in the street.

"We collected such footage for 18,000 pairs of people - except that in our case, we were looking at 18,000 genes. We already knew that genes often interact with each other, but we didn't know who interacts with whom. Now we know."

Scientists say the study accounts for around 40% of the 1,000 different microRNAs active in the human body, many of which had not been investigated before. The research, published in the journal Cell, is also the first to describe the distinguishing traits of a large number of microRNAs in one go.

Lead researcher Professor David Tollervey, of the university's School of Biological Sciences, said the study gives scientists a rule book for how individual genes are controlled and will also help in the understanding of a range of diseases caused when the regulation process goes wrong.

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Tiny genetic code strands observed

New measure may aid treatment planning, future studies for broad range of tumors

Newswise BOSTON A new measure of the heterogeneity the variety of genetic mutations of cells within a tumor appears to predict treatment outcomes of patients with the most common type of head and neck cancer. In the May 20 issue of the journal Cancer, investigators at Massachusetts General Hospital (MGH) and Massachusetts Eye and Ear Infirmary describe how their measure was a better predictor of survival than most traditional risk factors in a small group of patients with squamous cell carcinoma of the head and neck.

"Our findings will eventually allow better matching of treatments to individual patients, based on this characteristic of their tumors," says Edmund Mroz, PhD, of the MGH Center for Cancer Research, lead author of the Cancer report. "This method of measuring heterogeneity can be applied to most types of cancer, so our work should help researchers determine whether a similar relationship between heterogeneity and outcome occurs in other tumors."

For decades investigators have hypothesized that tumors with a high degree of genetic heterogeneity the result of different subgroups of cells undergoing different mutations at different DNA sites would be more difficult to treat because particular subgroups might be more likely to survive a particular drug or radiation or to have spread before diagnosis. While recent studies have identified specific genes and proteins that can confer treatment resistance in tumors, there previously has been no way of conveniently measuring tumor heterogeneity.

Working in the laboratory of James Rocco, MD, PhD director of the Mass. Eye and Ear /MGH Head and Neck Molecular Oncology Research Laboratory, principal investigator at the MGH Center for Cancer Research and senior author of the Cancer report Mroz and his colleagues developed their new measure by analyzing advanced gene sequencing data to produce a value reflecting the genetic diversity within a tumor not only the number of genetic mutations but how broadly particular mutations are shared within different subgroups of tumor cells. They first described this measure, called mutant-allele tumor heterogeneity (MATH), in the March 2013 issue of Oral Oncology. But that paper was only able to show that patients with known factors predicting poor outcomes including specific mutations in the TP53 gene or a lack of infection with the human papillomavirus (HPV) were likely to have higher MATH values.

In the current study, the investigators used MATH to analyze genetic data from the tumors of 74 patients with squamous cell head and neck carcinoma for whom they had complete treatment and outcome information. Not only did they find that higher MATH values were strongly associated with shorter overall survival with each unit of increase reflecting a 5 percent increase in the risk of death but that relationship was also seen within groups of patients already at risk for poor outcome. For example, among patients with HPV-negative tumors, those with higher MATH values were less likely to survive than those with lower MATH values. Overall, MATH values were more strongly related to outcomes than most previously identified risk factors and improved outcome predictions based on all other risk factors the researchers examined.

The impact of MATH value on outcome appeared strongest among patients treated with chemotherapy, which may reflect a greater likelihood that highly heterogeneous tumors contain treatment-resistant cells, Mroz says. He also notes that what reduces the chance of survival appears to be the subgroups of cells with different mutations within a tumor, not the process of mutation itself. "If all the tumor cells have gone through the same series of mutations, a single treatment might still be able to kill all of them. But if there are subgroups with different sets of mutations, one subgroup might be resistant to one type of treatment, while another subgroup might resist a different therapy."

In addition to combining MATH values with clinical characteristics to better predict a patient's chance of successful treatment, Mroz notes that MATH could someday help determine treatment choice directing the use of more aggressive therapies against tumors with higher values, while allowing patients with lower values to receive less intense standard treatment. While MATH will probably be just as useful at predicting outcomes for other solid tumors, the investigators note, that will need to be shown in future studies.

"Our results have important implications for the future of oncology care," says Rocco, the Daniel Miller Associate Professor of Otology and Laryngology at Harvard Medical School. "MATH offers a simple, quantitative way to test hypotheses about intratumor genetic heterogeneity, including the likelihood that targeted therapy will succeed. They also raise important questions about how genetic heterogeneity develops within a tumor and whether heterogeneity can be exploited therapeutically."

Additional co-authors of the Cancer paper are Aaron Tward, MD, PhD, Mass. Eye and Ear; Curtis Pickering, PhD, and Jeffrey Myers, MD, PhD, University of Texas M.D. Anderson Cancer Center; and Robert Ferris, MD, PhD, University of Pittsburgh Cancer Institute. The study was supported by National Institute of Dental and Craniofacial Research grants R01DE022087 and RC2DE020958, National Cancer Institute grant R21CA119591, Cancer Prevention Research Institute of Texas grant RP100233, and the Bacardi MEEI Biobank Fund. The MGH has filed a patent application for the MATH measure.

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Genetic Diversity Within Tumors Predicts Outcome in Head and Neck Cancer