Archive for August, 2012

Public release date: 2-Aug-2012 [ | E-mail | Share ]

Contact: Jane E. Rubinstein jrubinstein@rubenstein.com 212-843-8287 Autism Speaks

New York, N.Y. (August 2, 2012) Autism Speaks, the world's leading autism science and advocacy organization, today announced its expanded collaboration with Sigma Advanced Genetic Engineering (SAGE) Labs, an initiative of Sigma Life, to develop the first rat models with modified autism associated genes, intended to accelerate discovery and translational autism research.

Expansion of the collaboration follows initial behavioral studies demonstrating that the first two publicly available gene knockout rats, part of the seven rats generated through the collaboration to date, exhibit hallmark characteristics of autism, such as social deficits and repetitive behaviors. Many behavioral characteristics of autism observed in these rats are not seen in other animal models currently used for autism research. SAGE Labs and Autism Speaks now plan to generate additional genetically modified rat models of key autism-associated genes, including CNTNAP2 and MET.

"Autism spectrum disorders are a complex condition with significant unmet medical needs. Although uniquely human, fundamental aspects of the biology underlying autism can be effectively modeled in animals to advance our understanding of cause and enable translation of basic scientific discovery into medical breakthroughs that improve the quality of life for individuals on the spectrum," says Robert Ring, Ph.D., Vice President of Translational Research at Autism Speaks. "These new autism-relevant rat models have already demonstrated great potential for the field. Our new agreement ensures that additional models will continue to be developed and made available to accelerate progress along the entire translational research continuum, from academia to the pharmaceutical industry."

"Modeling human conditions in rats, rather than the mice that have come to predominate preclinical studies, enables more predictive studies of complex neurobehavioral conditions. Rats are unique in that they exhibit richer, more human-like social behaviors than mice, juvenile play being one example. The more complex neural circuitry and greater cognitive capacity in rats also enables researchers to complete many of the demandingand crucially informativecognitive tests that mice cannot perform. In addition, on a practical level, performing initial studies in rats also provides a direct path for drug development," says Edward Weinstein, Ph.D., Director of SAGE Labs.

Initial behavioral studies of the gene knockout rats generated by SAGE Labs are being conducted by Richard E. Paylor, Ph.D., Professor at the Baylor College of Medicine. In some cases, behaviors observed in the rat models have differed from existing mouse models. For example, whereas FMR1 knockout mice exhibit elevated social interactions, rats lacking the same gene participate much less in social play and emit fewer ultrasonic squeaks during play sessions than control rats. These types of social impairments, such as reduced verbal and interactive play, more closely parallel social behavior symptoms seen in humans with FMR1 mutations. Rat models lacking functional NLGN3 and FMR1 genes also display other unexpected characteristics, including compulsive chewing on water bottles and wood blocks. Compulsive and repetitive behaviors are core symptoms in individuals with autism spectrum disorders.

"At SAGE Labs we use CompoZr Zinc Finger Nuclease technology to perform targeted genetic modifications in species previously not amenable to such modifications be it gene knockout, transgene insertion, point mutations, or conditional gene knockout. We can help researchers and pharmaceutical companies access rats, rabbits and other species that best model a medical condition of interest and provide a direct path for preclinical efficacy and toxicology testing," says Weinstein.

Currently SAGE Labs publicly provides two rat lines with knockouts of autism-associated FMR1 and NLGN3 genes. The remaining five gene knockout rat lines developed in the original collaborationfor the genes MECP2, NRXN1, CACNA1C, PTEN, and MGLUR5are expected to be released soon. The CNTNAP2 and MET knockout rat lines to be generated in the expanded collaboration are expected to be available in 2013.

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Autism Speaks and SAGE® Labs develop rat models for translational autism research

ST. LOUIS, August 2, 2012 /PRNewswire/ -- Sigma-Aldrich Corporation (SIAL) today announced that Sigma Advanced Genetic Engineering (SAGE) Labs, an initiative of Sigma Life Science and Autism Speaks, the nation's largest autism science and advocacy organization, expanded a collaboration to develop the first rat models with modified autism associated genes, intended to accelerate discovery and translational autism research.

Expansion of the collaboration follows initial behavioral studies demonstrating that the first two publicly available gene knockout rats, part of the seven rats generated through the collaboration to date, exhibit hallmark characteristics of autism, such as social deficits and repetitive behaviors. Many behavioral characteristics of autism observed in these rats are not seen in other animal models currently used for autism research. SAGE Labs and Autism Speaks now plan to generate additional genetically modified rat models of key autism-associated genes, including CNTNAP2 and MET.

"Autism spectrum disorders are a complex condition with significant unmet medical needs. Although uniquely human, fundamental aspects of the biology underlying autism can be effectively modeled in animals to advance our understanding of cause and enable translation of basic scientific discovery into medical breakthroughs that improve the quality of life for individuals on the spectrum," says Robert Ring, Ph.D., Vice President of Translational Research at Autism Speaks. "These new autism-relevant rat models have already demonstrated great potential for the field. Our new agreement ensures that additional models will continue to be developed and made available to accelerate progress along the entire translational research continuum, from academia to the pharmaceutical industry."

"Modeling human conditions in rats, rather than the mice that have come to predominate preclinical studies, enables more predictive studies of complex neurobehavioral conditions. Rats are unique in that they exhibit richer, more human-like social behaviors than mice, juvenile play being one example. The more complex neural circuitry and greater cognitive capacity in rats also enables researchers to complete many of the demandingand crucially informativecognitive tests that mice cannot perform. In addition, on a practical level, performing initial studies in rats also provides a direct path for drug development," says Edward Weinstein, Ph.D., Director of SAGE Labs.

Initial behavioral studies of the gene knockout rats generated by SAGE Labs are being conducted by Richard E. Paylor, Ph.D., Professor at the Baylor College of Medicine. In some cases, behaviors observed in the rat models have differed from existing mouse models. For example, whereas FMR1 knockout mice exhibit elevated social interactions, rats lacking the same gene participate much less in social play and emit fewer ultrasonic squeaks during play sessions than control rats. These types of social impairments, such as reduced verbal and interactive play, more closely parallel social behavior symptoms seen in humans with FMR1 mutations. Rat models lacking functional NLGN3 and FMR1 genes also display other unexpected characteristics, including compulsive chewing on water bottles and wood blocks. Compulsive and repetitive behaviors are core symptoms in individuals with autism spectrum disorders.

"At SAGE Labs we use CompoZr Zinc Finger Nuclease technology to perform targeted genetic modifications in species previously not amenable to such modifications be it gene knockout, transgene insertion, point mutations, or conditional gene knockout. We can help researchers and pharmaceutical companies access rats, rabbits and other species that best model a medical condition of interest and provide a direct path for preclinical efficacy and toxicology testing," says Weinstein.

Currently SAGE Labs publicly provides two rat lines with knockouts of autism-associated FMR1 and NLGN3 genes. The remaining five gene knockout rat lines developed in the original collaborationfor the genes MECP2, NRXN1, CACNA1C, PTEN, and MGLUR5are expected to be released soon. The CNTNAP2 and MET knockout rat lines to be generated in the expanded collaboration are expected to be available in 2013.

In a separate collaboration with The Michael J. Fox Foundation, SAGE Labs created the first animal models of Parkinson's disease that display deficits in movement similar to those developed by humans. Other genetically modified research models created by SAGE Labs include rats for Alzheimer's, schizophrenia, cancer, and cardiovascular disease research, as well as rats for toxicology testing in drug development. SAGE Labs' model generation services are available for rats, rabbits, mice and other organisms.

For more information, visit http://www.sageresearchmodels.com.

Cautionary Statement: The foregoing release contains forward-looking statements that can be identified by terminology such as "enable," "initial data demonstrates," "predictive," "encourage" or similar expressions, or by expressed or implied discussions regarding potential future revenues from products derived there from. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that gene knockout rat models of autism-associated genes or related services will assist the Company to achieve any particular levels of revenue in the future. In particular, management's expectations regarding products associated with gene knockout rat models of autism-associated genes or related services could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; the Company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; the impact that the foregoing factors could have on the values attributed to the Company's assets and liabilities as recorded in its consolidated balance sheet, and other risks and factors referred to in Sigma-Aldrich's current Form 10-K on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Sigma-Aldrich is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

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SAGE® Labs and Autism Speaks Expand Collaboration to Develop Rat Models for Translational Autism Research

HOLLISTON, Mass., Aug. 2, 2012 (GLOBE NEWSWIRE) -- Harvard Bioscience, Inc. (HBIO), a global developer, manufacturer, and marketer of a broad range of tools to advance life science research and regenerative medicine, today reported unaudited financial highlights for the three and six months ended June 30, 2012.

Second Quarter Reported Results

Revenues for the three months ended June 30, 2012 were $28.5 million, an increase of $1.4 million, or 5.0% compared to revenues of $27.1 million for the three months ended June 30, 2011. Currency exchange rates had a negative 2.1% effect on revenues in the second quarter of 2012 compared with the second quarter of 2011. Our acquisitions of CMA Microdialysis AB ("CMA") in July 2011, AHN Biotechnologie GmbH ("AHN") in February 2012 and Modular SFC, Inc. ("Modular") in May 2012 had a positive 4.9% effect on revenues. Excluding the effects of currency changes and acquisitions, our organic revenue growth for the second quarter of 2012 was 2.2% over the same period in the previous year.

Net income, as measured under U.S. generally accepted accounting principles ("GAAP"), was $0.8 million, or $0.03 per diluted share for the three months ended June 30, 2012 compared to $1.4 million, or $0.05 per diluted share, for the same period in 2011. The unfavorable year-to-year quarterly GAAP earnings comparison was primarily due to increased spending in our development-stage Regenerative Medicine Device ("RMD") business.

On a non-GAAP adjusted basis, earnings per share for our core Life Science Research Tools ("LSRT") business for the second quarter of 2012 and 2011 were $0.10 per diluted share. Non-GAAP adjusted earnings per share for our RMD business for the second quarter of 2012 was a loss of $0.04 per diluted share, compared with a loss of $0.01 per diluted share for the second quarter of 2011, and reflected greater activities in developing this initiative. Our total non-GAAP adjusted earnings per share, reflecting LSRT and RMD combined, was $0.06 per diluted share for the second quarter of 2012 compared with $0.08 per diluted share for the second quarter of 2011.

Year to Date Reported Results

Revenues for the six months ended June 30, 2012 were $56.8 million, an increase of $3.4 million, or 6.3% compared to revenues of $53.5 million for the six months ended June 30, 2011. Currency exchange rates had a negative 1.6% effect on revenues in the first half of 2012 compared with the same period in the previous year. Our acquisitions of CMA in July 2011, AHN in February 2012 and Modular in May 2012 had a positive 4.7% effect on revenues. Excluding the effects of currency changes and acquisitions, our organic revenue growth for the first half of 2012 was 3.2% over the same period in the previous year.

Net income, as measured under GAAP, was $1.3 million, or $0.04 per diluted share for the six months ended June 30, 2012 compared to $3.0 million, or $0.10 per diluted share, for the same period in 2011. The unfavorable year-to-year quarterly GAAP earnings comparison was primarily due to increased spending in our development-stage RMD business.

On a non-GAAP adjusted basis, earnings per share for our core LSRT business for the six months ended June 30, 2012 was $0.20 per diluted share, compared with $0.18 per diluted share for the same period in 2011. Non-GAAP adjusted earnings per share for our RMD business for the six months ended June 30, 2012 was a loss of $0.07 per diluted share, compared with a loss of $0.03 per diluted share for the same period in 2011, and reflected greater activities in developing this initiative. Our total non-GAAP adjusted earnings per share, reflecting LSRT and RMD combined, were $0.13 per diluted share for six months ended June 30, 2012 compared with $0.16 per diluted share for the same period in 2011.

Commenting on the Company's performance, Chane Graziano, CEO, stated, "Despite a relatively soft global economy we ended the second quarter 2012 in our core LSRT business with revenues of $28.5 million and non-GAAP adjusted diluted earnings per share of 10 cents in line with our expectations and our guidance for the quarter. We also continued to show improvements in our gross profit margins primarily driven by the introduction of new pumps at Harvard Apparatus, a new spectrophotometer at Biochrom and operational improvements at Hoefer, Coulbourn and Panlab."

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HBIO Reports Second Quarter 2012 Revenue Growth of 5% Over Second Quarter 2011

ScienceDaily (Aug. 1, 2012) Researchers at the National Institutes of Health have found evidence that a unique type of immune cell contributes to multiple sclerosis (MS). Their discovery helps define the effects of one of the newest drugs under investigation for treating MS -- daclizumab -- and could lead to a new class of drugs for treating MS and other autoimmune disorders.

In these disorders, the immune system turns against the body's own tissues. Ongoing clinical trials have shown that daclizumab appears to help quiet the autoimmune response in MS patients, but its precise effects on the legions of cells that make up the immune system are not fully understood.

The new study, published in Science Translational Medicine, shows that one effect of daclizumab is to thin the ranks of lymphoid tissue inducer (LTi) cells. These cells are known to promote the development of lymph nodes and related tissues during fetal life, but their role during adulthood has been unclear. The new study marks the first time that LTi cells have been implicated in any human autoimmune disorder.

"While further study is required to confirm the role of LTi cells in autoimmunity, our results point to the cells as a promising target for the development of new drugs to treat autoimmune disorders," said Bibiana Bielekova, M.D., an investigator at NIH's National Institute of Neurological Disorders and Stroke (NINDS).

Dr. Bielekova and her team found that among MS patients participating in clinical trials of daclizumab, the number of LTi cells was elevated in patients not receiving daclizumab compared to those on the drug. Patients receiving daclizumab also had reduced signs of inflammation in the cerebrospinal fluid (CSF) that surrounds the brain. And the researchers found that daclizumab appears to steer the body away from producing LTi cells, in favor of another cell type that counteracts autoimmunity.

In MS, the immune system attacks myelin, a material that insulates nerve fibers running throughout the brain and spinal cord. This typically leads to vision loss, and other sensory changes such as numbness and tingling, weakness, and fatigue. The disorder affects approximately 400,000 people in the United States. In about 85 percent of patients, MS starts as a relapsing-remitting form, in which symptoms come and go. Many patients eventually develop secondary progressive MS, in which symptom flare-ups are followed by worsening disability. Many medications are available to decrease the number of flare-ups, but no medication is effective at slowing the course of progressive MS.

The newer, sophisticated drugs for relapsing-remitting MS target key cells and molecules responsible for triggering and maintaining autoimmunity. Cytotoxic T cells, the immune system's specialized mobile infantry, are known to lead the attack. Antibodies, the immune system's guided missiles, appear to help reinforce it.

Daclizumab is a lab-engineered antibody, or monoclonal antibody, that alters signaling by interleukin-2 (IL-2), a key factor that mobilizes T cells. In a large clinical trial (NCT00109161), it has shown promise as an add-on therapy for patients taking the approved MS drug interferon-beta. Another ongoing trial (NCT00390221) is investigating whether or not daclizumab is effective as a stand-alone therapy for reducing relapses in MS.

The drug was designed to suppress T cell responses to IL-2, and it does so -- but Dr. Bielekova had found previously that this suppression is indirect and depends on other immune cells. For example, one effect of daclizumab is to stimulate the non-specialized counterparts of T cells, called natural killer cells. These cells in turn suppress T cell activity.

In their new study, Dr. Bielekova and her team discovered that daclizumab's stimulatory effect on natural killer cells is paired with an inhibitory effect on LTi cells. They found evidence that the drug, via its effects on IL-2 signaling, acts on a type of stem cell. The drug appears to decrease the likelihood that this stem cell will develop into LTi cells, and sway it toward becoming natural killer cells.

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Unique cell type in implicated in multiple sclerosis

ScienceDaily (Aug. 1, 2012) Worldwide cabbage farmers have vast problems with the diamond-back moth. It lays its eggs on the cabbage plants and the voracious appetite of the larvae ruins the yield. However, Morten Emil Mldrup from the University of Copenhagen has developed a method to deceive the greedy insects. Mldrup presents his spectacular research results at a public PhD defense on August 3.

"We have discovered a way to cheat the diamond-back moths to lay their eggs on tobacco plants. As their larvae cannot survive on tobacco leaves they will soon starve to death. In the mean time you can cultivate your cabbage at peace," explains MSc in Biology and Biotechnology Morten Emil Mldrup from DynaMo, Center for Dynamic Molecular Interactions, University of Copenhagen.

It sounds like an imaginative scenario too good to be true. None the less Morten Emil Mldrup and his colleagues from DynaMo at University of Copenhagen have shown that it is indeed possible 'to cheat' the greedy little insects in exactly this way. Morten Emil Mldrup has studied the defence compounds of the cabbage family, the so called glucosinolates, exhaustively. Glucosinolates are toxic to cabbage pests in general, the diamond-back moth being one of very few exemptions.

Away with pesticides

The odour of the cabbage defense compounds attracts the pregnant diamond-back moths. To them the 'defence odour' is a signal of an ideal place to lay their eggs. In this way they ensure their larvae plenty of food without competition from others. After having thoroughly established how a cabbage plants produces defence compounds, Morten Emil Mldrup and his colleagues have successfully transferred the genes responsible for the production of glucosinolates from cabbage into tobacco plants.

"Our experiments show that it is indeed possible to fool the diamond-back moth to lay its eggs on tobacco plants. This is fantastic because the larvae are a major problem all over the world. At present we are aiming at making glucosinolate producing potato plants. The goal is to avoid diamond-back moths' larvae in cabbage by cultivating potato and cabbage plants together. In this way a lot of money is to be saved, and in addition the growers do not need to use the big amounts of pesticides commonly used today. In this way one may say that our discovery is also of benefit to nature," Morten Emil Mldrup tells.

Defense against attacks

Morten Emil Mldrup researches the bioactive molecules that plants are using to protect themselves against pests and how the plants produce these natural defence compounds.

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Gene technology helps deceive greedy pest insects

ScienceDaily (July 31, 2012) Researchers at the University of Iowa Carver College of Medicine have discovered a genetic process that can restore function to a defective protein, which is the most common cause of cystic fibrosis (CF).

Cystic fibrosis is an inherited disease caused by mutations in a gene that adversely affect its protein product. In its correct form and cellular location, this protein, cystic fibrosis transmembrane conductance regulator (CFTR), functions as a channel for ions to move across cell membranes, and is critical for maintaining cellular salt and water balance.

The most common CF-causing genetic mutation, known as delta F508, disrupts the process whereby the CFTR protein is folded into its correct shape and shipped to the membranes of cells that line the airways and other organs. Most of the defective CFTR protein is misprocessed and gets degraded. The lack of normal CFTR ion channels leads to numerous problems, including lung infection and inflammation, the major causes of disease and death in cystic fibrosis.

Despite its importance, how the CFTR protein is made and delivered to cell membranes in its functioning form is not well understood. The UI team led by Paul McCray, M.D., professor of pediatrics and microbiology with UI Health Care and the Roy J. Carver Chair in Pulmonary Research and Vice Chair for Research in Pediatrics, investigated the role of microRNAs -- small non-coding stretches of RNA -- in regulating expression of CFTR.

In their research, McCray and colleagues discovered that one particular microRNA, called miR-138, helps control the biosynthesis of CFTR by regulating a network of genes involved in the production and processing of the protein. The study, published online the week of July 30 in the Proceedings of the National Academy of Sciences (PNAS) Early Edition, shows that that miR-138 acts on the other genes to orchestrate a cellular program that increases production of CFTR and increases the amount of the protein that is transported to the cell membrane where it functions as an ion channel.

"We first wanted to determine how this gene network impacts the CFTR protein produced in people who don't have cystic fibrosis," says lead author Shyam Ramachandran, Ph.D.. "We identified a novel regulatory circuit, but then asked ourselves if any of this affected the mutant protein."

Surprisingly, the researchers found that when the gene network was activated by miR-138, it not only increased the amount of the mutated protein, but also partially restored the protein's function.

By manipulating the microRNA network, the UI team was able to change the fate of the misfolded CFTR from being degraded in the cell to functioning as an ion channel in the cell membrane.

"This was a very surprising finding," Ramachandran says. "It unexpectedly helps rescue the function of the mutant protein."

Because most people with CF have one or two copies of the delta F508 mutation, interventions that overcome the CFTR protein-processing problems caused by this mutation might have important implications for new ways of treating CF.

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Gene network restores cystic fibrosis protein function

Public release date: 31-Jul-2012 [ | E-mail | Share ]

Contact: Molly Rossiter molly-rossiter@uiowa.edu 319-356-7127 University of Iowa Health Care

Researchers at the University of Iowa Carver College of Medicine have discovered a genetic process that can restore function to a defective protein, which is the most common cause of cystic fibrosis (CF).

Cystic fibrosis is an inherited disease caused by mutations in a gene that adversely affect its protein product. In its correct form and cellular location, this protein, cystic fibrosis transmembrane conductance regulator (CFTR), functions as a channel for ions to move across cell membranes, and is critical for maintaining cellular salt and water balance.

The most common CF-causing genetic mutation, known as delta F508, disrupts the process whereby the CFTR protein is folded into its correct shape and shipped to the membranes of cells that line the airways and other organs. Most of the defective CFTR protein is misprocessed and gets degraded. The lack of normal CFTR ion channels leads to numerous problems, including lung infection and inflammation, the major causes of disease and death in cystic fibrosis.

Despite its importance, how the CFTR protein is made and delivered to cell membranes in its functioning form is not well understood. The UI team led by Paul McCray, M.D., professor of pediatrics and microbiology with UI Health Care and the Roy J. Carver Chair in Pulmonary Research and Vice Chair for Research in Pediatrics, investigated the role of microRNAs -- small non-coding stretches of RNA -- in regulating expression of CFTR.

In their research, McCray and colleagues discovered that one particular microRNA, called miR-138, helps control the biosynthesis of CFTR by regulating a network of genes involved in the production and processing of the protein. The study, published online the week of July 30 in the Proceedings of the National Academy of Sciences (PNAS) Early Edition, shows that that miR-138 acts on the other genes to orchestrate a cellular program that increases production of CFTR and increases the amount of the protein that is transported to the cell membrane where it functions as an ion channel.

"We first wanted to determine how this gene network impacts the CFTR protein produced in people who don't have cystic fibrosis," says lead author Shyam Ramachandran, Ph.D.. "We identified a novel regulatory circuit, but then asked ourselves if any of this affected the mutant protein."

Surprisingly, the researchers found that when the gene network was activated by miR-138, it not only increased the amount of the mutated protein, but also partially restored the protein's function.

By manipulating the microRNA network, the UI team was able to change the fate of the misfolded CFTR from being degraded in the cell to functioning as an ion channel in the cell membrane.

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Gene network restores CF protein function

August 1, 2012

Connie K. Ho for redOrbit.com Your Universe Online

23andMe, a company focused on personal genetics, recently took the first step in working towards Food and Drug Administration (FDA) clearance; the FDA plans to review the paperwork over the next few months.

Previously, the Silicon Valley company refrained from government regulation, claiming that the service provided consumers with information rather than a medical service. CBS News reported that 23andMe submitted the first group of seven health-related tests for the FDA to review. By the end of next year, the organization will have submitted another 100 tests to the agency. With these exams, the organization hopes to obtain government approval and scientific credibility.

23andMe has pioneered the direct-to-consumer genetic testing industry and we are committed to helping individuals understand their own genetic information through proven DNA analysis technologies and web-based interactive tools, commented Anne Wojcicki, 23andMe CEO and Co-Founder, in a prepared statement. 23andMe is working proactively with the FDA to ensure the industry delivers high quality information that consumers can trust.

23andMes Personal Genome Service allows individuals to learn more about their personal DNA, offering over 200 health and trait reports along with information on genetic ancestry. The company believes that the service helps scientists better understand how genetics plays into health and diseases. The test makers looked at genetic code, which could possibly help doctors determine if patients have treatable health problems. For individuals, the service will help them make decisions with their healthcare provider.

I think weve now entered an era where these direct-to-consumer offerings are beginning to have real medical relevance, and therefore I am in favor of them being done within some regulatory context, said Dr. James Evans, a professor of genetics and medicine at the University of North Carolina Medical School, told National Public Radio (NPR).

The DNA samples are processed by a CLIA-certified laboratory and 23andMe hopes that the filing with the FDA will make their product fluid and transparent for the public.

23andMe has always valued the guidance of the FDA and, in fact, engaged the agency in conversations prior to launching the Personal Genome Service in 2007. Our ongoing conversations with the FDA in the last year, in particular, resulted in a focused approach that resulted in our ability to compile a comprehensive analysis of 23andMes direct-to-consumer testing for FDA consideration, noted Ashley Gould, 23andMe VP Corporate Development and Chief Legal Officer, in the statement.

According to NPR, if approved, the personalized DNA test by the genetic test maker could possibly increase acceptance of a technology that is questionable to various scientists. The saliva-based kits do not completely help explain the biology of DNA variations related to certain disease. A bulk of geneticists believes that the tests are not reliable.

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23andMe Genetic Testing Company Applies For FDA Approval

ALACHUA, Fla.--(BUSINESS WIRE)--

AxoGen, Inc. (AXGN) a leading regenerative medicine company focused on the commercialization of proprietary products and technologies for peripheral nerve reconstruction and regeneration, today announced that it will release its financial results for the period ended June 30, 2012, after the close of U.S. financial markets on August 14, 2012. An accompanying conference call hosted by Karen Zaderej, Chief Executive Officer, and Greg Freitag, Chief Financial Officer, to discuss results will be held at 11:00 a.m. ET, on August 15, 2012.

A webcast replay of the conference call will be available on the company's website, http://www.axogeninc.com, under Investors.

About AxoGen, Inc.

AxoGen (AXGN) is a leading regenerative medicine company with a portfolio of proprietary products and technologies for peripheral nerve reconstruction and regeneration. Every day, people suffer traumatic injuries or undergo surgical procedures that impact the function of their peripheral nerves. Peripheral nerves provide the pathways for both motor and sensory signals throughout the body and their damage can result in the loss of function and feeling. In order to improve surgical reconstruction and regeneration of peripheral nerves, AxoGen has developed and licensed patented and patent-pending technologies, which are used in its portfolio of products. This portfolio includes Avance Nerve Graft, a commercially available allograft nerve for bridging nerve discontinuities (a gap created when the nerve is severed) of 5mm to 70mm in length. Avance Nerve Graft is sterilized and processed using a patented cleaning process that preserves the inherent and relevant structural characteristics of the tissue, allowing regenerating axons to grow through the scaffold to the motor or sensory organ.

AxoGens portfolio also includes AxoGuard Nerve Connector, a coaptation aid allowing for close approximation of severed nerves, and AxoGuard Nerve Protector, a bioscaffold used to reinforce a coaptation site, wrap a partially severed nerve or isolate and protect nerve tissue.

AxoGen is bringing the science of nerve repair to life with thousands of surgical implants of AxoGen products performed in hospitals and surgery centers across the United States, including military hospitals serving U.S. service men and women.

AxoGen is the parent of its wholly owned operating subsidiary, AxoGen Corporation. AxoGens principal executive office and operations are located in Alachua, FL.

To receive email alerts directly from AxoGen, please click here http://www.axogeninc.com/emailalerts.html.

Cautionary Statements Concerning Forward-Looking Statements

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AxoGen, Inc. Announces Release Date of 2012 Second Quarter Results and Conference Call

Newswise Researchers at Case Western Reserve University School of Medicine have discovered a mutant form of the gene, Chk1, that when expressed in cancer cells, permanently stopped their proliferation and caused cell death without the addition of any chemotherapeutic drugs. This study illustrates an unprecedented finding, that artificially activating Chk1 alone is sufficient to kill cancer cells.

We have identified a new direction for cancer therapy and the new direction is leading us to a reduction in toxicity in cancer therapy, compared with chemotherapy or radiation therapy, said Dr. Zhang, assistant professor, Department of Pharmacology at the School of Medicine, and member of the universitys Case Comprehensive Cancer Center. With this discovery, scientists could stop the proliferation of cancer cells, allowing physicians time to fix cells and genetic errors.

While studying the basic mechanisms for genome integrity, Dr. Zhangs team unexpectedly discovered an active mutant form of human Chk1, which is also a non-natural form of this gene. This mutation changed the protein conformation of Chk1 from the inactive form into an active form. Remarkably, the research team discovered that when expressed in cancer cells, this active mutant form of Chk1 permanently stopped cancer cell proliferation and caused cell death in petri dishes even without the addition of any chemotherapeutic drugs.

The biggest advantage of this potential strategy is that no toxic chemotherapeutic drug is needed to achieve the same cancer killing effect used with a combination of Chk1 inhibitors and chemotherapeutic drugs.

Cells respond to DNA damage by activating networks of signaling pathways, termed cell cycle checkpoints. Central to these genome pathways is the protein kinase, called Chk1. Chk1 facilitates cell survival, including cancer cells, under stressful conditions, such as those induced by chemotherapeutic agents, by placing a temporary stop on the cell cycle progression and coordinating repair programs to fix the DNA errors.

It has long been suggested that combining Chk1 inhibition with chemotherapy or radiotherapy should significantly enhance the anticancer effect of these therapies. This idea has serves as the basis for multiple pharmaceutical companies searching for potential Chk1 inhibitors that can effectively combine with chemotherapy in cancer therapy. To date, no Chk1 inhibitor has passed the clinical trial stage III . This led Dr. Zhangs team to look for alternative strategies for targeting Chk1 in cancer therapy.

Future research by Dr. Zhang and his team will consider two possible approaches to artificially activating Chk1 in cancer cells. One possibility is to use the gene therapy concept to deliver the active mutant form of Chk1 that the team discovered, into cancer cells. The other is to search for small molecules that can induce the same conformational change of Chk1, so that they can be delivered into cancer cells to activate Chk1 molecules. The consequence of either would be permanent cell proliferation inhibition and cancer.

All three authors of this study, Jingna Wang, Xiangzi Han and Youwei Zhang hold the title of Ph.D. and are members of the Department of Pharmacology, Case Western Reserve University School of Medicine, as well as members of the universitys Case Comprehensive Cancer Center. Dr. Wang and Dr. Han are postdoctoral fellows. Dr. Zhang is an assistant professor.

This study is published in Cancer Research. Support for the study comes from the National Cancer Institute at the National Institute of Health,

Grants that supported this study are NCI R00CA126173 and R01CA163214.

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Researchers Discover Gene that Permanently Stops Cancer Cell Proliferation

Public release date: 1-Aug-2012 [ | E-mail | Share ]

Contact: Christine A. Somosi 216-368-6287 Case Western Reserve University

Researchers at Case Western Reserve University School of Medicine have discovered a mutant form of the gene, Chk1, that when expressed in cancer cells, permanently stopped their proliferation and caused cell death without the addition of any chemotherapeutic drugs. This study illustrates an unprecedented finding, that artificially activating Chk1 alone is sufficient to kill cancer cells.

"We have identified a new direction for cancer therapy and the new direction is leading us to a reduction in toxicity in cancer therapy, compared with chemotherapy or radiation therapy," said Dr. Zhang, assistant professor, Department of Pharmacology at the School of Medicine, and member of the university's Case Comprehensive Cancer Center. "With this discovery, scientists could stop the proliferation of cancer cells, allowing physicians time to fix cells and genetic errors."

While studying the basic mechanisms for genome integrity, Dr. Zhang's team unexpectedly discovered an active mutant form of human Chk1, which is also a non-natural form of this gene. This mutation changed the protein conformation of Chk1 from the inactive form into an active form. Remarkably, the research team discovered that when expressed in cancer cells, this active mutant form of Chk1 permanently stopped cancer cell proliferation and caused cell death in petri dishes even without the addition of any chemotherapeutic drugs.

The biggest advantage of this potential strategy is that no toxic chemotherapeutic drug is needed to achieve the same cancer killing effect used with a combination of Chk1 inhibitors and chemotherapeutic drugs.

Cells respond to DNA damage by activating networks of signaling pathways, termed cell cycle checkpoints. Central to these genome pathways is the protein kinase, called Chk1. Chk1 facilitates cell survival, including cancer cells, under stressful conditions, such as those induced by chemotherapeutic agents, by placing a temporary stop on the cell cycle progression and coordinating repair programs to fix the DNA errors.

It has long been suggested that combining Chk1 inhibition with chemotherapy or radiotherapy should significantly enhance the anticancer effect of these therapies. This idea has serves as the basis for multiple pharmaceutical companies searching for potential Chk1 inhibitors that can effectively combine with chemotherapy in cancer therapy. To date, no Chk1 inhibitor has passed the clinical trial stage III . This led Dr. Zhang's team to look for alternative strategies for targeting Chk1 in cancer therapy.

Future research by Dr. Zhang and his team will consider two possible approaches to artificially activating Chk1 in cancer cells. One possibility is to use the gene therapy concept to deliver the active mutant form of Chk1 that the team discovered, into cancer cells. The other is to search for small molecules that can induce the same conformational change of Chk1, so that they can be delivered into cancer cells to activate Chk1 molecules. The consequence of either would be permanent cell proliferation inhibition and cancer.

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CWRU School of Medicine researchers discover gene that permanently stops cancer cell proliferation

LONDON--(BUSINESS WIRE)--

Despite the fact that China lags behind in terms of the overall level of genetic engineering drugs, the industry has accumulated rich R&D and industrialization experience as well as capital reserves. Thus, with a host of genetic engineering drug patents to become due, Chinese enterprises, such as Walvax, are committed to the industrialization research of monoclonal antibody, long-acting recombinant protein drugs and other generic drugs with high technical barriers. In April this year, Walvax announced to invest in Shanghai Fengmao to develop and produce genetic engineering generic drugs such as rituximab, bevacizumab, adalimumab, panitumumab, denosumab and long-acting EPO.

New market report China Genetic Engineering Drug Industry Report, 2011-2012 worked out by ResearchInChina delves into a comprehensive discussion of the genetic engineering drug industry from a global perspective and focuses on an insightful review of the sector in China.

Key Topics Covered:

Companies profiled in the report include: Shanghai Lansheng Guojian Pharmaceutical Co., Ltd; Biotech Pharmaceutical Co., Ltd; Anhui Anke Biotechnology (Group) Co., Ltd; GeneScience Pharmaceuticals Co., Ltd; Beijing SL Pharmaceutical Co., Ltd; Jiangsu Sihuan Bioengineering Co., Ltd; Shenzhen Neptunus Interlong Bio-Technique Co., Ltd; 3SBio Inc.; and Tonghua Dongbao Pharmaceutical Co., Ltd.

Report Details:

Title: China Genetic Engineering Drug Industry Report, 2011-2012

Published: July, 2012

Pages: 80

Price: US$ 1,900.00

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Genetic Engineering Drug Sector Globally & in China Examined in New Report Published at MarketPublishers.com

Public release date: 31-Jul-2012 [ | E-mail | Share ]

Contact: Jessica Maki jmaki3@partners.org 617-534-1603 JAMA and Archives Journals

CHICAGO Among patients with multiple colorectal polyps, the prevalence of certain gene mutations varied considerably by polyp count, according to a study in the August 1 issue of JAMA.

"Patients with multiple colorectal adenomas [polyps] may carry germline [those cells of an individual that have genetic material that could be passed to offspring] mutations in the APC or MUTYH genes," according to background information in the article. The authors write that guidelines for when genetic evaluation should be performed in individuals with multiple colorectal adenomas vary, and data to support such guidelines are limited.

Shilpa Grover, M.D., M.P.H., of Brigham and Women's Hospital, Boston, and colleagues conducted a study to evaluate the frequency of APC and MUTYH mutations by the number of colorectal adenomas among individuals who had undergone clinical genetic testing. The researchers also studied the relationship between the number of adenomas and age at diagnosis of adenoma and colorectal cancer and the prevalence of pathogenic APC or MUTYH mutations. The study included 8,676 individuals who had undergone full gene sequencing between 2004 and 2011. Individuals with a certain mutation of the MUTYH gene (Y179C and G396D) underwent full MUTYH gene sequencing. APC and MUTYH mutation prevalence was evaluated by the number of polyps.

Colorectal adenomas were reported in 7,225 individuals; 1,457 with classic polyposis (100 adenomas or more) and 3,253 with attenuated (diminished) polyposis (20-99 adenomas). "The prevalence of pathogenic APC and biallelic [pertaining to both alleles (both alternative forms of a gene)] MUTYH mutations was 95 of 119 (80 percent) and 2 of 119 (2 percent), respectively, among individuals with 1,000 or more adenomas, 756 of 1,338 (56 percent) and 94 of 1,338 (7 percent) among those with 100 to 999 adenomas, 326 of 3,253 (10 percent) and 233 of 3,253 (7 percent) among those with 20 to 99 adenomas, and 50 of 970 (5 percent) and 37 of 970 (4 percent) among those with 10 to 19 adenomas. Adenoma count was strongly associated with a pathogenic mutation in multivariable analyses," the authors write.

The researchers note that their evaluation of individuals who underwent genetic testing because of a personal or family history suggestive of a familial polyposis syndrome suggests that genetic evaluation for APC and MUTYH mutations may be considered in individuals with 10 or more adenomas. "However, our results are derived from a selected cohort of high-risk individuals and need to be validated in larger populations of unselected patients."

"The mutation probabilities reported here may assist clinicians in their decision to recommend genetic evaluation and counsel patients undergoing genetic testing. However, it remains important to also consider the limitations of genetic testing at present, because one-third of patients with a classic familial adenomatous polyposis [FAP; a polyposis syndrome resulting from mutations in the APC gene characterized by multiple colorectal polyps] phenotype are found to not carry a mutation in either the APC or MUTYH gene. Such individuals should undergo periodic re-evaluation as other susceptibility genes are identified."

(JAMA. 2012;308[5]:485-492. Available pre-embargo to the media at http://media.jamanetwork.com)

Editor's Note: This study was supported by National Cancer Institute grants and by a National Institutes of Health grant. Please see the article for additional information, including other authors, author contributions and affiliations, financial disclosures, etc.

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Study finds correlation between number of colorectal polyps and genetic mutations

ACMG Foundation for Genetic and Genomic Medicine Announces Two 2013-2014 ACMG Foundation/Genzyme Biochemical Genetics Fellowships Now Available -- Deadline August 15, 2012

Newswise BETHESDA, Md., July 31, 2012 /PRNewswire-USNewswire/ -- The ACMG Foundation for Genetic and Genomic Medicine announced that applications for the prestigious ACMG Foundation/Genzyme Clinical Genetics Fellowship in Biochemical Genetics award Program are now being accepted. For 2012-2013, two awardees will be given the opportunity to participate in an in-depth clinical experience at a premier medical center with expertise and significant clinical volume in the area of biochemical genetics. The Fellowship comes with a $75,000 award.

"Advances in newborn screening and in therapeutics offer unprecedented opportunities for early diagnosis and treatment of inborn errors of metabolism, including lysosomal storage disorders," says Bruce Korf MD, PhD, FACMG, President of the ACMG Foundation. "Though we are at the forefront in the integration of genetics and genomics into medical practice, with this growth there is a deficit of physicians specifically trained in the diagnosis, management and treatment of individuals with metabolic diseases. Genzyme and the ACMG Foundation have created a robust national fellowship program to promote and encourage the recruitment and training of clinicians for this purpose."

The goals of the ACMG Foundation/Genzyme Clinical Genetics Fellowship in Biochemical Genetics award Program are to:

1. Advance education, research and standards of practice in medical genetics. 2. Develop and expand clinical and laboratory expertise in medical genetics in the United States. 3. Initiate and develop a broad-based infrastructure for industry funding of high quality projects in the field of medical genetics.

Applications for this program will be considered in one round, based on the following schedule:

* Application Submission Deadline: August 15, 2012 * Awardee Notification by: September 2012 * Award Presentation: at the 2013 ACMG Annual Clinical Genetics Meeting in Phoenix, AZ

For more information and to download the application please visit http://www.acmgfoundation.org or email cpowell@acmgfoundation.org.

"Since 2005, the ACMG Foundation for Genetic and Genomic Medicine has coordinated this outstanding opportunity for geneticists, and we have had many out-standing applicants and fellows. We appreciate Genzyme's financial support of the fellowships, and I feel that they are an excellent example of a successful partnership between industry and a nonprofit organization to help train the next generation of geneticists," said David Cotter, ACMG Foundation Director of Development.

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2013-2014 ACMG Foundation/Genzyme Biochemical Genetics Fellowships

On Tuesday, the FDA granted approval for a first-ever human trial of cellular transplant to cure paralysis

Andrew H. Walker / Getty Images for The Buoniconti Fund

Marc Buoniconti attends the 26th Annual Great Sports Legends Dinner to benefit the Buoniconti Fund To Cure Paralysis at The Waldorf-Astoria on September 26, 2011 in New York City.

In the early winter of 1988, I traveled to Miami to visit Marc Buoniconti. He was 24 years old at the time, and in many ways looked quite fit full of energy, chattering on about his plans, exactly what youd expect from a person his age. But Buoniconti wasnt fit. He was in a wheelchair and hadnt moved a muscle below his shoulders since fracturing his spine between the 3rd and 4th cervical vertebrae in a college football game in October 1985.

By the time I met him, he had already done the grueling work of weaning himself from his respirator training new muscles and learning new techniques to breathe on his own. And that freedom allowed him to assume the job of point man for The Miami Project to Cure Paralysis, an organization co-founded by Buoniconti, his father NFL Hall of Famer Nick Buoniconti the University of Miami and a handful of local surgeons.

I was never in denial about my injury, Marc Buoniconti told me at the time. When you cant move, you move through that phase pretty fast. But the absence of denial did not mean the absence of hope. Buoniconti was adamant that he would dedicate his life to getting out of his chair and helping the 300,000 other Americans living with spinal cord injury do the same. In the meantime, hed keep himself as fit as possible. When the cure comes, he said, I plan to be ready.

Bouniconti is now a 45-year-old-man with a degree in psychology, still with the Miami Project and still in a wheelchair. But the cure he spoke of 27 years ago just got a very big step closer. On Tuesday morning, Miami Project doctors convened a press conference to announce that the U.S. Food and Drug Administration (FDA) had just granted them a green light to begin Phase 1 human trials for a new surgical technique in which nerve cells from the leg would be transplanted to the spine of newly paralyzed patients in the hope that they would grow restoring at least some function and sensation.

I am more optimistic now than I have ever been, says Buoniconti. He has reason to be.

What makes spinal cord injuries as devastating as they are is that everything about them plays out in absolutes: they are instantaneous, utterly disabling and horribly permanent. That last fact has always presented both a puzzle and an opportunity. Nerves in the peripheral nervous system (PNS) those that carry signals in an arm or leg, say are able to regenerate after injury. Thats why you can badly lacerate a finger and retain full use of it. The central nervous system (CNS) is a different matter. An injured spine remains an injured spine, period. Find and harness what it is that makes things so different in the PNS and you just might get a wounded spine to heal.

As researchers learned, there are a lot of things that drive peripheral regrowth, but perhaps the most important are known as Schwann cells, which are not nerve cells themselves, but a kind of attendant, helping neurons regrow myelin the fatty insulation that covers nerve strands and otherwise stay healthy and functioning. For some years, Miami Project scientists have been transplanting Schwann cells from the legs of paralyzed rats, mice, pigs and primates to the site of the spinal injury and have been astonished at the results: in many cases, the animals recovered 70% of the lost sensation and function. That is exactly 70% more than most spinal injured patients have been told to hope for.

Excerpt from:
A Breakthrough for Spinal Cord Injury Research?

ALACHUA, Fla.--(BUSINESS WIRE)--

AxoGen, Inc. (AXGN), a leading regenerative medicine company focused on the development and commercialization of products and technologies for peripheral nerve reconstruction and regeneration, announced today that it will hold its Annual Shareholders Meeting on Thursday August 30th, 2012. The meeting will take place at 1:00 p.m. Central Time at the following location:

Participants may also attend the meeting virtually via the Internet link http://www.virtualshareholdermeeting.com/axogen. The Meeting will include an introduction, adjournment and Company overview provided by Karen Zaderej, Chief Executive Officer of AxoGen, and formal business matters presided over by Greg Freitag, Chief Financial Officer and General Counsel.

The Proxy Statement, with the accompanying Notice of Annual Meeting, and 2011 Annual Report on Form 10K are available on the Companys website at http://www.axogeninc.com/proxyStatement.html.

Shareholders of record of the Companys common stock at the close of business on July 9, 2012 will be entitled to receive notice of and to vote at the Meeting.

About AxoGen, Inc.

AxoGen (AXGN) is a leading regenerative medicine company with a portfolio of proprietary products and technologies for peripheral nerve reconstruction and regeneration. Every day, people suffer traumatic injuries or undergo surgical procedures that impact the function of their peripheral nerves. Peripheral nerves provide the pathways for both motor and sensory signals throughout the body and their damage can result in the loss of function and feeling. In order to improve surgical reconstruction and regeneration of peripheral nerves, AxoGen has developed and licensed patented and patent-pending technologies, which are used in its portfolio of products. This portfolio includes Avance Nerve Graft, a commercially available allograft nerve for bridging nerve discontinuities (a gap created when the nerve is severed) of 5mm to 70mm in length. Avance Nerve Graft is sterilized and processed using a patented cleaning process that preserves the inherent and relevant structural characteristics of the tissue, allowing regenerating axons to grow through the scaffold to the motor or sensory organ.

AxoGens portfolio also includes AxoGuard Nerve Connector, a coaptation aid allowing for close approximation of severed nerves, and AxoGuard Nerve Protector, a bioscaffold used to reinforce a coaptation site, wrap a partially severed nerve or isolate and protect nerve tissue.

AxoGen is bringing the science of nerve repair to life with thousands of surgical implants of AxoGen products performed in hospitals and surgery centers across the United States, including military hospitals serving U.S. service men and women.

AxoGen is the parent of its wholly owned operating subsidiary, AxoGen Corporation. AxoGens principal executive office and operations are located in Alachua, FL.

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AxoGen, Inc. Announces 2012 Annual Shareholders’ Meeting