Archive for the ‘Gene Therapy Research’ Category

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Gene therapy, once off to a rocky start, transforms medicine by getting at the root cause of many diseases

The Science Of The Next 150 Years: 50 Years in the Future

It is 2063. you walk into the doctor's office, and a nurse takes a sample of saliva, blood or a prenatal cell and applies it to a microchip the size of a letter on this page on a handheld device. Minutes later the device reads the test results. The multicolored fluorescence pattern on its display reveals the presence of DNA sequences that cause or influence any of 1,200-plus single-gene disorders. Fortunately, regulatory authorities have approved a cure for each one of these diseases: gene therapy.

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Gene Therapies Will Cure Many a Disease

PUBLIC RELEASE DATE:

5-Mar-2014

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

New Rochelle, NY, March 5, 2014Chronic hepatitis C virus (HCV) infection can lead to serious diseases such as cirrhosis and cancer of the liver, so viral clearance and prevention of relapse are important treatment goals. Low-dose oral interferon may reduce the risk of HCV relapse in patients with mild liver fibrosis according to a study published in Journal of Interferon & Cytokine Research, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Interferon & Cytokine Research website.

In "A Double-Blind Randomized Controlled Study to Evaluate the Efficacy of Low-Dose Oral Interferon-Alpha in Preventing Hepatitis C Relapse," Chuan-Mo Lee and coauthors from several universities and hospitals in Taiwan present the results of a clinical trial comparing the effects of 24 weeks of treatment with two doses of oral interferon-alpha or placebo in patients who achieved viral clearance after successful HCV therapy.

"This is a highly significant study relevant to the optimal use of IFN for HCV treatment," says Co-Editor-in-Chief Ganes C. Sen, PhD, Chairman, Department of Molecular Genetics, Cleveland Clinic Foundation, Ohio.

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About the Journal

Journal of Interferon & Cytokine Research (JICR), led by Co-Editors-in-Chief Ganes C. Sen, PhD, and Thomas A. Hamilton, PhD, Chairman, Department of Immunology, Cleveland Clinic Foundation, is an authoritative peer-reviewed journal published monthly online with Open Access options and in print that covers all aspects of interferons and cytokines from basic science to clinical applications. JICR is an official journal of the International Cytokine and Interferon Society. Complete tables of content and a sample issue may be viewed online on the Journal of Interferon & Cytokine Research website.

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Can low-dose interferon prevent relapse of hepatitis C virus infection?

PUBLIC RELEASE DATE:

4-Mar-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-710-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, March 4, 2014Only about 37% of babies around the world are exclusively breastfed for the first 6 months of life, as recommended by the World Health Organization (WHO). The benefits of breastfeeding for both infants and mothers are well-established. The effectiveness of different types of interventions for promoting exclusive breastfeeding in high-income countries is the focus of a Review article published in Breastfeeding Medicine, the official journal of the Academy of Breastfeeding Medicine published by Mary Ann Liebert, Inc., publishers. The article is available free on the Breastfeeding Medicine website at http://www.liebertpub.com/bfm.

Most interventions designed to encourage women to breastfeed use supportive or educational approaches, with varying levels of success, according to study authors Helen Skouteris and colleagues from Deakin University and University of Melbourne (Melbourne, Australia), and Leeds Metropolitan University (Leeds, UK).

In the article "Interventions Designed to Promote Exclusive Breastfeeding in High-Income Countries: A Systematic Review" the authors evaluate the effectiveness of different interventions, comparing prenatal and postnatal approaches, the duration of the interventions, and identify whether they focus on educating mothers or providing emotional support.

"The search for successful interventions that promote the international goal of exclusive breastfeeding for the first six months of an infant's life has been continual but inconclusive," says Ruth Lawrence, MD, Editor-in-Chief of Breastfeeding Medicine and Professor of Pediatrics, University of Rochester School of Medicine. "Authors Helen Skouteris and colleagues in their extensive review point out that a trial of more support and interventions in the postpartum period may be critical to solving this issue."

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About the Journal

Breastfeeding Medicine, the Official Journal of the Academy of Breastfeeding Medicine, is an authoritative, peer-reviewed, multidisciplinary journal published 10 times per year in print and online. The Journal publishes original scientific papers, reviews, and case studies on a broad spectrum of topics in lactation medicine. It presents evidence-based research advances and explores the immediate and long-term outcomes of breastfeeding, including the epidemiologic, physiologic, and psychological benefits of breastfeeding. Tables of content and a sample issue may be viewed on the Breastfeeding Medicine website at http://www.liebertpub.com/bfm.

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Which interventions are most effective to promote exclusive breastfeeding?

PUBLIC RELEASE DATE:

4-Mar-2014

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

New Rochelle, NY, March 4, 2014Nearly a billion people use Facebook, the largest social networking site, but interacting with someone on social media is not the same as meeting them in person. The results of a study to determine whether Facebook exposure increases or reduces arousal during initial face-to-face encounters, especially among socially anxious individuals, are presented in Cyberpsychology, Behavior, and Social Networking, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Cyberpsychology, Behavior, and Social Networking website.

"Face to Face Versus Facebook: Does Exposure to Social Networking Web Sites Augment or Attenuate Physiological Arousal Among the Socially Anxious?" Shannon Rauch and colleagues, Benedictine University at Mesa, AZ and Providence College, RI, evaluated the study participants for their level of social anxiety and then exposed each of them to a person via Facebook, a face-to-face encounter, or both. During the exposures the researchers measured physiological arousal using the galvanic skin response measure.

"Results appear to indicate that initial exposure to an individual via Facebook may have a negative impact on consequent face-to-face encounters with that individual for those with high social anxiety," says Brenda K. Wiederhold, PhD, MBA, BCB, BCN, Editor-in-Chief of Cyberpsychology, Behavior, and Social Networking, from the Interactive Media Institute, San Diego, CA.

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About the Journal

Cyberpsychology, Behavior, and Social Networking is a peer-reviewed journal published monthly online with Open Access option and in print that explores the psychological and social issues surrounding the Internet and interactive technologies, plus cybertherapy and rehabilitation. Complete tables of content and a sample issue may be viewed on the Cyberpsychology, Behavior, and Social Networking website.

About the Publisher

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Meeting face to face vs. meeting on Facebook -- new study on social anxiety

PUBLIC RELEASE DATE:

4-Mar-2014

Contact: John Ascenzi ascenzi@email.chop.edu 267-426-6055 Children's Hospital of Philadelphia

Two recent genetic studies expand the list of genes involved with body fat and body mass index, and their connection to major Western health problems: heart disease, high blood pressure and diabetes. One study showed that higher body mass index (BMI) caused harmful effects on the risk of type 2 diabetes, high blood pressure and inflammation, while another study found gene signals linked to higher levels of body fat metrics, without showing causality.

"These findings are highly relevant to the obesity pandemic in the United States and many other countries," said geneticist Brendan J. Keating, D. Phil., of the Center for Applied Genomics at The Children's Hospital of Philadelphia. "Of course, much research remains to be performed to discover further genes involved in these complex metabolic diseases, and to better understand how to improve treatments."

Keating, who previously helped create a large gene-discovery tool called the Cardio Chip, was a co-leader of both studies, which drew on large international teams of scientists using DNA, laboratory and disease data from tens of thousands of people.

In the BMI research, published in the Feb. 6 issue of the American Journal of Human Genetics, Keating collaborated with clinical epidemiologist Michael V. Holmes, M.D., Ph.D., of the Perelman School of Medicine at the University of Pennsylvania. That study used a recently developed epidemiology tool called Mendelian randomization (MR) that rules out confounding factors such as behavioral and environmental influences to construct genetic risk scores for specific traits of interest.

The study team analyzed eight population cohorts including over 34,000 individuals of European descent, of whom over 4,400 had type 2 diabetes, over 6,000 had coronary heart disease and over 3,800 had a previous stroke.

Their analysis, concluded the authors, supports the importance of BMI in regulating cardiometabolic traits and the risk of type 2 diabetes. "Our findings suggest that lowering BMI is likely to result in multiple reductions of cardiovascular traits: in blood pressure, inflammation, fasting glucose and insulin, and in the risk of type 2 diabetes," said Keating.

"This study is the first to use this emerging MR technique with a combination of genetic markers known to impact BMI, to assess the causal relationship of BMI and a comprehensive repertoire of traits," said Holmes. He added that, although the study showed that increasing BMI has an undesirable effect on cardiometabolic factors, interestingly, it did not show that higher BMI increased the risk of coronary heart disease.

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CHOP researcher finds more genetic signals linking weight and heart health risk factors

(PRWEB) March 05, 2014

In Latin America, Mexico is the pioneer in genetic test applications. Personalized medicine has only been around for the last 15 years. The ALAMPs intention with personalized medicines was to decrease and identify the predisposition of diseases, and to increase the success rate of therapy.

A list of international and national guests gathered at this ALAMP sponsored event. Attendees included Stefan Long, director of the science department at General Genetics Corporation, the number one laboratory in the study of ADN in the world; Dr Felipe Vzquez Estupin, specialist in family therapy; and Dr. Bernard Esquivel, president of ALAMP, who discussed the transition that personalized medicine proposes for healthy and sick individuals, as well as for health systems, through the integration of personalized medicine into clinical practice of genetic examinations that strengthen such care.

The first international symposium of personalized medicine was directed at any health professional that wanted to incorporate their field in the diverse areas of personalized medicine. This included establishment of the protocols of prevention, follow up and monitoring to the development of specific adequate treatments, and the genetic characteristics of each individual.

As defined by the Presidents Council of Advisors of Science and Technology, personalized medicine discerns if the processes that apply to a patient, or a to a group of patients, are appropriate from view point of the proposed strategies as the response to a specific medicine will be different from the present responses of a patient affected by the same condition. This has allowed, and will continue to allow, the introduction of predictors of any disease, whether it be the presence of mutations of oncogenes, or of regular tumor genes.

This algorithm explains the vision of Mexico for personalized medicine: based on the molecular profile of the patient (his genome), regardless of his age, it identifies the susceptibility of various conditions to develop in the patient. Subsequently they establish educational mechanisms/institutions like nutrition (Nutrigenomics; the individualization of micro-macro nutrients according to the metabolic level expressed by the genes), habits, etc., with the intention of preventing the onset of the disease. Since we are not aware of all the environmental factors that trigger diseases, there is always the possibility of more developing factors. Therefore, it is very important to establish a customized program aimed at the early detection of such pathogens. If one detects many diseases in their initial stage (among these are many types of cancer), one can implement appropriate therapeutic measures that eradicate these diseases or control them quickly, preventing further damage and degeneration of the patient (which is the case in non-communicable chronic diseases, or diabetes).

The first international symposium of personalized medicine addressed issues in various areas of genetic medicine, and addressed the steps that Mexico is taking as the pioneer in its application of personalized medicine in Latin America. As a country, Mexico hopes to reduce the unfavorable economic impact of the 25 chronic-degenerative prevalent diseases in the next 20 years by applying immediate preventive measures through a simple genetic test. The proposed test would cost $420 dollars, which will allow saving on treatment costs. Currently, there are 12.8 million future diabetics that could spend up to $448,000 if they dont detect their disease earlier.

For example, as we know, type 2 diabetes mellitus is one of the major causes of death in Mexico and has a pre-pathogenic period (before it appears, which highlights the genetic susceptibility), and a pathogenic period (with the onset of the disease) stage that doesnt present symptoms. Approximately two years later after the onset of the disease, a large number of the patients are not yet diagnosed, until they have an acute complication, i.e., with the hospital emergency room.

Applying the proposed model of personalized medicine to these patients may:

A)Delay the onset of the diseases by many years, which allows for a better quality of life for the patient and a very important economic savings cost for his or her social security.

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Personalized Medicine: The New Paradigm in the Prevention and Treatment of Diseases

PUBLIC RELEASE DATE:

5-Mar-2014

Contact: Shawna Williams shawna@jhmi.edu 410-955-8632 Johns Hopkins Medicine

Johns Hopkins researchers say they have found one way that a recently discovered genetic mutation might cause two nasty nervous system diseases. While the affected gene may build up toxic RNA and not make enough protein, the researchers report, the root of the problem seems to be snarls of defective genetic material created at the mutation site.

The research team, led by Jiou Wang, Ph.D., an assistant professor of biochemistry and molecular biology and neuroscience at the Johns Hopkins University School of Medicine, reports its finding March 5 on the journal Nature's website.

Two years ago, researchers linked the gene C9orf72, named for its location on the ninth human chromosome, to amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease, and to frontotemporal dementia (FTD).

In ALS, motor neurons nerve cells that carry messages from the brain to muscles degenerate and eventually die, which gradually paralyzes the patient. In FTD, neurons in the frontal and temporal lobes of the brain die. Some scientists think the same genetic and biological processes cause both disorders, but with very different symptoms, depending on where in the brain they occur.

The mutation in C9orf72 is called a hexanucleotide repeat expansion, a six-letter "word" of DNA repeated over and over, in a part of the gene that doesn't contain instructions for making any proteins. Although it's normal to have up to 20 such repeats, some people with ALS or FTD have dozens or even hundreds of them. Studies show the mutation is likely responsible for 4 to 8 percent of cases of sporadic ALS the kind that isn't necessarily hereditary and, in some groups, up to 40 percent of the kind that is.

To learn how the repeated sequence causes disease, the Johns Hopkins scientists looked at the structure of the DNA that makes up the gene and the RNA that carries its instructions. Although DNA and RNA are generally seen as long strands, they can bunch and curl to make 3-D structures.

Working with DNA and RNA they made that bore the six-letter "word" repeat, the researchers figured out that both were forming structures called G-quadruplexes. In these formations, guanines called "G" for short, one of the letters in the repeating DNA "word" link up, making stacks that stick together like tiny shelves. The RNA also forms other shapes in the repeating section hairpins and bulges. The researchers speculate that the G-quadruplexes and other structures might be getting in the way of the nucleic acids' normal functions.

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ALS-linked gene causes disease by changing genetic material's shape

FORTUNE --FirstMark Capital is known for investing in consumer-facing software companies like Pinterest, Riot Games, and Aereo, as well as enterprise deals. But since Matt Turck, a former investor with Bloomberg Ventures, joined the firm one year ago, he's been looking at deals that are more out-there, related to big data and the Internet of Things. Take, for example, his latest investment: Recombine, a bioinformatics company.

Alongside new early-stage firm Vast Ventures, FirstMark has invested $3.3 million in the New York-based startup. Angel investors who participated in the round include Vivek Garipalli, formerly of Blackstone Group (BX), as well as Alexander Saint-Amand, President and CEO of Gerson Lehrman Group, and Zach Weinberg and Nat Turner, who co-founded health care startup Flatiron Health.

Currently, Recombine provides clinical genetic testing services which are faster and cheaper than those offered by LabCo Diagnostic Network and Quest Diagnostics (DGX), the incumbent providers. Those companies' technology, much of which was created in the 1970s, requires a separate test (and separate blood samples) to test for each individual disease, and it costs up to $1,000 for each test. Recombine tests for 213 genetic disorders at once, costing $500 before insurance.

MORE:Apple's CFO says see you in Sept., after 100,000 shares vest

Recombine CEO Alexander Bisignano says the incumbent services use older technology and aren't incentivized to upgrade because they have exclusive contracts with insurance companies. "It is sort of a monopoly," he says. Recombine's services are in use at 60 different medical practices.

But disrupting the medical lab industry is not Recombine's ultimate goal. The company has its sights on something it believes is much more lucrative: big data for genetics. That's where Recombine fits into FirstMark's investment thesis. More than a biotech company, it's a big data company, Turck argues. From his blog post Tuesday:

Recombine has ambitious plans to fully leverage Big Data technology to help decode the myriad aspects of our genome that are still not well understood.

Naturally, Bisignano agrees. "Genetics is entering a future where it is nothing but a data science," he says.

By processing all of the data from its tests, Recombine can glean insights about genes and diseases. The data is anonymous, and Recombine has already obtained Institutional Review Board (IRB) approval to conduct its first large-scale study. "We're getting really high-quality medical data that allows us to be more confident that the signal is outweighing the noise in our results," Bisignano says.

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Genetic testing company Recombine raises $3.3 million

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Newswise Philadelphia, March 4, 2014 Two recent genetic studies expand the list of genes involved with body fat and body mass index, and their connection to major Western health problems: heart disease, high blood pressure and diabetes. One study showed that higher body mass index (BMI) caused harmful effects on the risk of type 2 diabetes, high blood pressure and inflammation, while another study found gene signals linked to higher levels of body fat metrics, without showing causality.

These findings are highly relevant to the obesity pandemic in the United States and many other countries, said geneticist Brendan J. Keating, D. Phil., of the Center for Applied Genomics at The Childrens Hospital of Philadelphia. Of course, much research remains to be performed to discover further genes involved in these complex metabolic diseases, and to better understand how to improve treatments.

Keating, who previously helped create a large gene-discovery tool called the Cardio Chip, was a co-leader of both studies, which drew on large international teams of scientists using DNA, laboratory and disease data from tens of thousands of people.

In the BMI research, published in the Feb. 6 issue of the American Journal of Human Genetics, Keating collaborated with clinical epidemiologist Michael V. Holmes, M.D., Ph.D., of the Perelman School of Medicine at the University of Pennsylvania. That study used a recently developed epidemiology tool called Mendelian randomization (MR) that rules out confounding factors such as behavioral and environmental influences to construct genetic risk scores for specific traits of interest.

The study team analyzed eight population cohorts including over 34,000 individuals of European descent, of whom over 4,400 had type 2 diabetes, over 6,000 had coronary heart disease and over 3,800 had a previous stroke.

Their analysis, concluded the authors, supports the importance of BMI in regulating cardiometabolic traits and the risk of type 2 diabetes. Our findings suggest that lowering BMI is likely to result in multiple reductions of cardiovascular traits: in blood pressure, inflammation, fasting glucose and insulin, and in the risk of type 2 diabetes, said Keating.

This study is the first to use this emerging MR technique with a combination of genetic markers known to impact BMI, to assess the causal relationship of BMI and a comprehensive repertoire of traits, said Holmes. He added that, although the study showed that increasing BMI has an undesirable effect on cardiometabolic factors, interestingly, it did not show that higher BMI increased the risk of coronary heart disease.

Keating also co-led a second study, published Jan. 6 in Human Molecular Genetics, analyzing genes associated with central adiposity. Measures of central adiposity, or body fat, can be derived using waist circumference and waist-to-hip ratio. For assessing the influence of weight-related genes, central adiposity is preferable to BMI, because BMI also reflects the influence of genes affecting height, said Keating.

Excerpt from:
Researcher Finds More Genetic Signals Linking Weight and Risk Factors in Heart Health

GATE BT (Biotechnology) Sample Video Lecture by Career Avenues on Genetics
A detailed presentation and VDO by one of our IIT alumni faculty for GATE Biotechnology course.

By: Career Avenues GATE Coaching

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GATE BT (Biotechnology) Sample Video Lecture by Career Avenues on Genetics - Video

2014 St Pauls Genetics Total Herd Offering Sale Preview GTSM

By: clearvisionimaging

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2014 St Pauls Genetics Total Herd Offering Sale Preview GTSM - Video

Becoming Immortal with Advanced Genetics -- BKC Attack of the B-Team -- Episode 4
What does the ultimate lifeform consist of? Flight; immunity to lava, water, and damage in general; speed; health; and some other cool abilities. Today, I sh...

By: martyrsvale

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Becoming Immortal with Advanced Genetics -- BKC Attack of the B-Team -- Episode 4 - Video

Collegiate Reynolds Genetics VL 2.mp4
Table of Contents: 00:00 - Mendelian Genetics terms 03:07 - Mendel #39;s first cross 03:08 - Mendel #39;s second cross (getting gutsy!) 03:09 - Mendel #39;s first cross ...

By: Marc Reynolds

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Collegiate Reynolds Genetics VL 2.mp4 - Video

Collegiate Reynolds Genetics VL3.mp4
Table of Contents: 00:00 - Mendel #39;s second cross (getting gutsy!) 00:17 - Mendel #39;s first cross 02:49 - Mendel #39;s second cross (getting gutsy!) 04:05 - Mendel #39;...

By: Marc Reynolds

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Collegiate Reynolds Genetics VL3.mp4 - Video

Recently, favorable data from Myriad Genetics Inc. 's ( MYGN ) PROCEDE 500 study was published in the journal Current Medical Research and Opinion . Per the findings of this data, 65% of physicians changed their original treatment plan on prostate cancer patients based on Myriad Genetic's 46-gene molecular diagnostic testProlaris. Following this news, shares of Myriad Genetics gained 2.2% at yesterday's market close.

As per the PROCEDE 500 study data, 65% cases were voted by physicians as requiring a change in treatment options. Among them, therapeutic burden had been reduced for 40% of the cases while the remaining experienced an increase in therapeutic burden. Further, 37% of the patients revealed a reduction in the need for interventional therapy while 23% recorded an increase in the need for the same.

Overall, there was a 50% reduction in surgical interventions and 30% fall in radiation treatment. Moreover, 96% of the patients whose treatment options were initially undecided, selected non-interventional options after receiving the Prolaris score. According to the company, all these positive data signify high clinical utility of the Prolaris test among urologists.

Myriad is currently opting for Medicare reimbursement of Prolaris and has submitted the PROCEDE 500 data as part of the Prolaris clinical dossier to the Centers for Medicare & Medicaid Services (CMS). Last month, the company had released positive data on its Prolaris testin the Journal of Urology. According to the company, the Prolaris test has been evaluated in more than 5,000 patients across 11 clinical studiesand six publications in peer-reviewed medical journals. Based on this strong data set, Myriad Genetics is highly optimistic about the reimbursement coverage by CMS which the company expects by the end of June 2014.

The Prolaris test correctly predicted those prostate cancer patients who had the risk of biochemical recurrence (BCR) or metastatic disease following a radical prostrate surgery. The predictions were made based on biopsies conducted earlier on these patients. With more than 30,000 patients who might perish on account of this fatal disease in 2014, there is urgent need for improved diagnosis. This is a clear indication of the huge market potential that Prolaris test holds in the forthcoming period.

Myriad Genetics is currently targeting expansion of its pipeline with products for diverse indications including oncology, women's health, urology, dermatology, autoimmune and inflammatory disease and neuroscience. To achieve this objective, the company has decided to pursue internal developments, in-licensing of technologies and acquisitions to expand its business. We are sanguine about these developments as some of the pipeline candidates look promising enough to cater to a billion-dollar market size.

Myriad Genetics' increasing focus on the companion diagnostic market should work in its favor fuelling growth. We look forward to the expansion of indications and derive comfort from the company's plan to foray into the dermatology, autoimmune and neuroscience market in the near future, on the back of portfolio development.

Currently, the stock carries a Zacks Rank #3 (Hold). Better-placed stocks that are worth a look include Amgen Inc. ( AMGN ), Biogen Idec Inc. ( BIIB ) and Actelion Ltd. ( ALIOF ). All the three stocks carry a Zacks Rank #2 (Buy).

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Myriad Genetics Up on Prolaris Data; Aims Reimbursement - Analyst Blog

PUBLIC RELEASE DATE:

4-Mar-2014

Contact: Press Office presse@helmholtz-muenchen.de 49-893-187-2238 Helmholtz Zentrum Mnchen - German Research Center for Environmental Health

Lentiviruses, which belong to the family of retroviruses, are used as vectors to exchange genetic material in cells and can be used to replace a defective gene as defined by gene therapy. Increasing the efficiency of such a treatment poses a major medical challenge: the virus should specifically track the target cells, but the number of virus used should be as low as possible.

A research team led by Dr. Ines Hfig and Dr. Natasa Anastasov from the Institute of Radiation Biology (ISB) at Helmholtz Zentrum Mnchen in cooperation with Sirion Biotech GmbH in Munich and the Fraunhofer Institute in Aachen has now developed an adjuvant which enhances the effect of the virus transduction. Thus the transfer into the target cells is optimized without additional toxicity.

Surface molecules fuse viruses with target cells

The scientists equipped the viruses with additional surface molecules that facilitate the attachment of the viruses to their target cells. The surface molecules consist of a glycoprotein which is fused to an antibody fragment. This antibody fragment detects the surface receptors of specific target cells, such as EGFR+ or CD30+, and binds to these.

Higher transduction rate less virus used

"Through this specific binding to the target cell we can enhance three fold the transduction rate (transfer of the viruses into the target cells)," said research group leader Anastasov. "Thus, the transduction efficiency is improved, and at the same time fewer transfer viruses are needed."

In further studies, analog to the established system, suitable antibody fragments shall be evaluated for specific surface markers of various target cells, e.g. for bone marrow stem cells and immune cells. Gene therapy can thus be used as a treatment for specific genetic disorders (e.g. metachromatic leukodystrophy, Wiskott-Aldrich syndrome).

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Gene transfer optimization

The synergy demonstrated between SNS01-T and current standard-of-care drugs further emphasizes the therapeutic potential of SNS01-T. We are excited by these results since there may be utility for SNS01-T-like therapeutics in a range of human malignancies by making simple changes to customize our drug candidate for particular cancer types, stated Professor John Thompson, Ph.D., Chief Scientific Officer of Senesco. Particularly striking is the observation that cancer cells take up more SNS01-T than normal cells and that the malignant cells are also more sensitive to the drug.

SNS01-T was taken up by a series of B-cell tumor cell lines, including multiple myeloma, where uptake was up to 5-fold higher than uptake by normal nave B cells. Uptake into myeloma cells induced ~ 45% cell death within 24 hours, whereas there was almost no measureable death of normal nave B cells. Treatment with SNS01-T resulted in significant dose-dependent inhibition of tumor growth in animal models of multiple myeloma, mantle cell lymphoma and diffuse large B-cell lymphoma, with up to 85-90% inhibition at the highest doses. SNS01-T at 0.18 mg/kg significantly extended the life span of treated mice. There was also a reduction in the pro-survival form of the eIF5A protein in tumor tissue, consistent with drug activity. Finally, the combination of SNS01-T and lenalidomide (the active component of Revlimid) resulted in 100% survival of mice compared to 60% (SNS01-T) and 20% (lenalidomide) survival for either drug alone. Tumors were eradicated after a single 6-week cycle of the combination in 67% of the animals, and there was no regrowth after an additional 8 weeks without further treatment. Similarly, the combination of SNS01-T and bortezomib (the active component of Velcade) inhibited tumor growth by 89% compared to 59% (SNS01-T) and 39% (bortezomib) for either drug alone.

We are pleased that the manuscript from Professor Thompsons lab at the University of Waterloo has been published by Molecular Therapy, stated Dr. Leslie J. Browne, President & CEO of Senesco. Our work at Waterloo in cancer models provides evidence that SNS01-T could become a viable treatment strategy for multiple myeloma, mantle cell lymphoma, diffuse large B-cell lymphoma and other B-cell cancers.

About Senesco Technologies, Inc.

Senesco Technologies is a clinical-stage biotech company specializing in cancer therapeutics. Its proprietary gene regulation technology has demonstrated the ability to eliminate cancer cells and protect healthy cells from premature death. The Company is currently in a Phase 1b/2a trial with a product candidate that is designed to treat B-cell cancers, which include multiple myeloma, chronic lymphocytic leukemia, and non-Hodgkins B-cell lymphomas. The technology was developed over the last 15 years through the discovery that the genetic pathway for cell growth control is common to both plants and humans. For more information, please visit Senesco.com or connect with us on Facebook, Twitter, LinkedIn and Google+.

About SNS01-T

SNS01-T is a novel approach to cancer therapy that is designed to selectively trigger apoptosis in B-cell cancers such as multiple myeloma, and, mantle cell and diffuse large B-cell lymphomas. Senesco is the sponsor of the Phase 1b/2a study that is actively enrolling patients at Mayo Clinic in Rochester, MN, the University of Arkansas for Medical Sciences in Little Rock, AK, the Mary Babb Randolph Cancer Center in Morgantown, WV, the John Theurer Cancer Center at Hackensack University Medical Center in Hackensack, NJ and the Seattle Cancer Care Alliance in Seattle, WA. http://www.clinicaltrials.gov/ct2/show/NCT01435720?term=SNS01-T&rank=1

Forward-Looking Statements

Certain statements included in this press release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results could differ materially from such statements expressed or implied herein as a result of a variety of factors, including, but not limited to: the Companys ability to continue as a going concern; the Companys ability to recruit patients for its clinical trial; the ability of the Company to consummate additional financings; the development of the Companys gene technology; the approval of the Companys patent applications; the current uncertainty in the patent landscape surrounding small inhibitory RNA and the Companys ability to successfully defend its intellectual property or obtain the necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Companys research and development programs and collaborations; the success of the Company's license agreements; the acceptance by the market of the Companys products; the timing and success of the Companys preliminary studies, preclinical research and clinical trials; competition and the timing of projects and trends in future operating performance, the quotation of the Companys common stock on an over-the-counter securities market, the Companys ability to execute a transaction with Fabrus, Inc., as well as other factors expressed from time to time in the Companys periodic filings with the Securities and Exchange Commission (the "SEC"). As a result, this press release should be read in conjunction with the Companys periodic filings with the SEC. The forward-looking statements contained herein are made only as of the date of this press release, and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.

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In Non-clinical Cancer Studies Senescos Therapeutic Candidate, SNS01-T, Works Synergistically in Combination with the ...

Experience and expertise run in the genes of this doctor, a third-generation fresh cell therapy practitioner

It was a pleaseant, winter morning in Edenkoben, Germany and a group of 15 people from various countries such as Indonesia, the Philippines, Italy, and Germany congregated for breakfast in a coffee shop in this quaint city. Most of these people just flew in from their respective countries, or drove in from different European cities.

But they were not there for an international conference. They were all there for their shots of fresh cell from Dr. Robert Janson-Mueller.

For the past couple of years, through his solo practice, Dr. Robert Mueller has been sharing the benefits of fresh cell therapy with people who need to seek alternative means to remedy various diseases or chronic conditions of their body, or anti-aging solutions.

Although Filipinos has heard of stem cell therapy only in recent years, thanks to celebrities and politicians who have undergone the treatment and do swear by its efficacy, fresh cell therapy has been around since the 1930s.

The Swiss doctor Prof. Paul Niehans first injected cells originating from animal organs intramuscular into patients in 1931 and is thus considered the founder of live cell therapy. Dr. Robert Muellers grandfather, Dr. Philipp Janson, was one of the first doctors to introduce this method in Germany in 1949. His father, Dr. Wolfgang Janson-Meller, also extensively practiced for 35 years.

Since the 90s, I have been able to participate in the wealth of experience that my father, who is always available for help and advice, has gladly passed on to me. I have been using this method of treatment in my own practice since 2003, says Dr. Mueller.

However, the 47-year old doctor differentiates his practice from others (there are only five known doctors who do fresh cell therapy in Germany) because his clinic tailor-fits the fresh cell injections according to the specific needs of the individuals body. A patient thus gets from about eight to 30 injections, depending on the needs.

In this interview with Dr. Mueller, the German expert sheds more light on this therapy that is attracting more and more Filipinos as an alternative treatment. He also explains why fresh cell therapy is not a cure-all or a miracle therapy, why cells from the sheep embryo is being used, why the treatment is becoming popular in Asia, and why it is not possible, up to now, that these therapies can be done in the Philippines.

For more information on fresh cell therapy, visit the website http://www.janson-mueller.de or call Joey Santos at 0917898-6564 or 633-8653.

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Cracking the fresh cell code

Cancer is the second most common cause of death in Switzerland. There are many reasons why in the era of cutting-edge medicine it is still difficult to cure this disease. A tumor may, for instance, consist of different tumor cell subpopulations, each of which has its own profile and responds differently to therapy -- or not. Furthermore, the cancer cells and the healthy cells in the body interact and communicate with one another. How a tumor then actually develops and whether metastases form depends on which signals a tumor cell receives from its environment.

With the development of a new method the team around Prof. Bernd Bodenmiller from the Institute of Molecular Life Sciences at the University of Zurich -- in cooperation with ETH Zurich and University Hospital Zurich -- has succeeded in comprehensively profiling and visualizing tumor cells from patient samples. This promising method has now been published in Nature Methods.

New imaging method -- major opportunity

Setting out to determine a tumor's cell profile, its neighborhood relationships and the circuit structure within and in between cells is a highly complex endeavour. This is because the biomarkers, i.e. the specific molecules of the various cell types and their circuits, have to be measured in their spatial relationships. "With our method it is possible to obtain a comprehensive picture using a novel imaging technique that currently can simultaneously record 32, and in the near future more than one hundred biomarkers," explains Bernd Bodenmiller, the study coordinator. Furthermore, thanks to state-of-the-art imaging the information about the cells' neighborhood relationships is kept and their direct impact on the cellular switch and control circuits can be visualized.

The new technique is based on methods which are already routinely used in hospitals -- with two important innovations. First, the biomarkers are visualized using pure metal isotopes instead of dyes. To do so, biomarkers on very thin tissue sections are labelled with antibodies. The antibodies are coupled to the pure metal isotopes. Then tiny pieces of tissue are removed with a laser system developed by Prof. Detlef Gnther from the ETH Zurich, and the metal isotopes of the pieces are measured with a mass spectrometer which can determine the mass and quantity of the individual metal isotopes. "This trick gets round the problem of the limited number of colours in the analysis of biological samples," comments Bodenmiller.

Secondly, information about the cells, and their control circuits, is no longer qualitative. With the new measurement method it is possible to precisely determine which cells experience what effect and to which extent. In this way the weak points of the control system can be pinpointed and this helps in the development of new therapeutic approaches. This is the reason, so Bodenmiller, why it is becoming increasingly important to understand these interactions for diagnosis and therapy.

Customized treatment is the goal

The initial measurement results of the new biomarker technique for breast cancer have revealed the heterogeneity of tumors. As a consequence of major growth, some tumors suffer from oxygen deficiency on the inside, other misuse the body's own immune cells to drive their growth. Cell-cell interaction and cell location in the centre or on the edges of the tumor also have a decisive influence. One thing is clear: no tumor is like any other and Bodenmiller believes that treatment should reflect this. In a next step his research team wishes to use the new measurement method to explore the roles played by control circuits and cell communication in metastasis formation.

Story Source:

The above story is based on materials provided by University of Zurich. Note: Materials may be edited for content and length.

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Homing in on cancer with new imaging method

Scientists have identified a rare gene mutation that prevents Type 2 diabetes, the New York Times reported.

In a study of 150,000 people, researchers identified a mutation that destroys a gene used by pancreas cells where insulin is made. People with the mutation were found to produce slightly higher levels of insulin and have slightly lower blood glucose levels for life. As a result, that the mutation reduces diabetes risk by two-thirds even among people who are overweight.

The results, published in Nature Genetics, are a first for diabetes research, as they show a benefit of a mutation that destroys a gene.

Researchers hope they may someday be able to develop a drug that mimics the mutation, offering protection against diabetes. However, Pfizer, which helped finance the study, cautions that bringing a new drug to market can take 10 to 20 years.

Scientists say these results are surprising and a powerful step for drug development.

The study is a tour de force, and the authors are the top people in the field, Dr. Samuel Klein, director of the center for human nutrition at Washington University School of Medicine, who was not involved in the study, told The New York Times.

Click for more from The New York Times.

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Gene mutation may prevent Type 2 diabetes

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Almost 26 million children and adults in the US have diabetes, while 79 million of us have pre-diabetes. Now, researchers have identified rare mutations in a gene that they say could prevent type 2 diabetes - the most common form of the disease - even in people who have risk factors for the condition.

The international research team, led by investigators from the Broad Institute of the Massachusetts Institute of Technology and Harvard, and the Massachusetts General Hospital, recently published the findings in the journal Nature Genetics.

The researchers say that if a drug can be created that mimics the protective effects of these mutations, this could open the doors to the prevention of type 2 diabetes.

To reach their findings, the investigators analyzed the genes of 150,000 individuals over five ancestry groups using next-generation sequencing.

All participants had severe risk factors for diabetes, including advanced age and obesity. However, none of the subjects had developed the condition and had normal blood sugar levels.

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Genetic mutations discovered that could prevent type 2 diabetes

Genetic engineering talk show

By: Victor Rodriguez

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Genetic engineering talk show - Video

What many didnt realise was that the freaky looking ear was never grown, had nothing to do with genetic engineering and wasnt really a scientific breakthrough at all! Instead, it served as the publics introduction to the new field of tissue engineering, through which researchers attempt to create replacement tissues in the laboratory by combining resorbable materials with stem cells.

Tissue engineers, like those in my laboratory at Kings College London, work to build everything from cartilage to fix creaky arthritic knees to coronary arteries to patch up heart patients. What looked like a human ear grown on a mouse was simply what we call a scaffold, an implantable 3D structure made of a plastic that safely dissolves in the body.

Twenty years later, a UCL-based team led by Dr Patrizia Ferretti is continuing to build on this work to reconstruct ears. Surgeons currently treat microtia, a condition in which children are born with a malformed or missing ear, by taking cartilage from the patients rib and implanting it in the head to form something that looks like an ear.

Dr Ferretti hopes to eliminate the need for this second cartilage-harvesting surgery by growing ear cartilage in the laboratory.

The difference here is that whereas in the 1990s tissue engineers thought that merely forming a scaffold of the correct shape and size would allow us to create a tissue, we now understand that a stem cells perception of its nano-environment plays an important role in determining the tissue it creates.

In short, we can now tailor a scaffold with nano-cues that tell a stem cell to become a liver cell instead of lung.

Dr Ferrettis scaffold does just this. Her team utilises a new nanocaged POSS-PCU scaffold to coax stem cells collected from fat to form cartilage whilst the scaffold slowly melts away.

This exciting material came to light in 2011 when it was used to replace the windpipe of a patient who had to have his own removed because of cancer.

The scaffold here instructed stem cells to create the windpipes lining, essentially using the body as an incubator to help direct their fate. This time, the UCL team utilised a cocktail of chemicals to help push the stem cells to make cartilage, so it remains to be seen if the scaffold will similarly drive ear cartilage formation once placed in the body.

What is clear, however, is that the field of tissue engineering is progressing at a remarkable pace and tailor-made tissues to treat a range of conditions are a real possibility in the near future."

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Commentary: field of tissue engineering is progressing at remarkable pace

Westport, CT - infoZine - E/The Environmental Magazine - "Synthetic biology" (or "synbio") refers to the design and fabrication of novel biological parts, devices and systems that do not otherwise occur in nature. Many see it as an extreme version of genetic engineering (GE). But unlike GE, whereby genetic information with certain desirable traits is inserted from one organism into another, synbio uses computers and chemicals to create entirely new organisms.

Proponents of synbio, which include familiar players such as Cargill, BP, Chevron and Du Pont, tout its potential benefits. According to the Synthetic Biology Engineering Research Center (SYNBERC), a consortium of leading U.S. researchers in the field, some promising applications of synthetic biology include alternatives to rubber for tires, tumor-seeking microbes for treating cancer, and photosynthetic energy systems. Other potential applications include using synbio to detect and remove environmental contaminants, monitor and respond to disease and develop new drugs and vaccines.

"This is the first major use of a synbio ingredient in food, and dozens of other flavors and food additives are in the pipeline, so synbio vanilla could set a dangerous precedent for synthetic genetically engineered ingredients to sneak into our food supply and be labeled as natural," reports Friends of the Earth (FoE), a leading environmental group. "Synthetic biology vanillin poses several human health, environmental and economic concerns for consumers, food companies and other stakeholders."

For example, FoE worries that synbio vanilla (and eventually other synthetic biology additives) could exacerbate rainforest destruction while harming sustainable farmers and poor communities around the world. "Synbio vanillacould displace the demand for the natural vanilla market," reports FoE. "Without the natural vanilla market adding economic value to the rainforest in these regions, these last standing rainforests will not be protected from competing agricultural markets such as soy, palm oil and sugar." Critics of synbio also worry that releasing synthetic life into the environment, whether done intentionally or accidentally, could have adverse effects on our ecosystems.

Despite these risks, could the rewards of embracing synthetic biology be great? Could it help us deal with some of the tough issues of climate change, pollution and world hunger? Given that the genie is already out of the bottle, perhaps only time will tell.

Related Links SYNBERC: http://www.synberc.org FoE: http://www.foe.org Evolva: http://www.evolva.com

Send your environmental questions to: EarthTalk, c/o E - The Environmental Magazine, P.O. Box 5098, Westport, CT 06881;

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EarthTalk: What is Synthetic Biology?

PHILADELPHIA Using new genetic evidence, an international team of scientists led by experts at the Perelman School of Medicine at the University of Pennsylvania and Childrens Hospital of Philadelphia has found that an increased body mass index (BMI) raised the risk for both type 2 diabetes and higher blood pressure. The results add to mounting evidence about the risks of obesity and are of major importance for the obesity pandemic that is affecting the United States where two-thirds of adults are overweight or obese and other countries. According to the findings, published online in The American Journal of Human Genetics, for every 1 kg/m2 increase in BMI equivalent to a 196-pound, 40-year old man of average height gaining seven pounds the risk of developing type 2 diabetes increases by 27 percent. The same rise in BMI also increases blood pressure by 0.7 mmHg.

Our findings provide solid genetic support indicating that a higher body mass index causes a raised risk of type 2 diabetes and high blood pressure, said the studys lead author, Michael V. Holmes, MD, PhD, research assistant professor of Surgery in the division of Transplant at Penn Medicine.

In the new study, the research team used a recently developed statistical tool called Mendelian randomization (MR), which helps researchers identify genes responsible for particular diseases or conditions (such as obesity), independent of potentially confounding factors such as differences in behavior and lifestyle, which can lead to false-positive associations. In this case, the use of MR virtually rules out the possibility that both a high BMI and type 2 diabetes are caused by a third, unidentified factor.

Whether high BMI raises the risk of adverse outcomes is of critical importance given that BMI is modifiable, said Holmes. Now that we know high BMI is indeed a direct cause of type 2 diabetes, we can reinforce to patients the importance of maintaining body mass within established benchmarks.

Results of the new study were based on the assessment of the genotypes for over 34,500 patients from previous studies. In addition to the results on diabetes and blood pressure, Holmes and his colleagues found that an elevated BMI has potentially harmful effects on several blood markers of inflammation. While this could be tied to increased risk for coronary heart disease, the researchers suggest it requires further study.

While this study has strong foundations and implications, there are many more BMI signals emerging, said senior author Brendan Keating, PhD, research assistant professor of Pediatrics and Surgery at Penn Medicine and lead clinical data analyst in the Center for Applied Genomics at The Childrens Hospital of Philadelphia. Future research will likely generate even more useful information about genetics and the associated risks for disease for both physicians and patients.

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Penn Medicine is one of the world's leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of theRaymond and Ruth Perelman School of Medicine at the University of Pennsylvania(founded in 1765 as the nation's first medical school) and theUniversity of Pennsylvania Health System, which together form a $4.3 billion enterprise.

The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 16 years, according toU.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $398 million awarded in the 2012 fiscal year.

The University of Pennsylvania Health System's patient care facilities include: The Hospital of the University of Pennsylvania -- recognized as one of the nation's top "Honor Roll" hospitals byU.S. News & World Report; Penn Presbyterian Medical Center; Chester County Hospital; Penn Wissahickon Hospice; and Pennsylvania Hospital -- the nation's first hospital, founded in 1751. Additional affiliated inpatient care facilities and services throughout the Philadelphia region include Chestnut Hill Hospital and Good Shepherd Penn Partners, a partnership between Good Shepherd Rehabilitation Network and Penn Medicine.

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Penn Study Results Confirm BMI is a Direct Cause of Type 2 Diabetes and High Blood Pressure