Archive for the ‘Gene Therapy Research’ Category

St. Louis /PRNewswire/ -- Sigma-Aldrich Corporation (Nasdaq: SIAL) today announced that Sigma Advanced Genetic Engineering (SAGE) Labs, an initiative of Sigma Life Science, extended CompoZr Zinc Finger Nuclease (ZFN) technology to achieve the first tissue-specific conditional knockout of an endogenous gene in rats. For two decades this approach for generating sophisticated disease models could be performed only in mice. Rats, however, are preferred by drug discovery and basic researchers because the animal's physiology, neurobiology and other features are more predictive of human conditions. Rats engineered to contain tissue-specific conditional gene knockouts are available exclusively through the SAGEspeed Custom Model Development Service. Details are available at http://www.sageresearchmodels.com/conditional-KO.

Conventional gene knockout eliminates a gene throughout an entire animal. In contrast, conditional gene knockout can eliminate a gene solely in the relevant tissue or organ, leading to a more accurate understanding of the gene's function. Conditional gene knockout can also knock out genes at certain points in development, enabling studies of genes whose absence in embryos is lethal, but whose loss of function in adulthood is critical to investigate for many human diseases.

"Almost 89% of drug candidates fail to achieve approval," said Edward Weinstein, Director of SAGE Labs. "Basic and drug discovery researchers need access to more predictive animal models whose physiology, biology, and genetics more closely reflect specific human conditions. SAGE Labs is applying ZFN technology to achieve previously impossible genetic manipulations, such as tissue-specific gene deletion in rats."

Using the conditional knockout methodology, scientists at SAGE Labs have generated a pair of rat lines in which two important neuronal genes, Crhr1 and Grin1, were removed in specific neuronal populations. Crhr1 and Grin1 have been implicated as playing a role in depression and schizophrenia, respectively. The rat lines were developed through the SAGEspeed model creation process, which uses Sigma's CompoZr ZFN technology to create sophisticated genetic modifications in rats, mice, rabbits, and other organisms. CompoZr ZFN technology is the first to enable highly efficient, targeted editing of the genome of any species.

For more information and to request pricing, visit http://www.sageresearchmodels.com.

Cautionary Statement: The foregoing release contains forward-looking statements that can be identified by terminology such as "enable," "enabling," "leading to," "achieve," "predictive" 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 iPS cells, iPS-cell derived primary cell lines, novel assays, or related custom services will assist the Company to achieve any particular levels of revenue in the future. In particular, management's expectations regarding products associated iPS cells, iPS-cell derived primary cell lines, novel assays, or related custom 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.

About Sigma Life Science: Sigma Life Science is a Sigma-Aldrich business that represents the Company's leadership in innovative biological products and services for the global life science market and offers an array of biologically-rich products and reagents that researchers use in scientific investigation. Product areas include biomolecules, genomics and functional genomics, cells and cell-based assays, transgenics, protein assays, stem cell research, epigenetics and custom services/oligonucleotides. Sigma Life Science also provides an extensive range critical bioessentials like biochemicals, antibiotics, buffers, carbohydrates, enzymes, forensic tools, hematology and histology, nucleotides, amino acids and their derivatives, and cell culture media.

About Sigma-Aldrich: Sigma-Aldrich is a leading Life Science and High Technology company whose biochemical, organic chemical products, kits and services are used in scientific research, including genomic and proteomic research, biotechnology, pharmaceutical development, the diagnosis of disease and as key components in pharmaceutical, diagnostics and high technology manufacturing. Sigma-Aldrich customers include more than 1.3 million scientists and technologists in life science companies, university and government institutions, hospitals and industry. The Company operates in 40 countries and has nearly 9,000 employees whose objective is to provide excellent service worldwide. Sigma-Aldrich is committed to accelerating customer success through innovation and leadership in Life Science and High Technology. For more information about Sigma-Aldrich, please visit its website at http://www.sigma-aldrich.com.

Sigma-Aldrich and Sigma are trademarks of Sigma-Aldrich Co, LLC registered in the US and other countries. SAGE and CompoZr are registered trademarks of Sigma-Aldrich Co. LLC. SAGEspeed is a trademark of Sigma-Aldrich Co. LLC.

SOURCE: Sigma-Aldrich Corporation

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SAGE® Labs Creates The First Tissue-Specific Gene Deletion In Rats

Researchers have created artificial genetic material known as Xenonucleic acids, or XNAs, that can store information and evolve over generations in a comparable way to DNA.

The research, reported Friday in the journal Science, has implications for the fields of molecular medicine and biotechnology, and sheds new light on how molecules first replicated and assembled into life billions of years ago.

Living systems owe their existence to the information-carrying molecules DNA and RNA. These fundamental chemical forms have two features essential for life: they display heredity, meaning they can encode and pass on genetic information, and they can adapt over time.

Whether these traits could be performed by molecules other than DNA and RNA has been a long-debated issue.

For the current study, an international team of researchers developed chemical procedures to convert DNA and RNA into six genetic polymers known as XNAs. The process switches the deoxyribose and ribose (the d and r in DNA and RNA) for other molecules.

The researchers demonstrated for the first time that all six XNAs could form a double helix with DNA, and were more stable than natural genetic material. Moreover, one of these XNAs, a molecule known as anhydrohexitol nucleic acid, or HNA, was capable of undergoing directed evolution and folding into biologically useful forms.

Philipp Holliger of MRC Laboratory of Molecular Biology in Cambridge, the studys senior author, said the work demonstrated that heredity and evolution were possible using alternatives to natural genetic material.

There is nothing Goldilocks about DNA and RNA, he told Science.

There is no overwhelming functional imperative for genetic systems or biology to be based on these two nucleic acids.

Both RNA and DNA embed data in their sequences of four nucleotides. This information is vital for conferring hereditary traits and for supplying the coded recipe essential for building proteins from the 20 naturally occurring amino acids. However, precisely how and when this system began remains one of the most perplexing and hotly contested areas of biology.

The rest is here:
Researchers Create Synthetic DNA/RNA That Can Evolve

MOUNTAIN VIEW, Calif., April 20, 2012 /PRNewswire/ --To celebrate DNA Day, Khan Academy, the world's online classroom, and 23andMe, a leading personal genetics company, are working together to promote the importance of genetics education. 23andMe's Genetics 101 and Human Prehistory 101 video series are now available on KhanAcademy.org.

DNA Day commemorates the day in 1953 when James Watson, Francis Crick, and colleagues published papers in the journal Nature on the structure of DNA. Additionally, on the same date in 2003 it was declared that the Human Genome Project was very close to complete.

"The paper publications in the Nature journal and the Human Genome Projects are both landmark achievements, and more people should know about them. These events helped launch the field of genetics, and this field is a critical force that will shape our society in the future," said Sal Khan, Founder of Khan Academy.

Khan Academy is changing education for the better by providing a free world-class education for anyone anywhere. 23andMe is dedicated to helping individuals understand their own genetic information through DNA analysis technologies and web-based interactive tools. The company's Personal Genome Service enables individuals to gain deeper insights into their ancestry and inherited traits. Together, the two organizations are celebrating DNA Day by focusing on genetics and what individuals can learn from exploring their own DNA.

"A solid foundation in genetics is increasingly important for everyone," said Anne Wojcicki, CEO of 23andMe. "Our understanding of how DNA informs our health and development is advancing at an incredible pace.This research is critical for adopting a preventative and personalized approach to healthcare. It's also fascinating to better understand what causes the incredible human diversity."

23andMe also partners with high schools, colleges, and universities to improve understanding of genetic information and to foster a dialogue about personalized medicine. For academic classes with a focus on genetics, medicine, human biology, anthropology, or bioethics, 23andMe offers several resources, including its Personal Genome Service at a significantly reduced rate. You can learn more about 23andMe's Academic program at: http://www.23andme.com/academic.

Khan Academy currently offers several lessons on genetics that are typically seen in high school or first-year college curriculums, including: An introduction to DNA, Introduction to Heredity, the Hardy-Weinberg Principle, and Punnett Square Fun. All Khan Academy content is available for free at: http://www.khanacademy.org

About Khan Academy

Khan Academy is a 501(c)(3) non-profit with the mission of providing a free, world-class education for anyone, anywhere. The organization believes that students of all ages should have free access to top-notch educational content and be able to learn and master the content at their own pace. Khan Academy has delivered over 140 million lessons, and users have completed over 500 million exercises on http://www.khanacademy.org.

About 23andMe

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Khan Academy and 23andMe Partner to Promote Genetics Education on DNA Day

A gathering of scientists might not seem very glamorous, but the cancer researchers who came together at the Hyatt Regency Greenwich Thursday evening were celebrities at a gala marking the 10th anniversary of a local nonprofit that has provided more than $22.5 million to help investigate innovative therapies.

The Alliance for Cancer Gene Therapy celebrated its founding 10 years ago by Greenwich residents Barbara and Edward Netter, who decided to raise money for research into gene therapy after losing their daughter-in-law to breast cancer.

The event Thursday served as a tribute to Edward, who died last February. It also allowed the scientists to gather and discuss the cutting-edge research they are doing, and how the Alliance can help along the often painstakingly slow process of bringing new therapies to the market.

A few years ago, during the event celebrating the fifth anniversary of the Alliance, the scientists' discussion led to the eventual awarding of grants that recently paid off with a major breakthrough.

The nonprofit had already been funding so-called young investigators conducting exploratory research.

"We also envisioned there was a lack of science and clinical development," said Dr. Savio Woo, chairman of the Alliance's scientific advisory council. "You need to test patients."

Since then, the Alliance has begun giving out its Fund for Advancement awards, which provide up to $1 million to scientists conducting research that will move cell and gene therapies from the laboratory toward clinical trials.

Last year, an Alliance-funded gene therapy trial at the University of Pennsylvania caused two patients with chronic lymphocytic leukemia to go into complete remission, and significantly reduced the cancer in a third patient.

Dr. Carl June, who headed the small trial, which genetically modified the patients' own T-cells to target and kill the tumors, called the gathering Thursday a "celebration" of the work the Alliance has been supporting.

"After a decade, it's clear there are going to be FDA-approved products in cell and gene therapy that there weren't 10 years ago," June said.

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Greenwich gala celebrates cancer therapy breakthrough

THE WOODLANDS, Texas--(BUSINESS WIRE)--

Opexa Therapeutics, Inc. (NASDAQ: OPXA - News), a biotechnology company developing Tovaxin, a novel T-cell therapy for multiple sclerosis (MS), announced today that the Company will be holding a preliminary meeting with prospective clinical trial investigators at the 64th Annual American Academy of Neurology (AAN) Meeting in New Orleans on April 24, 2012. The purpose of this meeting will be to discuss the upcoming Phase IIb clinical trial using Opexas T-cell therapy in patients with Secondary Progressive Multiple Sclerosis (SPMS).

"We are honored to introduce our next clinical trial to a group of invited neurologists at this years AAN meeting in New Orleans, commented Neil K. Warma, President and Chief Executive Officer of Opexa. The meeting will be an opportunity to discuss with select clinicians and their study coordinators potential participation in the SPMS clinical trial as well as present the final protocol for the trial including the design, structure and patient selection criteria. This is an exciting time for Opexa, neurologists and SPMS patients as this study will provide an innovative opportunity for treatment in an area where currently there are very few treatment options. The annual AAN meeting is an excellent forum for the MS community to discuss and present new therapies that could have an important impact on the treatment of MS. We are pleased that Tovaxin is generating a great deal of enthusiasm among physicians, key opinion leaders and patients in the lead up to this meeting and are equally pleased to be advancing our clinical plans for Tovaxin.

Mark Freedman, M.D., director of the Multiple Sclerosis Research Unit at the Ottawa Hospital and member of Opexas Scientific Advisory Board, commented, I am pleased to contribute my expertise to Opexa with their design and planning of this Phase IIb study. Opexa is now in the process of selecting clinical trial investigators and finalizing the remaining steps in order to conduct a study of optimal quality. I am pleased to facilitate the introduction of the Phase IIb study to prospective clinical trial investigators at this years AAN meeting. Patients with SPMS have few treatment options and Tovaxins safety profile certainly justifies investigation of this therapy in the challenging SPMS patient population.

The proposed Phase IIb clinical trial will be a randomized, double-blind, placebo-controlled study of Opexas T-cell therapy in SPMS patients with evidence of disease progression without associated relapses. The study, to be initiated once the necessary resources are secured, is expected to treat approximately 180 patients in up to 30 sites in the United States and Canada with annual courses of treatment for two years.

About Opexa

Opexa Therapeutics, Inc. is dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS). The Companys leading T-cell therapy, a personalized cellular immunotherapy treatment, is in clinical development targeting both Secondary Progressive and Relapsing Remitting MS. Opexas T-cell therapy is derived from T-cells isolated from peripheral blood, expanded ex vivo and reintroduced into the patients via subcutaneous injections. This process triggers a potent immune response against specific subsets of autoreactive T-cells known to attack myelin and, thereby, reduces the risk of relapse over time.

For more information, visit the Companys website at http://www.opexatherapeutics.com.

Cautionary Statement Relating to Forward - Looking Information for the Purpose of "Safe Harbor" Provisions of the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The words expects, believes, anticipates, estimates, may, could, intends, and similar expressions are intended to identify forward-looking statements. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding the development of the Companys product candidate, Tovaxin, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: our capital position, the ability of the Company to enter into and benefit from a partnering arrangement for the Company's product candidate, Tovaxin, on reasonably satisfactory terms (if at all), our dependence (if partnered) on the resources and abilities of any partner for the further development of Tovaxin, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of developing a marketable product, our ability to raise additional capital to continue our treatment development programs and to undertake and complete any further clinical studies for Tovaxin, the success of our clinical trials, the efficacy of Tovaxin for any particular indication, such as Relapsing Remitting MS or Secondary Progressive MS, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights (including for Tovaxin), the risk of litigation regarding our intellectual property rights, the success of third party development and commercialization efforts with respect to products covered by intellectual property rights that the Company may license or transfer, our limited manufacturing capabilities, our dependence on third-party manufacturers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in our filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date made. We assume no obligation or undertaking to update any forward-looking statements to reflect any changes in expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based. You should, however, review additional disclosures we make in our reports filed with the Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2011.

Originally posted here:
Opexa to Hold Preliminary Investigator’s Meeting for MS Trial at American Academy of Neurology Annual Meeting

Researchers from the Gladstone Institutes in the US showed for the first time that injecting a combination of genes into the damaged heart tissue of a living animal could make it beat again.

Although the technique is a long way from being fit for human trials, experts said it could eventually lead to a way of repairing the debilitating damage sustained in heart attacks.

The results, published in the Nature journal, replicate previous test tube studies which suggested that the three genes, which normally guide the development of the heart in embryos, could reprogram non-beating cells into fully functional heart muscle.

Dr Deepak Srivastava, who led the study, said: "These findings could have a significant impact on heart-failure patients, whose damaged hearts make it difficult for them to engage in normal activities like walking up a flight of stairs."

Prof Peter Weissberg, Medical Director of the British Heart Foundation, added: This research illustrates one of many routes scientists are exploring to try and repair damage caused by a heart attack.

"If this is confirmed by further studies, it is a remarkable achievement. But a great deal more research will be needed before we will know whether such an approach is feasible, or indeed safe, in patients.

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Heart damage repaired in mice by gene therapy

A brain study has found a gene linked to intelligence, a small piece in the puzzle as to why some people are smarter than others. shutterstock.com

A variant of this gene can tilt the scales in favour of a higher intelligence, study leader Paul Thompson said, stressing though that genetic blessings were not the only factor in brainpower.

Searching for a genetic explanation for brain disease, the scientists stumbled upon a minute variant in a gene called HMGA2 among people who had larger brains and scored higher on standardised IQ tests.

Thompson dubbed it an intelligence gene and said it was likely that many more such genes were yet to be discovered.

The variant occurs on HMGA2 where there is just a single change in the permutation of the four letters of the genetic code.

DNA, the blueprint for life, comprises four basic chemicals called A (for adenine), C (cytosine), T (thymine) and G (guanine), strung together in different combinations along a double helix.

In this case, the researchers found that people with a double C and no T in a specific section of the HMGA2 gene had bigger brains on average.

It is a strange result, you wouldnt think that something as simple as one small change in the genetic code could explain differences in intelligence worldwide, said Thompson, a neurologist at the University of California at Los Angeles.

The discovery came in a study of brain scans and DNA samples from more than 20,000 people from North America, Europe and Australia, of European ancestry.

People who received two Cs from their parents, a quarter of the population, scored on average 1.3 points higher than the next group half of the population with only one C in this section of the gene.

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Tiny gene change affects brain size, IQ: Scientists

Public release date: 18-Apr-2012 [ | E-mail | Share ]

Contact: Vanessa Pavinato media@esmo.org European Society for Medical Oncology

Lugano-CH/Aurora-US-CO/Geneva-CH, 18 April 2012 -- A new genetic signature identified by Spanish researchers may provide doctors with robust and objective information about which patients with early stage lung cancer are at low or high risk of relapse following surgery, investigators report at the 3rd European Lung Cancer Conference in Geneva. Their work also opens new avenues for immunotherapy for lung cancer.

Non-small cell lung cancer is a disease that is often not diagnosed until it has grown and spread throughout the body. Even those patients who are diagnosed early enough to undergo surgical removal of the tumor still have a discouraging 30% rate of relapse.

Researchers hope that identifying which patients have the greatest risk of relapse will allow doctors to focus other treatment strategies, in order to improve their chance of being cured after surgery.

A multidisciplinary team of researchers from Hospital Clinico San Carlos, Madrid, have found a 50-gene predictor that appears to be capable of doing just that. In a study of 84 patients with stage I and II non-small cell lung cancer, who had undergone surgery to remove their tumor, the gene signature accurately predicted which patients were at low risk of relapse.

The researchers analyzed genetic expression in the tumor mass using microarray technology. Following patients for six years, they were able to correlate gene expression patterns with the clinical course of disease, and the risk of relapse.

The Spanish research team's report at the meeting shows that the genes of the predictor were over-expressed in roughly one-third of patients, all of whom had a low risk of relapse. Further analysis showed that these genes were related to the activity of important immune system white blood cells, known as B lymphocytes.

"All of these genes overexpressed in the low-risk group are highly related to B lymphocyte activity," said Dr Florentino Hernando presenting the results at the meeting. "So, the B cell-mediated immune response seems to have a very important role."

The genetic profile identified by the researchers suggests that low-risk patients have an enhanced immune response against the tumor. "Thus, treatments that may interfere with this response such as post-surgical chemotherapy must be reconsidered for the low-risk subgroup," he says.

Original post:
Gene signature helps identify risk of relapse in lung cancer patients

PISCATAWAY, N.J., April 19, 2012 /PRNewswire-Asia/ -- GenScript USA Inc., an internationally recognized biology contract research organization (CRO) has recently launched Rush Gene Synthesis service in the global market on Dec. 22, 2011, and has achieved 100% success and on time delivery rates ever since its kick-off.

"Two decades ago, a 2 Kb gene took me nearly two years to obtain the construct using conventional PCR cloning technologies in my PhD research. I did believe this could be changed. Nowadays, GenScript Rush Gene Synthesis service delivers synthetic genes in as little as 5 business days. The proprietary technology and a dedicated Rush Gene service team are the key points to making GenScript Rush Gene Synthesis service a big success," commented Frank Zhang, the CEO and co-founder of GenScript.

"Delivering synthetic genes in as little as 5 days, GenScript Rush Gene Synthesis service is not only fast but is accurate and worry-free to our customers," says Frank, "all our synthesized genes are packed into cloning vectors, picked from single clones and fully sequence-verified before the delivery. Therefore, none of our customers is bothered with picking up the right gene from the tube. With this rush service, we aim to drive molecular biology research faster than ever."

Besides the world-leading gene synthesis service, GenScript, as a contract research organization (CRO), also provides comprehensive services for biological research and early-phase drug discovery, such as bio-reagents, assay development & screening, lead optimization, antibody drug development and animal model services. The bio-reagents services include custom gene synthesis and molecular biology, custom protein expression and purification, custom peptide synthesis, antibody production, and custom cell line development. Headquartered in Piscataway, New Jersey, GenScript has three subsidiaries located in France, Japan, and China.

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GenScript Rush Gene Synthesis - Driving Molecular Biology Research Faster

ScienceDaily (Apr. 19, 2012) Scientists led by Washington University School of Medicine in St. Louis have identified the first gene directly linked to the most common form of psoriasis, a chronic skin condition.

The research shows that rare mutations in the CARD14 gene, when activated by an environmental trigger, can lead to plaque psoriasis. This type of psoriasis accounts for 80 percent of all cases and is characterized by dry, raised, red patches covered with silvery scales that can be itchy and painful.

The new findings also indicate that mutations in CARD14 can be involved in the pustular form of psoriasis and in a debilitating arthritis linked to the psoriasis. The discovery may lead to more effective, targeted therapies for plaque psoriasis and other forms of the disease.

The research is published May 4 in two separate papers in The American Journal of Human Genetics.

"We have searched for almost two decades to find a single gene linked to plaque psoriasis," says the senior author of both papers, Anne Bowcock, PhD, professor of genetics. "Individually, the rare mutations we have found likely confer a high risk for the disease, and we think they will be important in the search to find new, more effective treatments."

Although psoriasis has long been thought to be caused by an overactive immune system, the genetic pathway uncovered by the scientists points to defects in the skin as the main culprit of the condition and to immune cells as secondary players.

Now, the researchers want to find out how common the altered pathway is in the different types of psoriasis and in patients with psoriatic arthritis. Their work suggests that in at least some patients with different forms of psoriasis, this pathway is the same.

An estimated 7.5 million Americans have psoriasis, and about 30 percent of them develop psoriatic arthritis. Like other common diseases, psoriasis runs in families and has been thought to have a genetic component, but it's been difficult to pin down the genes involved. That's because common variations in genes likely contribute very little to the overall genetic risk of the disease, and mutations that substantially increase a person's risk are so rare they have been impossible to find.

With early support from the National Psoriasis Foundation, Bowcock initiated the research with co-author Alan Menter, MD, of the Psoriasis Research Institute at the Baylor College of Medicine.

Using the latest DNA technology to sequence all of a patient's genes, Bowcock and her colleagues uncovered a rare CARD14 mutation in a large family of northern European descent in which plaque psoriasis was prevalent. They also found the mutation in the one-third of family members who had developed psoriatic arthritis, suggesting that the same rare mutation can play a role in both conditions.

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First gene linked to common form of psoriasis identified

ST. LOUIS, April 19, 2012 /PRNewswire/ --Sigma-Aldrich Corporation (Nasdaq: SIAL - News) today announced that Sigma Advanced Genetic Engineering (SAGE) Labs, an initiative of Sigma Life Science, extended CompoZr Zinc Finger Nuclease (ZFN) technology to achieve the first tissue-specific conditional knockout of an endogenous gene in rats. For two decades this approach for generating sophisticated disease models could be performed only in mice. Rats, however, are preferred by drug discovery and basic researchers because the animal's physiology, neurobiology and other features are more predictive of human conditions. Rats engineered to contain tissue-specific conditional gene knockouts are available exclusively through the SAGEspeed Custom Model Development Service. Details are available at http://www.sageresearchmodels.com/conditional-KO.

(Logo: http://photos.prnewswire.com/prnh/20050215/CGSIGMAALLOGO)

Conventional gene knockout eliminates a gene throughout an entire animal. In contrast, conditional gene knockout can eliminate a gene solely in the relevant tissue or organ, leading to a more accurate understanding of the gene's function. Conditional gene knockout can also knock out genes at certain points in development, enabling studies of genes whose absence in embryos is lethal, but whose loss of function in adulthood is critical to investigate for many human diseases.

"Almost 89% of drug candidates fail to achieve approval," said Edward Weinstein, Director of SAGE Labs. "Basic and drug discovery researchers need access to more predictive animal models whose physiology, biology, and genetics more closely reflect specific human conditions. SAGE Labs is applying ZFN technology to achieve previously impossible genetic manipulations, such as tissue-specific gene deletion in rats."

Using the conditional knockout methodology, scientists at SAGE Labs have generated a pair of rat lines in which two important neuronal genes, Crhr1 and Grin1, were removed in specific neuronal populations. Crhr1 and Grin1 have been implicated as playing a role in depression and schizophrenia, respectively. The rat lines were developed through the SAGEspeed model creation process, which uses Sigma's CompoZr ZFN technology to create sophisticated genetic modifications in rats, mice, rabbits, and other organisms. CompoZr ZFN technology is the first to enable highly efficient, targeted editing of the genome of any species.

For more information and to request pricing, visit http://www.sageresearchmodels.com.

Cautionary Statement: The foregoing release contains forward-looking statements that can be identified by terminology such as "enable," "enabling," "leading to," "achieve," "predictive" 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 iPS cells, iPS-cell derived primary cell lines, novel assays, or related custom services will assist the Company to achieve any particular levels of revenue in the future. In particular, management's expectations regarding products associated iPS cells, iPS-cell derived primary cell lines, novel assays, or related custom 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.

About Sigma Life Science: Sigma Life Science is a Sigma-Aldrich business that represents the Company's leadership in innovative biological products and services for the global life science market and offers an array of biologically-rich products and reagents that researchers use in scientific investigation. Product areas include biomolecules, genomics and functional genomics, cells and cell-based assays, transgenics, protein assays, stem cell research, epigenetics and custom services/oligonucleotides. Sigma Life Science also provides an extensive range critical bioessentials like biochemicals, antibiotics, buffers, carbohydrates, enzymes, forensic tools, hematology and histology, nucleotides, amino acids and their derivatives, and cell culture media.

About Sigma-Aldrich: Sigma-Aldrich is a leading Life Science and High Technology company whose biochemical, organic chemical products, kits and services are used in scientific research, including genomic and proteomic research, biotechnology, pharmaceutical development, the diagnosis of disease and as key components in pharmaceutical, diagnostics and high technology manufacturing. Sigma-Aldrich customers include more than 1.3 million scientists and technologists in life science companies, university and government institutions, hospitals and industry. The Company operates in 40 countries and has nearly 9,000 employees whose objective is to provide excellent service worldwide. Sigma-Aldrich is committed to accelerating customer success through innovation and leadership in Life Science and High Technology. For more information about Sigma-Aldrich, please visit its website at http://www.sigma-aldrich.com.

Sigma-Aldrich and Sigma are trademarks of Sigma-Aldrich Co, LLC registered in the US and other countries. SAGE and CompoZr are registered trademarks of Sigma-Aldrich Co. LLC. SAGEspeed is a trademark of Sigma-Aldrich Co. LLC.

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SAGE® Labs Creates the First Tissue-Specific Gene Deletion in Rats

Public release date: 18-Apr-2012 [ | E-mail | Share ]

Contact: Michael Bernstein m_bernstein@acs.org 202-872-6042 American Chemical Society

Scientific insights that expand on the teachings of Mendel, Watson and Crick, and underpinnings of the Human Genome Project are moving drug companies along the path to development of new medicines based on deeper insights into how factors other than the genetic code influence health and disease. That's the topic of the cover story in the current edition of Chemical & Engineering News (C&EN), the weekly newsmagazine of the American Chemical Society (ACS), the world's largest scientific society.

The article, by C&EN Senior Editor Lisa M. Jarvis, focuses on the quiet revolution in epigenetics that has been sweeping through biology, chemistry and other scientific fields for the last several years. It explains how scientists initially believed that cracking the genetic code, achieved a decade ago, would lay out a straight path for inventing new medicines: Identify the genetic mutation behind a disease and then find a drug that overcomes it. But scientists now know that another layer of biochemical controls, an epigenetics layer, influences how and when genes work in health and disease without changing DNA itself. Early epigenetics research already produced four drugs currently approved to treat blood cancer. But these treatments lack selectivity, limiting their effectiveness.

Now, Jarvis explains, companies like GlaxoSmithKline, Epizyme and Constellation Pharmaceuticals are moving ahead to develop the next generation of epigenetic drugs, particularly for cancer. Armed with a better understanding of how specific epigenetic enzymes are implicated in disease, they are designing compounds to block the activity of those enzymes. The article describes GSK's announcement earlier this month of an epigenetic inhibitor it has developed that might fight lymphoma. "Although no one will know the value of the new epigenetic compounds until they are tested in humans, scientists are confident that the field is moving forward with the right balance of caution and enthusiasm," Jarvis concludes.

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Not by DNA alone: How the epigenetics revolution is fostering new medicines

A team of UCLA researchers has found a genetic engineering technique that suppresses the HIV virus in mice, an encouraging step toward potentially fighting the disease in humans, researchers said.

The study, led by Scott Kitchen, a member of the UCLA AIDS Institute and assistant professor at the David Geffen School of Medicine, draws upon previous UCLA research findings. It was published last week in the epidemiology journal PLoS Pathogens and funded in part by the UCLA Center for AIDS Research.

Researchers examined the effectiveness of genetically engineered killer T cells, which are capable of fighting off disease, at combating the HIV virus in a mouse. The team used a humanized mouse engineered to have a human immune system. In the humanized mouse, the disease progressed similarly to its progression in humans, making it a reliable tool for the study and providing powerful predictive value for the therapy in humans, Kitchen said. Its a major advance and a step closer in demonstrating the potential use of this in people, he said.

A few months ago, the team introduced a population of the engineered T cells into a mouse so they could develop and grow into a human immune system, Kitchen said. The researchers then conducted blood and organ tests at the second and sixth weeks, finding a decrease in the HIV levels and an increase in the cells HIV typically kills, according to the journal article.

The findings could theoretically be used to support a clinical trial in humans, said Jerome Zack, associate director at the UCLA AIDS Institute and co-author of the study.

A benefit genetic engineering is that it opens the field to therapeutic HIV treatments, and that it can be extended to potentially treat other diseases such as cancer, said co-author Arumugam Balamurugan.

In 2009, the lead scientists from the most recent study showed that human blood stem cells in mice could be genetically engineered to grow large quantities of killer T cells, As a result of their genetic engineering, these T cells grew to a large population and targeted HIV-infected cells in the mice.

We had the idea that we could take the elements of immune response (the T cells) that are successful in suppressing HIV in infected people to see if it was possible to identify a receptor specific to HIV, Kitchen said, referring to the teams research in 2009.

Though advances have been made in the fight against HIV, an estimated 50,000 new cases are diagnosed in the United States each year, and there are more than 33 million people living with the disease worldwide, according to the National Institutes of Health. Factors that make the disease difficult to fight include its rapid rate of spread and lack of preventative measures. The findings could lead to more comprehensive methods of fighting the disease and eventually to a clinical trial in humans, Kitchen said.

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UCLA researchers develop genetically engineered stem cells to fight HIV in mice

SAN DIEGO, April 19, 2012 /PRNewswire/ -- Sequenom, Inc. (NASDAQ: SQNM - News), a life sciences company providing innovative genetic analysis solutions, today announced an agreement with MultiPlan, Inc., the nation's leading provider of healthcare cost management solutions (PHCS). Under this expanded agreement, Sequenom's participation in the PHCS and MultiPlan networks will now include its Center for Molecular Medicine's MaterniT21 PLUS laboratory-developed test to detect certain fetal trisomies in women at increased risk of carrying a child with one of these chromosomal abnormalities. More than 900,000 providers participate in MultiPlan's networks.

"We are very pleased to extend our relationship with MultiPlan, giving their clients' members and their network of participating providers access to the MaterniT21 PLUS test," said Harry F. Hixson, Jr., Ph.D., Chairman and CEO, Sequenom, Inc. "Of the many important decisions to make in a high-risk pregnancy, the coverage of a valuable noninvasive prenatal test will be a relief to many expectant families."

The MaterniT21 PLUS test is intended for use in pregnant women at increased risk for fetal aneuploidy and can be used as early as 10 weeks gestation. In the United States, there are an estimated 750,000 such high-risk pregnancies each year. Results of the MaterniT21 PLUS test delivered to ordering physicians will include the presence of any of the three trisomies for their high-risk patients. The MaterniT21 PLUS test is available through Sequenom CMM as a testing service to physicians. To learn more about the MaterniT21 PLUS test, please visit Sequenomcmm.com.

About SequenomSequenom, Inc. (NASDAQ: SQNM - News) is a life sciences company committed to improving healthcare through revolutionary genetic analysis solutions. Sequenom develops innovative technology, products and diagnostic tests that target and serve discovery and clinical research, and molecular diagnostics markets. The company was founded in 1994 and is headquartered in San Diego, California. Sequenom maintains a Web site at http://www.sequenom.com to which Sequenom regularly posts copies of its press releases as well as additional information about Sequenom. Interested persons can subscribe on the Sequenom Web site to email alerts or RSS feeds that are sent automatically when Sequenom issues press releases, files its reports with the Securities and Exchange Commission or posts certain other information to the Web site.

About Sequenom CMM, LLCSequenom Center for Molecular Medicine (Sequenom CMM), a CAP accredited and CLIA-certified molecular diagnostics laboratory, is developing a broad range of laboratory developed tests with a focus on prenatal and ophthalmic diseases and conditions. These laboratory-developed tests provide beneficial patient management options for obstetricians, geneticists and maternal fetal medicine specialists. Sequenom CMM is changing the landscape in genetic disorder diagnostics using proprietary cutting edge technologies.

About MultiPlanMultiPlan, Inc. is the industry's most comprehensive provider of healthcare cost management solutions. The company provides a single gateway to a host of primary, complementary and out-of-network strategies for managing the financial risks associated with healthcare claims. Clients include insurers/health plans, third party administrators, self-funded employers, HMOs and other entities that pay medical bills in the commercial healthcare, government, workers compensation and auto markets. MultiPlan is owned by BC Partners, a leading international private equity firm, and Silver Lake, the world's largest private investor in technology. For more information, visit http://www.MultiPlan.com.

Forward-Looking StatementsExcept for the historical information contained herein, the matters set forth in this press release, including statements regarding performance, participation, or expectations under Sequenom's agreement with MultiPlan, the impact or effect of the agreement or coverage on expectant families, the intended use of the MaterniT21 PLUS test and the results delivered to ordering physicians, Sequenom's commitment to improving healthcare through revolutionary genetic analysis solutions, developing a broad range of laboratory developed tests, and changing the landscape in genetic disorder diagnostics, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially, including the risks and uncertainties associated with Sequenom's ability to develop and commercialize new technologies and products, particularly new technologies such as prenatal and other diagnostics and laboratory developed tests, Sequenom's ability to manage its existing cash resources or raise additional cash resources, competition, intellectual property protection and intellectual property rights of others, government regulation particularly with respect to diagnostic products and laboratory developed tests, obtaining or maintaining regulatory approvals, ongoing litigation and other risks detailed from time to time in Sequenom, Inc.'s most recent Annual Report on Securities and Exchange Commission Form 10-K and other documents subsequently filed with or furnished to the Securities and Exchange Commission. These forward-looking statements are based on current information that may change and you are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. All forward-looking statements are qualified in their entirety by this cautionary statement, and Sequenom, Inc. undertakes no obligation to revise or update any forward-looking statement to reflect events or circumstances after the issuance of this press release.

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Sequenom Announces National Agreement with Provider MultiPlan, Inc.

ScienceDaily (Apr. 17, 2012) When a child has autism, siblings are also at risk for the disorder. New research from Washington University School of Medicine in St. Louis shows that the genetic reach of the disorder often extends to half siblings as well.

On the surface, the finding may not be surprising -- half siblings share about 25 percent of their genes. But the discovery is giving scientists new clues to how autism is inherited.

The study is published online in the journal Molecular Psychiatry.

According to principal investigator John N. Constantino, MD, the new research adds to recent evidence that even though autism is far more common in males, females still can inherit and pass along genetic risk for autism.

"We found that autism risk for half siblings is about half of what it is for full siblings," he says. "Most of the half siblings we studied had the same mothers. Given that half of the risk of transmission was lost and half was preserved among those maternal half siblings, mothers and fathers appear to be transmitting risk equally in families in which autism recurs."

Constantino, the Blanche F. Ittleson Professor of Psychiatry and Pediatrics and director of the William Greenleaf Eliot Division of Child and Adolescent Psychiatry at Washington University and psychiatrist-in-chief at St. Louis Children's Hospital, says the findings also suggest that in many families, the transmission of autism is the result of the effects of many genes -- not just one -- with each contributing a small proportion of risk.

Prior estimates of the extent to which autism is influenced by genetic factors are derived from studies of identical and fraternal twins where one, or both, are affected by the disorder. Since identical twins share 100 percent of their genes, and fraternal twins share 50 percent, inherited conditions tend to be twice as common in an identical twin pair compared to a fraternal twin pair. But twin studies of autism are too small to give precise estimates about how the disorder is inherited.

"The largest studies have included less than 300 clinically affected twin pairs," Constantino says. "And they include girls, boys and mixed twin pairs, which complicates the testing of models of inheritance in autism because the disorder is much more common in boys than girls."

Other studies have focused on siblings of children with autism, looking at how much more common autism recurrence is in siblings than the general population. But to derive more information on genetic structure from their family studies, Constantino's group looked at autism recurrence in half siblings and compared it to that in full siblings.

The researchers studied over 5,000 families in which there was a child with autism and at least one additional sibling -- the families were enrolled in a national volunteer, Internet-based family registry for autism, the Interactive Autism Network (IAN). Among those families, 619 included at least one maternal half-sibling. The researchers focused on maternal half-siblings rather than paternal half siblings because these children were more likely to live full-time with their biological mothers and to share the same environmental influences between the time they were born and the age of two, the time at which the onset of autistic syndromes occur. They compared autism recurrence among the 619 maternal half siblings to the rate among 4,832 full siblings.

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Study of half siblings provides genetic clues to autism

Public release date: 18-Apr-2012 [ | E-mail | Share ]

Contact: Raquel Maurier raquel.maurier@ualberta.ca 780-492-5986 University of Alberta Faculty of Medicine & Dentistry

(Edmonton) A medical researcher at the University of Alberta is working with scientists from across North America to find out if there are genetic markers for autism. More than 15 scientists will examine DNA samples from children with autism and their infant siblings to see if the siblings are at high risk.

And if siblings are at high risk for developing autism, could intervention start before symptoms develop?

If theres a family of a child with autism, they of course wonder what the risk for the younger infant is, says Lonnie Zwaigenbaum, a U of A researcher with the Department of Pediatrics in the Faculty of Medicine & Dentistry, who is the principal investigator for the two-year study.

We want to know if its possible to use genetic biomarkers as a way of giving these families a more accurate estimate of what that siblings risk is. If there was a way of using genetic biomarkers to identify infants at high-risk before the more overt manifestations of autism were obvious, it would really open the door to provide support and intervention at earlier stages of development.

According to the Autism Speaks Canadian website, autism, part of a group of disorders known as Autism Spectrum Disorders (ASD), is a complex neurobiological disorder that typically lasts throughout a persons lifetime. The disorder is characterized by varying degrees of impairment in communication skills and social abilities, and also by repetitive behaviors. Autism, which is more common among boys than girls, affects 1 in 88 children, according to the Centers for Disease Control and Prevention.

Zwaigenbaum is working with scientists in Toronto, Hamilton, Halifax and various American universities including: the University of Washington, UC Davis, Vanderbilt, UC San Diego, Kennedy Krieger Institute/Johns Hopkins University, and the University of Miami.

This research initiative is funded by the Simons Foundation and by Autism Speaks. The $1.3 million in funding will go towards the creation of a biorepository: a collection of DNA samples and other biospecimens from families with a child with autism and a younger infant, to complement the clinical data. This collection will be mined by this research group and the broader scientific community studying autism. Additional funding provided by Autism Speaks and Autism Speaks Canada will support the analysis of the collected blood samples.

Further studies of genetic risk factors will help us understand the full spectrum of autism, says Gerald D. Fischbach, scientific director of the autism research initiative and director of life sciences at the Simons Foundation. It is essential to identify such markers in young children. Lonnie Zwaigenbaum is one of the leaders in studies of infant siblings of children known to be on the autism spectrum. This vulnerable population would be best served by early diagnosis and that is the goal of this project.

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Researchers across North America team up to find genetic markers for autism

18-04-2012 02:33 Find out about the basics of cells, chromosomes, and the genes contained in your DNA.

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Genetics 101 Part 1: What are genes? - Video

18-04-2012 02:35 Learn about the variations in human DNA called SNPs, and how they can be used to understand relationships between people.

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Genetics 101 Part 2: What are SNPs? - Video

Even if humans hadn't hunted the Tasmanian tiger to extinction, its low genetic diversity may have naturally doomed the curious marsupial, researchers have found.

"We found that the thylacine had even less genetic diversity than the Tasmanian devil," study researcher Andrew Pask, of the University of Connecticut, said in a statement. "If they were still be around today, they'd be at a severe risk, just like the devil."

The Tasmanian tiger (Thylacinus cynocephalus), also known as the thylacine, was hunted to extinction in the early 1900s; the last one died in a Tasmanian zoo in 1936. Named for its telltale stripes, the Tasmanian tiger stood as tall as a medium-size dog and once roamed across both mainland Australia and Tasmania. [ Marsupial Gallery: A Pouchful of Cute ]

The new research captured some genetic fragments from the Tasmanian tiger, from 14 samples including pelts, bones and preserved specimens more than 100 years old. The scientists found the individuals to be 99.5 percent similar over a portion of the genome that normally has lots of differences.

"If we compare this same section of DNA, the Tasmanian tiger only averages one DNA difference between individuals, whereas the dog, for example has about five to six differences between individuals," study researcher Brandon Menzies, also of the University of Connecticut, said in a statement.

Genetic variability is basically the difference in the gene sequence between any two individuals. Analysis of the recovered genome indicates that the animal would have had too little genetic variability to survive. When this gets low, it spells doom for a species, because the species has more difficulty adapting to threats if it doesn't have a greater pool of genes to pull from.

Low genetic diversity can arise from many different situations: when a species consisting of many small isolated populations sees a precipitous drop in numbers or goes through a lot of inbreeding. In the case of the Tasmanian devil and the Tasmanian tiger, their low genetic diversity probably came from small groups that remained isolated from the main population in mainland Australia.

The tiger's extant cousin, the Tasmanian devil, is currently being decimated by a contagious cancer. The researchers say the devil's low genetic diversity allowed this disease to spread all the easier. The Tasmanian tiger, if around today, would also be exceptionally susceptible to diseases, the researchers said.

Knowing more about the Tasmanian tiger can help researchers fight for the still-living native species, like the Tasmanian devil. "From a conservation standpoint, we need to know these things about animals' genomes," Pask said. "There are a lot of fragile animals in Australia and Tasmania."

More science news from msnbc.com

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Tasmanian tiger likely doomed by genetics

Newswise BETHESDA, MD April 18, 2012 -- The Genetics Society of America (GSA) invites the public to visit its exhibit booth, Americas Next Top Model Organism, at the 2nd USA Science & Engineering Festival (USASEF) in Washington, D.C., April 28-29, 2012, and examine the workhorses of genetics research, including live mice, fruit flies, fungi, and Arabidopsis plants. Learn why geneticists conduct research on these systems and then vote for your favorite model organism.

The GSA exhibit will feature several activities. Participants will be able to observe normal (or wildtype) and mutant examples of mice, fruit flies, fungi, and Arabidopsis plants. Students will be able to build-a-fly based on common genetic traits eyes, wings and other characteristics to take home with them. GSA will also demonstrate the impact of evolution by illustrating how modern-day corn was bred from ancient maize. Most importantly, visitors will be able to learn about genetics research from practicing scientists who are GSA members. They will be volunteering at the booth to help with activities and answer questions.

This is an exciting outreach opportunity for GSA, said Adam Fagen, Ph.D., Executive Director. We look forward to engaging directly with the public to explain the value of genetics for enhancing our understanding of the natural world, including the contributions of scientific research on model organisms such as mice, fruit flies, fungi, and plants, he added.

The USAEF is designed to celebrate science and is being held Saturday, April 28 and Sunday, April 29, 2012 in downtown Washington, D.C. with satellite events worldwide. The Festival, which includes an Expo & Book Fair, is a free event open to children and adults, and features more than 3,000 fun, interactive exhibits including GSAs plus more than 100 stage shows and 33 author presentations. Several hundred thousand people attended the first USASEF in 2010.

The Exhibit Hall is open to the general public, Saturday, April 28, from 10:00 AM - 6:00 PM, and Sunday, April 29, from 10:00 AM - 4:00 PM at the Walter E. Washington Convention Center in Washington, D.C., 801 Mount Vernon Place, NW, near the Mt. Vernon Sq/7th St Convention Center Metro stop. The GSA booth is 2711 and is located in Hall A, to the left of the exhibit entrance and near the National Institutes of Health exhibit. For more information on GSAs involvement in the USASEF, contact Beth Ruedi at eruedi@genetics-gsa.org.

About the USA Science & Engineering Festival: The USA Science & Engineering Festival is the countrys only national science festival, and was developed to increase public awareness of the importance of science and to encourage youth to pursue careers in science and engineering by celebrating science in much the same way as we celebrate Hollywood celebrities, professional athletes and pop stars. Lockheed Martin is again the presenting host of the USA Science & Engineering Festival and is joined by many other Festival sponsors and partners. The USA Science & Engineering Festival is a grassroots collaboration of over 500 of the United States leading science and engineering organizations. For more information on the USA Science & Engineering Festival, please visit the Festival website.

ABOUT GSA: Founded in 1931, the Genetics Society of America (GSA) is the professional membership organization for scientific researchers, educators, bioengineers, bioinformaticians and others interested in the field of genetics. Its nearly 5,000 members work to advance knowledge in the basic mechanisms of inheritance, from the molecular to the population level. The GSA is dedicated to promoting research in genetics and to facilitating communication among geneticists worldwide through its conferences, including the biennial conference on Model Organisms to Human Biology, an interdisciplinary meeting on current and cutting edge topics in genetics research, as well as annual and biennial meetings that focus on the genetics of particular organisms, including C. elegans, Drosophila, fungi, mice, yeast, and zebrafish. GSA publishes GENETICS, a leading journal in the field and a new online, open-access publication, G3: Genes|Genomes|Genetics. For more information about GSA, please visit http://www.genetics-gsa.org. Also follow GSA on Facebook at facebook.com/GeneticsGSA and on Twitter @GeneticsGSA.

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Learn About Genetics at the USA Science & Engineering Festival

NEW HAVEN Conn., April 18, 2012 /PRNewswire/ -- JS Genetics announces the launch of XCAT-FX, its propitiatory buccal swab test for the detection of Fragile X syndrome. Fragile X syndrome is the most common cause of inherited intellectual disability (mental retardation) in males and a leading cause of infertility and fragile X-associated primary ovarian insufficiency in females. Fragile X is also the most common known genetic (single gene) cause of autism. This PCR based test can identify Fragile X in both males and female patients.

The result of XCAT-FX will typically be available within 3 business days following the receipt of the samples at JS Genetics' CLIA certified, CAP accredited laboratory.

"I am very pleased that we are now including Fragile X in the JS Genetics diagnostic menu," stated Henry Rinder MD, Medical Director of JS Genetics. "Fragile X testing will further enhance our laboratory's ability to aid physicians in making the sometimes difficult diagnosis of disorders of sex chromosomes, which also include Turner and Klinefelter syndromes and related sex chromosome aneuploidies."

About JS Genetics For more information about JS Genetics or XCAT-FX visit http://www.jsgenetics.com. Physicians who would like to obtain testing kits at no charge can order at http://www.jsgenetics.com or call1-888-217-8947.

JS Genetics Inc., a private company, develops and markets proprietary, high value DNA diagnostic tests for medical conditions in newborns, children, and adolescents. (CLIA ID# 07D1091103; CAP# 72115351)

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JS Genetics Announce Launch of its Bloodless, Cheek Swab Test for Fragile X Syndrome

SAN MARINO, Calif.--(BUSINESS WIRE)--

Viral Genetics (Pinksheets: VRAL.PK - News) today published its April 2012 Letter to Shareholders. Providing updates on the companys progress, the letter discusses the expanded product development pipeline for the Targeted Peptide and the Metabolic Disruption Technology platforms, as well as developments at the VG Energy subsidiary, and moves being made to manage this new growth.

The letter is available on the companys website at http://www.viralgenetics.com/shareholder-letters/Letter-to-Shareholders-Apr-2012.PDF.

About Viral Genetics, Inc.

San Marino, California-based Viral Genetics discovers drug therapies from two platform technologies based on over 60 patents: Metabolic Disruption (MDT) and Targeted Peptides (TPT). Founded in 1994, the biotech company is researching treatments for HIV/AIDS, Lyme Disease, Strep, Staph and drug resistant cancer. A majority-owned subsidiary, VG Energy (www.vgenergy.net), is dedicated to exploring biofuel and agricultural applications for the MDT platform. For more information, visit http://www.viralgenetics.com.

About VG Energy

VG Energy Inc. is an alternative energy and agricultural biotech company that is a majority-owned subsidiary of Viral Genetics Inc. Using its Metabolic Disruption Technology (MDT), Viral Genetics' cancer research led to discoveries with major consequences in a wide variety of other industries, including production of biofuel and vegetable oils. VG Energy holds the exclusive worldwide license to the MDT patent rights for use in the increase of production of various plant-derived oils from algae and seeds. Application of MDT technology to the biofuel industry could potentially allow it to overcome its major obstacle in the area of production efficiency: namely, an increase in production yields leading to feasible economic returns on investment, allowing renewable biodiesel to be competitive with fossil fuels. For more information, please visit http://www.vgenergy.net.

SAFE HARBOR FOR FORWARD-LOOKING STATEMENTS:

This news release contains forward-looking statements that involve risks and uncertainties associated with financial projections, budgets, milestone timelines, clinical trials, regulatory approvals, and other risks described by Viral Genetics, Inc. from time to time in its periodic reports, including statements about its VG Energy, Inc. subsidiary. None of Viral Genetics' drug compounds are approved by the US Food and Drug Administration or by any comparable regulatory agencies elsewhere in the world, nor are any non-pharmaceutical products of VG Energy, Inc. commercialized. While Viral Genetics believes that the forward-looking statements and underlying assumptions reasonable, any of the assumptions could be inaccurate, including, but not limited to, the ability of Viral Genetics to establish the efficacy of any of its drug therapies in the treatment of any disease or health condition, the development of studies and strategies leading to commercialization of those drug compounds in the United States, the obtaining of funding required to carry out the development plan, the completion of studies and tests including clinical trials on time or at all, the successful outcome of such studies or tests, or the successful commercialization of VG Energy, Inc.s non-pharmaceutical products. Therefore, there can be no assurance that the forward-looking statements included in this release will prove to be accurate. In light of the significant uncertainties inherent in the forward-looking statements included herein, the forward-looking statements should not be regarded as a representation by Viral Genetics or any other person that the objectives and plans of Viral Genetics will be achieved.

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Viral Genetics April 2012 Shareholder Update Alerts Investors to Product Pipeline Expansion and Developments at Algal ...

NEW YORK, April 19, 2012 /PRNewswire/ --N-Gene Research Laboratories, Inc. ("N-Gene"), today announces that Australian scientists in close cooperation with N-Gene have shown that BGP-15, a heat shock protein inducer, may be a novel therapy for treating the deadly, rare disease, Duchenne Muscular Dystrophy (DMD), as recently reported in the journal Nature.

Currently there is no cure and no adequate therapy for DMD. BGP-15 was not only able to make a key protein functional again, thereby reducing muscle damage, but also increase the strength and endurance, and ultimately the lifespan, of DMD animals.

"This pioneering work strengthens N-Gene's roleat the forefront of heat shock protein science," said Gabor K. Kalman, chief executive officer of N-Gene.

Peter Literati, Ph.D., co-founder and chief scientific officer of N-Gene said, "We believe this publication reinforces the expectation that N-Gene's platform technology, based on stress-response regulation, will eventually result in the emergence of a novel drug class with diverse therapeutic directions. We are remaining focused on advancing BGP-15 for the treatment of type 2 diabetes, but we look forward to seeking partners with which to advance this technology and realize its potential in the treatment of DMD as well as many other disease indications."

Dr. Literati will be speaking at a news event today in Budapest, Hungary, to discuss the data described above, the publication and the potential for N-Gene compounds to treat DMD and many other diseases.

About N-Gene Research Laboratories

N-Gene Research Laboratories, Inc. ("N-Gene"), is a biopharmaceutical research company with a pipeline of proprietary drugs to treat insulin-resistance syndrome, improve cancer chemotherapy and target other major disease classes. BGP-15, N-Gene's lead compound, is a co-inducer of HSP-72 that increases insulin sensitivity via blocking JNK phosphorylation. Results from a defining international Phase 2b study of BGP-15 in patients with type 2 diabetes are expected by the end of 2012.

For more information please visit http://www.ngene.us.

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Nature Publishes Work Utilizing N-Gene's Core Technology to Advance the Treatment of Duchenne Muscular Dystrophy

ANN ARBOR, Mich.--(BUSINESS WIRE)--

RetroSense Therapeutics, a biotechnology company dedicated to developing gene therapy approaches to vision restoration welcomes Peter Francis, MD, PhD to its senior management team as Clinical Director.

Dr. Francis brings extensive clinical experience in retinal degenerative conditions to the team. As we progress toward the clinic with our treatment, Dr. Francis experience in ocular gene therapy will be instrumental, stated Sean Ainsworth, CEO and founder of RetroSense Therapeutics. His passion and enthusiasm for helping patients will be well-placed at RetroSense.

Dr. Francis has been influential in the early clinical development of several novel therapeutic approaches, including gene therapies. For his outstanding work, throughout his career, he has received numerous awards including "best up-and-coming medical researcher in the United Kingdom 2002" and the Foulds Trophy best-research prize at the UK National Ophthalmology Conference. Dr. Francis recently directed a translational research center specializing in human clinical trials for orphan and inherited diseases of the retina, and has participated in preparation and submission of multiple INDs to the FDA.

I am very excited to join the RetroSense team. We are working to swiftly and safely bring our optogenetic therapy for the eye to the clinic. This treatment is exciting because it has great promise for vision restoration in patients with currently blinding diseases of the retina.

Dr. Francis graduated from medical school in the UK and undertook residency in London, UK. He is fellowship trained in retina and genetics (Moorfields Eye Hospital, London, and Casey Eye Institute, USA). Dr Francis has held academic faculty positions at St Thomas Hospital, London and most recently, Casey Eye Institute, Portland, OR.

About RetroSense Therapeutics:

RetroSense Therapeutics is a biotechnology company developing a game-changing gene therapy to restore vision in patients suffering from blindness due to retinitis pigmentosa (RP) and advanced dry age-related macular degeneration (advanced dry-AMD). There are currently no FDA approved therapies to improve or restore vision in patients with these retinal degenerative conditions. RetroSense is led by a team of seasoned veterans with deep experience in taking products from the discovery stage through to the clinic. For more information about RetroSense, visit http://www.retro-sense.com/.

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RetroSense Welcomes Dr. Peter Francis as Clinical Director

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

Contact: Bonnie Prescott bprescot@bidmc.harvard.edu 617-667-7306 Beth Israel Deaconess Medical Center

BOSTON -- In a finding that may challenge popular notions of body fat and health, researchers at Beth Israel Deaconess Medical Center (BIDMC) have shown how fat cells can protect the body against diabetes. The results may lead to a new therapeutic strategy for preventing and treating type 2 diabetes and obesity-related metabolic diseases, the authors say.

In the last decade, several research groups have shown that fat cells in people play a major role in controlling healthy blood sugar and insulin levels throughout the body. To do this crucial job, fat cells need a small portion of the sugars derived from food. Obesity often reduces the dedicated sugar transport molecules on fat cells, blocking the glucose from entering fat cells. As a result, the whole body becomes insulin resistant, and blood sugar rises, leading to diabetes.

The new study shows why glucose is so important to fat cells. The team discovered a new version of a gene inside fat cells that responds to sugar with a powerful systemic effect.

"If we change that one gene, that makes the animal more prone to or more protected from diabetes," said senior author Barbara Kahn MD, the George R. Minot Professor of Medicine at Harvard Medical School and Vice Chair of the Department of Medicine at BIDMC. "Many foods get converted into sugar, so there is no need to eat more sugar."

The paper is published online April 1 in the journal Nature. In the study, the BIDMC researchers pinpointed the fat gene and its effect in mouse models of human obesity and insulin resistance and reported supporting evidence from fat tissue samples from both lean and obese people.

"Two things were surprising first, that a lone gene could shift the metabolism of the fat cell so dramatically and then, that turning on this master switch selectively in adipose tissue is beneficial to the whole body," Kahn said. Twelve years ago, Kahn first demonstrated that fat cells are a master regulator of healthy levels of glucose and insulin in mice and require sugar to do the job.

"The general concept of fat as all bad is not true," said first author Mark Herman MD, an investigator in the Division of Endocrinology, Diabetes and Metabolism at BIDMC and Instructor of Medicine at Harvard Medical School (HMS). "Obesity is commonly associated with metabolic dysfunction that puts people at higher risk for diabetes, stroke and heart disease, but there is a large percentage of obese people who are metabolically healthy. We started with a mouse model that disassociates obesity from its adverse effects."

In the latest study, evidence suggests the newfound gene also may account for the protective effect of glucose uptake in human fat. German collaborators found more gene activity in people with greater insulin sensitivity, based on 123 adipose tissue samples from non-diabetic, glucose tolerant people. The fat gene activity also correlated highly with insulin sensitivity in obese, non-diabetic people, as measured in 38 fat samples by another pair of co-authors based in St. Louis.

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Study finds protective gene in fat cells