Archive for the ‘Gene Therapy Research’ Category

Public release date: 30-Aug-2012 [ | E-mail | Share ]

Contact: Quinn Eastman qeastma@emory.edu 404-727-7829 Emory University

Scientists have established a genetic mouse model for primary ovarian insufficiency (POI), a human condition in which women experience irregular menstrual cycles and reduced fertility, and early exposure to estrogen deficiency.

POI affects approximately one in a hundred women. In most cases of primary ovarian insufficiency, the cause is mysterious, although genetics is known to play a causative role. There are no treatments designed to help preserve fertility. Some women with POI retain some ovarian function and a fraction (5-10 percent) have children after receiving the diagnosis.

Having a mouse model could accelerate research on the causes and mechanisms of POI, and could eventually lead to treatments, says Peng Jin, PhD, associate professor of human genetics at Emory University School of Medicine.

The results were published online recently in the journal Human Molecular Genetics.

The paper was the result of a collaboration between researchers at Emory and the Institute of Zoology, Chinese Academy of Sciences in Beijing. Dahua Chen, PhD, associate director of the State Key Laboratory of Reproductive Biology, is the senior author and postdoctoral fellow Cuiling Lu is the first author. Stephanie Sherman, PhD, professor of human genetics at Emory, is a co-author.

The mouse model builds on research on women who are carriers of a "premutation" for fragile X syndrome, a leading cause of inherited intellectual disability.

The mice have a fragment of a human X chromosome from a fragile X premutation carrier. Other non-genetic mouse models used to study menopause include surgical removal of the ovaries, or exposure of mice to a chemical, 4-vinylcyclohexene diepoxide, which depletes the ovaries.

"While the fragile X premutation is a leading cause of POI, I think this model will be useful and relevant for all women with this condition," Jin says.

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Early menopause: A genetic mouse model of human primary ovarian insufficiency

NEW YORK (GenomeWeb News) Australian molecular diagnostics firm Genetic Technologies has filed a lawsuit against Chicago-based Reproductive Genetics Institute for alleged infringement of a patent covering its non-coding DNA technology.

The suit was filed this week in the US District Court for the Northern District of Illinois Eastern Division and alleges that RGI infringes US Patent No. 5,612,179, entitled, "Intron Sequence Analysis Method for Adjacent Locus Alleles as Haplotypes." Specifically, Genetic Technologies claims that RGI's screening services for cystic fibrosis infringe the patent.

The Australian firm has asked the court for a judgment that RGI is infringing the '179 patent, and it is seeking unspecified damages for that alleged infringement.

The '179 patent has been at the center of several suits that Genetic Technologies has filed against other firms including Applera, Beckman Coulter, Gen-Probe, Interleukin Genetics, Orchid Cellmark, Pioneer Hi-Bred and others. All of those parties have since taken a license to the '179 patent from Genetic Technologies, according to the suit filed this week.

The patent also is the center of a suit filed in May 2011 by Genetic Technologies against Agilent Technologies and nine other firms. Genetic Technologies settled that case with a few of the defendants GeneSeek, Navigenics, Hologic, and Eurofins earlier this year.

Genetic Technologies noted in the suit filed this week that it has secured more than $15 million in licensing revenue since filing its initial action against Beckman Coulter in 2010.

More here:
Genetic Technologies Sues Reproductive Genetics Institute

SALT LAKE CITY, Aug. 30, 2012 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN) announced today that Peter D. Meldrum, President and CEO, is scheduled to present at the Morgan Stanley Global Healthcare Conference, at 1:00 p.m. Eastern Time on Wednesday, September 12, 2012. The conference is being held at The Grand Hyatt New York in New York, New York.

The presentation will be available to interested parties through a live webcast accessible on the investor relations section of Myriad's website at http://www.myriad.com.

About Myriad Genetics

Myriad Genetics is a leading molecular diagnostic company dedicated to making a difference in patients' lives through the discovery and commercialization of transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess risk of disease progression and recurrence. Myriad's portfolio of molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a commitment to improving an individual's decision making process for monitoring and treating disease. Myriad is focused on strategic directives to introduce new products, including companion diagnostics, as well as expanding internationally. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com

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Myriad Genetics to Present at the Morgan Stanley Global Healthcare Conference

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The two rice plant rows on the left are bigger and growing much better they have the PSTOL1 gene, whereas the two rice plant rows on the right do not have the PSTOL1 gene and they look smaller. Part of the image collection of the International Rice Research Institute (IRRI).

BARCELONA, Spain - A multinational team headed by Dr. Sigrid Heuer of the International Rice Research Institute (IRRI), has pinpointed a gene that can significantly increase rice harvest grown in phosphorus-deficient soil.

The team, writing in the journal Nature, said that the gene can enhance grain yield by as much as 60% compared to rice varieties which do not have it based on their controlled experiment.

This finding is significant in increasing rice yield for two reasons:

The gene is named PSTOL1 or Phosphorus-starvation tolerance 1.

Its main function, according to the authors, is to regulate the early crown root development and root growth in rice. Hence, rice with an over expression of this gene produces more roots that are also healthier.

This fact also helps rice plants to have higher uptake of other nutrients such as nitrogen and potassium. This may translate to lesser dependency on fertilizers without a reduction in yield.

Jackpot

PSTOL1, phosphorus uptake research team members from right to left are: Sigrid Heuer, Cheryl Dalid, Rico Gamuyao, Matthias Wissuwa and Joong Hyoun Chin. Part of the image collection of the International Rice Research Institute (IRRI).

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Researchers find gene that can raise rice harvest

An August 25, 2012 Washington Post article talked about a superbug outbreak at the NIH. The article highlighted the problem that we are running out of useful antibiotics. Antibiotic resistance is an evolutionary question of great practical importance. I have recently been asked and agreed to sign on to petitions requesting that antibiotic use in animals be banned for the sake of human health.

There is no evolutionary phenomenon we understand better than multiple antibiotic resistance in bacteria. It has been occurring virtually synchronously with the development of molecular genetics for 60 years. For public health as well as scientific reasons, antibiotic resistance was a major focus of the first decades of molecular biology.

Let us use this well-studied phenomenon to disentangle some of the thorny questions raised and debated in my most recent two blogs dealing with evolutionary processes (you can read them here and here). Our knowledge of the underlying cell and molecular biology makes it possible to discuss these questions in terms of specific experimental results rather than abstract principles.

The chief factor in the rapid evolution of multiple antibiotic resistance is the presence of sequences encoding resistance mechanisms on transmissible plasmids (Watanabe 1967). It was my PhD supervisor, Bill Hayes, who first demonstrated the existence of transmissible plasmids. They have proved to be extremely important evolutionary tools in bacteria.

Working under minimalist conditions (Bill initially had to make his own petri dishes by cutting the bottoms off glass vials), he demonstrated that sexual recombination in E. coli required an infectious factor that he called F, for fertility (Hayes 1968). Bill demonstrated that F was independent of the E. coli chromosome by studying the kinetics of its spread in a bacterial population. He found that F could replicate and spread though the population far faster than the 30 minute division time of the bacteria. This kind of autonomously replicating element came to be called a plasmid (Novick 1980).

A graph showing the spread of an antibiotic resistance plasmid in the absence of antibiotic is included in Watanabe's Scientific American article on transmissible antibiotic (Watanabe 1967). This result is significant because it shows how the mobility apparatus of the plasmid can operate to distribute the plasmid and make it spread through an unselected population of bacteria. Later, when antibiotic is applied, a large fraction of the population is already resistant.

In my recent blogs, a number of commentators asserted that the source of variation is immaterial to evolution because natural selection works on any variants that appear. But, as I pointed out in the first blog on superbugs, cells which can only modifying their existing genomes cannot achieve the same high levels of antibiotic resistance as cells that can pick up DNA from outside. Moreover, they are certainly not able to establish a resistant population prior to encountering selection as quickly as cells that have received a resistance plasmid.

Acquiring DNA from other cells, "horizontal transfer," occurs among bacteria most commonly by plasmids. However, bacterial cells can also take DNA up directly from the environment or receive it by viral infection. All these processes have been amply documented to occur in nature.

The F plasmid that Bill Hayes identified did not carry any antibiotic resistance sequences. So it is important to ask how those particular sequences came to be associated with transmissible plasmids. The answer leads us to two intriguing natural genetic engineering systems, transposons and integrons.

Transposons are segments of DNA that have the capacity to move (or "transpose") from one location in a genome to another (Cohen and Shapiro 1980). In order to make an antibiotic resistance sequence itself part of a transposon, it is sufficient to surround it by two copies of an existing transposon. This sometimes occurs normally as part of the transposition process, but other classes of transposons have been found already associated with antibiotic resistance sequences incorporated inside.

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James A. Shapiro: The Distinct Roles of Selection, Horizontal Transfer and Natural Genetic Engineering in Dangerous ...

MELBOURNE, AUSTRALIA--(Marketwire -08/30/12)- Genetic Technologies Limited (GTG.AX) (GENE) wishes to advise that it has filed a patent infringement suit in respect of its non-coding DNA technologies against Reproductive Genetics Institute Inc. in the US District Court, for the Northern District of Illinois, Eastern Division.

Further details will be released to the Market as appropriate.

About BREVAGenThe BREVAGen breast cancer risk stratification test is a novel genetic test panel that examines a patient's DNA to detect the absence or presence of certain common genetic variations (SNPs) associated with an increased risk for developing breast cancer. The test is designed to help physicians assess aggregate breast cancer risk from these genetic markers, plus factors from a standard clinical assessment based on a patient's family and personal history, thus giving a clearer picture of an individual woman's risk of developing breast cancer. The BREVAGen test may be especially useful for women predisposed to hormone dependant breast cancer, including those who have undergone breast biopsies, as the test will provide information that can help physicians recommend alternative courses of action, such as more vigilant, targeted surveillance or preventive therapy, on a personalized patient-by-patient basis. For more information, please visit http://www.brevagen.com, or http://www.brevagen.com.au

About Genetic Technologies LimitedGenetic Technologies is an established diagnostics company with more than 20 years of experience in commercializing genetic testing, non-coding DNA and product patenting. The Company has operations in Australia and the U.S. and is dual-listed on the ASX (GTG.AX) and NASDAQ (GENE). Genetic Technologies is focused on the commercialization of its patent portfolio through an active out-licensing program and the global expansion of its oncology and cancer management diagnostics assets. Its U.S. subsidiary, Phenogen Sciences, offers novel predictive testing and assessment tools to help physicians proactively manage women's health. Phenogen's lead product, BREVAGen, is a first in class, clinically validated risk assessment test for non-familial breast cancer. For more information, please visit http://www.gtglabs.com, http://www.phenogensciences.com.

Safe Harbor StatementAny statements in this press release that relate to the Company's expectations are forward-looking statements, within the meaning of the Private Securities Litigation Reform Act The Private Securities Litigation Reform Act of 1995 (PSLRA) implemented several significant substantive changes affecting certain cases brought under the federal securities laws, including changes related to pleading, discovery, liability, class representation and awards fees. Since this information may involve risks and uncertainties and are subject to change at any time, the Company's actual results may differ materially from expected results. Additional risks associated with Genetic Technologies' business can be found in its periodic filings with the SEC.

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Genetic Technologies Files Patent Infringement Suit Against Reproductive Genetics Institute Inc. in USA

SOUTH SAN FRANCISCO, Calif. and CAMBRIDGE, Mass., Aug. 29, 2012 /PRNewswire/ -- Veracyte, Inc., a molecular diagnostics company pioneering the emerging field of molecular cytology, and Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced that the New York State Department of Health has issued a license enabling Veracyte's Afirma Gene Expression Classifier to be offered to patients in the state. The companies also announced that Memorial Sloan-Kettering Cancer Center will become one of the first medical institutions in the state to offer patients the genomic test, which helps resolve inconclusive thyroid nodule results following traditional evaluation of fine needle aspiration (FNA) samples.

"We are delighted that the Afirma Gene Expression Classifier will now be available to physicians and their patients in New York State and that Memorial Sloan-Kettering is among the first in the state to offer our test," said Bonnie Anderson, Veracyte's cofounder and chief executive officer. "These milestones underscore the clinical need for and strength of the clinical data behind our test. Our goal is to help identify patients whose thyroid nodules are actually benign so that they may avoid unnecessary, invasive surgery."

Thyroid cancer is the fastest-increasing cancer in the United States, with 56,460 new cases expected in 2012, according to the American Cancer Society. Approximately 450,000 thyroid nodule FNAs a minimally invasive procedure to extract cells for examination under the microscope are performed each year in the U.S. to rule out cancer. Up to 30% of the time, the results are inconclusive, and current protocols typically recommend thyroid surgery for final diagnosis. Following surgery, however, 70-80% of patients turn out to have benign nodules.

The Afirma Gene Expression Classifier measures the expression of 142 genes to reclassify ambiguous thyroid FNA samples as either benign or suspicious for cancer. A clinical validation study, published recently in the New England Journal of Medicine, showed that when applied to the major categories of indeterminate thyroid samples, the test reclassified the samples as benign with greater than 94% accuracy.

The Afirma Gene Expression Classifier is offered as part of Veracyte's comprehensive Afirma Thyroid FNA Analysis, which combines specialized cytopathology assessment for initial review of thyroid nodule FNAs, with the gene expression test used to clarify inconclusive results. The test is now covered for Medicare patients nationwide and is available throughout the U.S. through a global co-promotion partnership with Genzyme, a Sanofi company and one of the world's leading biotechnology companies.

"We look forward to bringing to New York endocrinologists and thyroid patients a complete solution, which includes the Afirma Thyroid FNA Analysis for thyroid nodule assessment and Thyrogen for the management of patients diagnosed with thyroid cancer," said Alicia Secor, Genzyme's vice president and general manager of Endocrinology.

Genzyme is an established leader in endocrinology globally, developing and marketing Thyrogen (thyrotropin alfa for injection) for patients with well-differentiated thyroid cancer. Thyrogen is used as an adjunctive diagnostic tool for serum thyroglobulin (Tg) testing with or without radioiodine imaging in the follow up of patients with well-differentiated thyroid cancer. Thyrogen is also approved in the U.S. and Europe as an adjunctive treatment for radioiodine ablation of thyroid tissue remnants in patients who have undergone a near total or total thyroidectomy for well-differentiated thyroid cancer and who do not have evidence of metastatic thyroid cancer.

About Veracyte

Veracyte, Inc., based in South San Francisco, Calif., is pioneering the emerging field of molecular cytology, applying molecular biomarkers to cytology samples in order to improve disease diagnosis by clarifying indeterminate results obtained from current methods. The company aims to enable doctors to make more informed treatment decisions early, thus improving patient care and providing cost savings to the healthcare system. The company utilizes rigorous science and an extensive, multicenter clinical program throughout discovery and development. Veracyte's first product the Afirma Thyroid FNA Analysis combines specialized cytopathology assessment with the Afirma Gene Expression Classifier, a genomic test that clarifies inconclusive thyroid nodule results as benign or suspicious for cancer. The company has formed a global co-promotion partnership with Genzyme, a Sanofi company, to make the Afirma Thyroid FNA Analysis available throughout the U.S. and, subsequently, globally. Veracyte is currently in the early biomarker discovery phase for lung cancer and interstitial lung diseases. Veracyte is privately held and funded by Domain Associates, Kleiner Perkins Caufield & Byers, TPG Biotech and Versant Ventures. For more information, visit http://www.veracyte.com.

About Genzyme, a Sanofi Company

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Veracyte Receives New York State License for Afirma® Gene Expression Classifier

Researchers have identified a gene in women that fuels happiness, Medical News Today reported.

Low expression of the gene MAOA (monoamine oxidase A) was associated with increased happiness in adult females, according to a study from the University of South Florida, Columbia University and the New York State Psychiatry Institute. However, the research found the gene did not have the same effects in men.

Lead author, Dr. Henian Chen wrote he was surprised by the results.

[L]ow expression of MAOA has been related to some negative outcomes like alcoholism, aggressiveness and anti-social behavior, Chen wrote. Its even called the warrior gene by some scientists; but, at least for women, our study points to a brighter side of this gene.

Chen and his colleagues analyzed the DNA of 193 women and 152 men, cross-checking the results with the participants self-reported happiness scores. Overall, women with low expression of MAOA reported much more happiness than those with no expression.

According to Medical News Today, MAOA works similarly to antidepressants by helping to break down neurotransmitters such as serotonin and dopamine often called the feel good chemicals.

While females often report more anxiety and mood disorders than men, they also tend to experience more happiness as well. Chen said this gene may explain that paradox.

Click for more from Medical News Today.

Continued here:
'Happiness' gene discovered in women- ABLOW: 'Heroin-like' drug may beat depression

ScienceDaily (Aug. 28, 2012) A new study has found a gene that appears to make women happy, but it doesn't work for men. The finding may help explain why women are often happier than men, the research team said.

Scientists at the University of South Florida (USF), the National Institutes of Health (NIH), Columbia University and the New York State Psychiatric Institute reported that the low-expression form of the gene monoamine oxidase A (MAOA) is associated with higher self-reported happiness in women. No such association was found in men.

The findings appear online in the journal Progress in Neuro-Psychopharmacology & Biological Psychiatry.

"This is the first happiness gene for women," said lead author Henian Chen, MD, PhD, associate professor in the Department of Epidemiology and Biostatistics, USF College of Public Health.

"I was surprised by the result, because low expression of MAOA has been related to some negative outcomes like alcoholism, aggressiveness and antisocial behavior," said Chen, who directs the Biostatistics Core at the USF Health Morsani College of Medicine's Clinical and Translational Sciences Institute. "It's even called the warrior gene by some scientists, but, at least for women, our study points to a brighter side of this gene."

While they experience higher rates of mood and anxiety disorders, women tend to report greater overall life happiness than do men. The reason for this remains unclear, Chen said. "This new finding may help us to explain the gender difference and provide more insight into the link between specific genes and human happiness."

The MAOA gene regulates the activity of an enzyme that breaks down serontin, dopamine and other neurotransmitters in the brain -- the same "feel-good" chemicals targeted by many antidepressants. The low-expression version of the MAOA gene promotes higher levels of monoamine, which allows larger amounts of these neurotransmitters to stay in the brain and boost mood.

The researchers analyzed data from a population-based sample of 345 individuals -- 193 women and 152 men -- participating in Children in the Community, a longitudinal mental health study. The DNA of study subjects had been analyzed for MAOA gene variation and their self-reported happiness was scored by a widely used and validated scale.

After controlling for various factors, ranging from age and education to income, the researchers found that women with the low-expression type of MAOA were significantly happier than others. Compared to women with no copies of the low-expression version of the MAOA gene, women with one copy scored higher on the happiness scale and those with two copies increased their score even more.

While a substantial number of men carried a copy of the "happy" version of the MAOA gene, they reported no more happiness than those without it.

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Gene that predicts happiness in women discovered

ScienceDaily (Aug. 27, 2012) Researchers at Uppsala University, Swedish University of Agricultural Sciences and their international collaborators have discovered a mutation in a single gene in horses that is critical for the ability to perform ambling gaits, for pacing and that has a major effect on performance in harness racing. Experiments on this gene in mice have led to fundamental new knowledge about the neural circuits that control leg movements. The study is a breakthrough for our understanding of spinal cord neuronal circuitry and its control of locomotion in vertebrates.

The study is being published August 29 in Nature.

Our ability to walk and run is dependent on a complex coordination of muscle contractions carried out by neuronal circuits in our spinal cord. But how does this work at the level of nerve cells and molecules? The researchers took advantage of the variability in the pattern of locomotion in horses. The three naturally occurring gaits in horses are, in order of increasing speed, walk, trot and canter/gallop. Some horses are able to perform ambling gaits and/or pace, for instance, Icelandic Horses can tlt (an ambling gait) and perform flying pace. The researchers decided to investigate the genetic basis explaining why some Icelandic Horses can pace but others cannot.

"We suspected a strong genetic component, but were almost shocked when we discovered that a single gene, DMRT3, largely explained the genetic difference between pacers and non-pacers," explains Lisa Andersson one of the PhD students involved in the project.

Independently, Klas Kullander's research group had discovered that this particular gene, DMRT3, is expressed in a previously unknown type of neurons in the spinal cord of mice. The characteristics of these neurons, including their location, suggested that they could take part in neuronal circuits coordinating movements. When the two research groups, both associated with Science for Life Laboratory in Uppsala, compared their data, they realized that an important biological finding was imminent.

"At that moment, we realized that our discovery did not only extend our understanding of spinal neuronal circuits in mouse, but that we had discovered a tangible population of nerve cells that also seemed to be critical for the control of gaits in horses. The new type of nerve cell is dependent on DMRT3, and is tentatively named after this gene," tells Klas Kullander.

The researchers demonstrated that a single base change in DMRT3, which resulted in the production of a truncated form of the DMRT3 protein, was the mutation associated with pacing in horses. They developed a diagnostic test for the mutation and discovered that it is widespread among horses that show alternate gaits like Tennessee Walking Horse from the USA and Paso Fino from South America. Moreover, to their surprise, the mutation is very common in horses bred for harness racing.

"The DMRT3 mutation shows a strong positive association with performance in harness racing, states Leif Andersson who led the hunt for the DMRT3 mutation."

As a horse increases its speed it will normally switch from trot to gallop, which is the natural gait at high speed, but this leads to disqualification for trotters.

"Our interpretation is that the mutation inhibits the transition from trot to gallop and thereby allows the horse to trot at very high speed, explains Leif Andersson."

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Single gene has major impact on gaits in horses and in mice

Public release date: 29-Aug-2012 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, August 29, 2012--Biomass recalcitrance--the problem of how to break down complex plant-based cellulosic feedstock into sugars that can be fermented to produce sustainable biofuels and other renewable biobased productscan be overcome through improved methods of biomass characterization. IB IN-DEPTH, a collection of articles from leading research laboratories describing advanced tools and techniques for analyzing the chemistry, structure, and interaction of biomass components, is published in Industrial Biotechnology, a peer-reviewed journal from Mary Ann Liebert, Inc. The articles are available free online at the Industrial Biotechnology website.

The future capability to commercialize large-scale, economical, plant-based biofuels and bioproducts depends on the development of efficient and effective strategies to break down lignocellulosic biomass and to release the carbohydrates that can then be converted into these valuable end-products. Substantial progress is being made in solving the problems of biomass recalcitrance, and Guest Editor Brian Davison, PhD, Chief Scientist for Systems Biology and Biotechnology at Oak Ridge National Laboratory, Oak Ridge, TN, and Science Coordinator for the BioEnergy Science Center of the Department of Energy's Office of Biological and Environmental Research, and a member of the Editorial Board of Industrial Biotechnology, gathered leading researchers to share their work and perspectives.

The special research section includes two Reviews: "Biomass Characterization: Recent Progress in Understanding Biomass Recalcitrance" by Marcus Foston and Arthur Ragauskas, BioEnergy Science Center, School of Chemistry and Biochemistry, Institute of Paper Science and Technology, Georgia Institute of Technology, Atlanta, GA; and "Neutron Technologies for Bioenergy Research" by Paul Langan and colleagues, Oak Ridge National Laboratory, University of Tennessee, Knoxville, and Georgia Institute of Technology. Also featured are Short Communications and Methods articles that present new or improved methods of biomass characterization, including strategies based on biomass accessibility to enzymes, glycomics, polysaccharide changes in plant cell walls, improvements to the Simon's stain technique, an updated method of mechanical stress testing, and a modification of atomic force microscopy.

"Much thanks to Dr. Brian Davison for pulling together this special issue of Industrial Biotechnology," says Larry Walker, PhD, Co-Editor-in-Chief and Professor, Biological & Environmental Engineering, Cornell University, Ithaca, NY. "The development of methods and approaches for characterizing biomass materials is an important step in driving biotechnology development from plant engineering to subsequent conversion to biofuels and bioproducts."

###

About the Journal

Industrial Biotechnology, led by Co-Editors-in-Chief Larry Walker, PhD, and Glenn Nedwin, PhD, MBA, is an authoritative journal focused on biobased industrial and environmental products and processes, published bimonthly in print and online. The Journal reports on the science, business, and policy developments of the emerging global bioeconomy, including biobased production of energy and fuels, chemicals, materials, and consumer goods. The articles published include critically reviewed original research in all related sciences (biology, biochemistry, chemical and process engineering, agriculture), in addition to expert commentary on current policy, funding, markets, business, legal issues, and science trends. Industrial Biotechnology offers the premier forum bridging basic research and R&D with later-stage commercialization for sustainable biobased industrial and environmental applications.

About the Publisher

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Biomass characterization technology research highlighted in Industrial Biotechnology journal

PESEUX, Switzerland--(BUSINESSWIRE)-- Pulmonx, an emerging leader in interventional pulmonology, announced today that its European notified body has granted CE approval for revised labeling of its Zephyr Endobronchial Valve (EBV). The approval was based upon an independent review of two separate clinical data sets in which patients with hyperinflation associated with a genetic form of emphysema, Alpha-1 Antitrypsin Deficiency (AATD), were treated using the Pulmonx Zephyr EBV.

These new data sets reported improvements for FEV1 (forced expiratory volume in one second) and Residual Volume in AATD patients treated with Zephyr endobronchial valves, showing that patients with hyperinflation associated with emphysema can benefit from EBV treatment, independent of the underlying cause of the disease. No new procedural or device risks that may be associated with the treatment of AATD patients with the Pulmonx EBV were observed. The newly approved labeling provides a therapeutic option for physicians to use the Zephyr EBV to treat patients afflicted with hyperinflation associated with Alpha-1 Antitrypsin Deficiency.

We have seen great success in the use of endobronchial valve therapy in selected patients suffering from Alpha-1 Antitrypsin Deficiency, said Gunnar Hillerdal, Associate Professor of Lung Medicine, Karolinska University Hospital, Stockholm, Sweden. The treatment options for these patients were previously very limited but their anatomy and the typical distribution of their disease can make them excellent candidates for EBV treatment. The recent label change should encourage pulmonologists to evaluate these patients for such treatment with confidence, he continued.

About Alpha-1

Alpha-1 antitrypsin deficiency (AATD) is an inherited condition in which the body does not make enough of a protein that protects the lungs and liver from damage. Severe AATD can lead to emphysema or chronic obstructive pulmonary disease (COPD) in adult life.

About Pulmonx

Pulmonx, based in Redwood City, CA, and Peseux, Switzerland, is focused on developing and marketing minimally invasive medical devices and technologies for the diagnosis and treatment of pulmonary disorders. The Chartis System and Zephyr EBV are the first diagnostic and therapeutic solution to the problem of emphysema-induced hyperinflation. The Pulmonx Zephyr EBV and Chartis System are the subject of numerous peer-reviewed studies, and the Zephyr EBV has already been used to treat thousands of patients worldwide. http://www.pulmonx.com

The Zephyr EBV is an investigational device in the United States. Limited by U.S. law to investigational use. The Chartis System is for use/sale outside the United States only.

Link:
Pulmonx to Market Zephyr® EBV Therapy for Patients with “Genetic” Emphysema

LOUISVILLE, Ky.--(BUSINESS WIRE)--

SureGene has chosen PGXL as laboratory provider for its proprietary STA2R genetic panel, which promises to revolutionize the treatment of schizophrenia.

The STA2R panel turns intuitive treatment into precision treatment, says Dr. Roland Valdes Jr., Chairman and President of PGXL. It removes the trial and error from the medication of schizophrenia. Its a perfect application of personalized medicine entirely aligned with PGXLs vision.

The STA2R agreement marks the first collaboration between SureGene and PGXL, two companies that spun out of University of Louisville research labs. SureGene researchers discovered and patented the SULT4A1-1 genetic signature. PGXL Laboratories independently developed the STA2R panel and will perform the tests for healthcare providers around the United States. Both SureGene and PGXL are promoting the test, PGXL through its own distribution system and SureGene direct to psychiatric healthcare providers.

PGXL has long been a leader and innovator in personalized medicine. When SureGene needed a lab partner, PGXL was the obvious choice. The synergies from both companies being located in Louisville was an added bonus, says Bill Massey, President of SureGene.

The panel analyzes five genes, including SureGenes patented SULT4A1, and uses the results of those tests to help identify the right treatment path for a patient based on available data. More than 100,000 Americans are diagnosed with schizophrenia every year. While most will eventually be successfully treated, about a third will never find the right balance of medications. The STA2R panel is designed to help guide psychiatrists to that balance quickly and confidently.

Every time I meet with patients and caregivers, they share heart-wrenching stories of their journey to finding the right medicine, says Tim Ramsey, Vice President of SureGene and one of the inventors of STA2R. With STA2R, SureGene and PGXL are giving doctors a powerful new tool to help their patients.

About PGXL Laboratories:

A privately-owned business located in Louisville, Kentucky, PGXL Laboratories was the first lab in the country CLIA-certified specifically to conduct pharmacogenetic tests. It offers pharmacogenetic testing, interpretive services, and assay design and validation. Along with its clinical practice, PGXL performs contract research for developers of pharmaceuticals and medical equipment.

About SureGene:

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PGXL to Provide Antipsychotic Drug Response Tests

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/49mg4f/personalized_medic) has announced the addition of the "Personalized Medicine - A Strategic Analysis of Industry Trends, Technologies, Participants and Environment" report to their offering.

Personalized Medicine- A Strategic Analysis of Industry Trends, Technologies, Participants, and Environment by Kelly Scientific Publications is a comprehensive assessment of this developing industry thus far. This report tackles the growing market interest in personalized medicine (PM), pharmacogenomics, companion diagnostics and the associated market environment.

Individualized or personalized medicine aims to increase the efficacy of therapeutics via genetic testing and companion diagnostics. As we progress through the era of genomic medicine, patients will benefit by more effective therapies and less side effects. Developmental and diagnostic companies will benefit from lower discovery and commercialization costs and more specific market subtypes.

The average drug-to-market cost varies but is estimated at $500 million, however with the advent of personalized therapeutics and companion diagnostics this cost could be dramatically reduced. Only 30% of drugs recover the cost to market however personalized therapeutics and associated companion diagnostics will be more specific and effective thereby giving pharma/biotech companies a significant advantage to recuperate costs. Personalized medicine will reduce the frequency of adverse drug reactions and therefore have a dramatic impact on health economics.

Companies Mentioned:

- 23andMe

- Affymetrix

- Astex Pharmaceuticals

- Atossa Genetics

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Research and Markets: Personalized Medicine - A Strategic Analysis of Industry Trends, Technologies, Participants and ...

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/vxph8f/quickfact_edition) has announced the addition of the "QUICKFACT Edition Personalized Medicine - A Strategic Analysis of Industry Trends, Technologies, Participants and Environment" report to their offering.

The QUICKFACT's edition of Personalized Medicine- A Strategic Analysis of Industry Trends, Technologies, Participants, and Environment by Kelly Scientific Publications is a compact report on key facts of the personalized medicine industry and its impact on the health system.

Individualized or personalized medicine aims to increase the efficacy of therapeutics via genetic testing and companion diagnostics. Personalized therapeutics and associated companion diagnostics will be more specific and effective thereby giving pharma/biotech companies a significant advantage to recuperate R&D costs. Personalized medicine will reduce the frequency of adverse drug reactions and therefore have a dramatic impact on health economics. Developmental and diagnostic companies will benefit from lower discovery and commercialization costs and more specific market subtypes.

The average drug-to-market cost varies but is estimated at $500 million, however with the advent of personalized therapeutics and companion diagnostics this cost could be dramatically reduced. Only 30% of drugs recover the cost to market however personalized therapeutics and associated companion diagnostics will be more specific and effective thereby giving pharma/biotech companies a significant advantage to recuperate costs. Personalized medicine will reduce the frequency of adverse drug reactions and therefore have a dramatic impact on health economics.

Functions of personalized medicine:

- Give developers an alternative to hit and miss' drug development.

- Streamline the research and discovery process.

- Decrease the time it takes to discover, develop and bring a therapeutic to market.

- Can be applied to current drugs and determine their efficiency and safety profiles in specific patient groups.

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Research and Markets: 2012 QUICKFACT Edition Personalized Medicine - A Strategic Analysis of Industry Trends ...

Rhesus monkeys on calorie-restricted diets age just as quickly as their chubbier counterparts.

E. Bmsch/Imagebroker/FLPA

To those who enjoy the pleasures of the dining table, the news may come as a relief: drastically cutting back on calories does not seem to lengthen lifespan in primates.

The verdict, from a 25-year study in rhesus monkeys fed 30% less than control animals, represents another setback for the notion that a simple, diet-triggered switch can slow ageing. Instead, the findings, published this week in Nature1, suggest that genetics and dietary composition matter more for longevity than a simple calorie count.

To think that a simple decrease in calories caused such a widespread change, that was remarkable, says Don Ingram, a gerontologist at Louisiana State University in Baton Rouge, who designed the study almost three decades ago while at the National Institute on Aging (NIA) in Bethesda, Maryland.

When the NIA-funded monkey study began, however, studies of caloric restriction in short-lived animals were hinting at a connection. Experiments had showed that starvation made roundworms live longer. Other studies had showed that rats fed fewer calories than their slow and balding brethren maintained their shiny coats and a youthful vigour. And more recently, molecular studies had suggested that caloric restriction or compounds that mimicked it might trigger a cascade of changes in gene expression that had the net effect of slowing ageing.

In 2009, another study2, which began in 1989 at the Wisconsin National Primate Research Center (WNPRC) in Madison, concluded that caloric restriction did extend life in rhesus monkeys. The investigators found that 13% of the dieting group died from age-related causes, compared with 37% of the control group.

One reason for that difference could be that the WNPRC monkeys were fed an unhealthy diet, which made the calorie-restricted monkeys seem healthier by comparison simply because they ate less of it. The WNPRC monkeys diets contained 28.5% sucrose, compared with 3.9% sucrose at the NIA. Meanwhile, the NIA meals included fish oil and antioxidants, whereas the WNPRC meals did not. Rick Weindruch, a gerontologist at the WNPRC who led the study, admits: Overall, our diet was probably not as healthy.

Further, the WNPRC control group probably ate more overall, because their meals were unlimited, whereas NIA monkeys were fed fixed amounts. As adults, control monkeys in the WNPRC study weighed more than their NIA counterparts. Overall, the WNPRC results might have reflected an unhealthy control group rather than a long-lived treatment group. When we began these studies, the dogma was that a calorie is a calorie, Ingram says. I think its clear that the types of calories the monkeys ate made a profound difference.

When we began these studies, the dogma was that a calorie is a calorie.

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Calorie restriction falters in the long run

Otago Uni. Genetics Lecture Outdated and Unfair

Otago University's genetics lecture presented last night in Wellington, and to be presented in Auckland tonight, is outdated and unfair.

The "Should We Swallow It" lecture by John Knight supports GE food but is wrong to be confusing traditional modification of food with the very different processes of genetic engineering.(1)

It is also inappropriate and adds to the lack of fairness in the lecture, that it features video of a live TV debate that was found to constitute a breach of broadcasting standards.

The lecture pushing a pro-GE agenda uses a clip from Holmes aired on TVNZ, that was found to lack balance by the Broadcasting Standards Authority.(2) The lecture also presents research that is out-dated and misleading about the growing evidence against GE release into the environment.

"The research is confused and a waste of public money," says Claire Bleakley from GE-Free NZ in food and environment.

One major problem that makes the findings deceptive is the failure to differentiate between natural selection (GM) and genetic engineering (GE) in the questions used.

Research questions need to be accurate and reflect participants understanding of what is asked, or they become misleading.

The data being presented is useless as input for Government decsion-making, and betrays a lack of understanding of the subject by the researcher.

"John Knight seems to be out of his depth and to have been seduced into a false understanding of GE. He knows little about the difference between natural selection of plant breeding and laboratory transgenics (GE) of plants," says Claire Bleakley.

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Otago Uni. Genetics Lecture Outdated and Unfair

SEATTLE, WA--(Marketwire -08/29/12)- Matrix Genetics ("Matrix"), a biotechnology company focused on producing renewable fuels and specialty chemicals derived from cyanobacteria (blue-green algae) announced today that Spokane, Wash. based Avista Development, Inc. has invested in the company. Avista Development is the venture arm of Avista Corp. (AVA), an energy company involved in the production, transmission and distribution of energy as well as other energy-related businesses. The investment provides Matrix with working capital needed to complete its spinout from Seattle-based Targeted Growth, an agricultural biotechnology company where the foundation of Matrix's technology was developed.

"Avista's investment is the springboard for Matrix to become an independent company with the resources to further develop our technologies that are creating a pathway to low-carbon, sustainable and renewable fuels and chemicals," said Margaret McCormick, CEO of Matrix Genetics. "Their support will enable us to add staff, expand our labs, and continue the great tradition of bioscience companies in the state of Washington."

Matrix is developing technologies to leverage the potential of cyanobacteria as a feedstock for the production of a rich diversity of valuable carbon-based chemicals. Cyanobacteria are the most abundant, diverse and robust micro-algae on Earth, using only the energy from sunlight to convert atmospheric carbon dioxide directly into fuels and other biological chemicals. They are also relatively simple, single cell organisms; the genome (the cell's DNA) has already been mapped for several cyanobacteria species; and there is a robust set of "tools" available to modify them.

Matrix has used these tools and the immense power of biotechnology to create new and proprietary strains of cyanobacteria that can produce oil in significant quantities and in a range of specifications. Second and third generation organisms are being developed that not only surpass these oil yields, but also contain additional new traits that enhance their production characteristics and make them suitable for a range of end products including fuels, chemicals and other products.

"Avista has a long history of fostering innovation within the energy sector," said Roger Woodworth, Vice President and Chief Strategy Officer at Avista Corp. "We appreciate the need to find alternatives to petroleum for a sustainable future, and we are excited by the progress and the promise of Matrix's approach."

As the company completes its spinout, Matrix is now focused on further developing its technology to: produce lipids (oils) for fuels and other products; develop production strains that are suitable for different growing environments, resistant to predators and harmless to the environment around them; create strains that continually produce and secrete oils, removing the need for costly harvesting; and increase the cultivation capacity to provide samples for testing with commercialization partners and prospects.

The announcement will be made today at a special event at Matrix Genetics' current lab facilities as part of the "Summer of Algae II," a national campaign sponsored by the Algae Biomass Organization, the trade association for the U.S. algae industry, which features similar open house-style events across the country.

About Matrix GeneticsMatrix Genetics, LLC ("Matrix"), located in Seattle, Wash., is a biotechnology company focused on producing renewable fuel and specialty chemicals derived from cyanobacteria (blue-green algae). The company's state-of-the-art, metabolic engineering and systems biology platform is the most cost-efficient method to customize organisms with a range of traits for these industries. More information is available at http://www.matrixgenetics.com.

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Biotech Firm Matrix Genetics Receives Investment From Avista Development

Public release date: 28-Aug-2012 [ | E-mail | Share ]

Contact: Anne DeLotto Baier abaier@health.usf.edu 813-974-3303 University of South Florida (USF Health)

Tampa, FL (Aug. 28, 2012) -- A new study has found a gene that appears to make women happy, but it doesn't work for men. The finding may help explain why women are often happier than men, the research team said.

Scientists at the University of South Florida (USF), the National Institutes of Health (NIH), Columbia University and the New York State Psychiatric Institute reported that the low activity form of the gene monoamine oxidase A (MAOA) is associated with higher self-reported happiness in women. No such association was found in men.

The findings appear online in the journal Progress in Neuro-Psychopharmacology & Biological Psychiatry.

"This is the first happiness gene for women," said lead author Henian Chen, MD, PhD, associate professor in the Department of Epidemiology and Biostatistics, USF College of Public Health.

"I was surprised by the result, because low expression of MAOA has been related to some negative outcomes like alcoholism, aggressiveness and antisocial behavior," said Chen, who directs the Biostatistics Core at the USF Health Morsani College of Medicine's Clinical and Translational Sciences Institute. "It's even called the warrior gene by some scientists, but, at least for women, our study points to a brighter side of this gene."

While they experience higher rates of mood and anxiety disorders, women tend to report greater overall life happiness than do men. The reason for this remains unclear, Chen said. "This new finding may help us to explain the gender difference and provide more insight into the link between specific genes and human happiness."

The MAOA gene regulates the activity of an enzyme that breaks down serontin, dopamine and other neurotransmitters in the brain -- the same "feel-good" chemicals targeted by many antidepressants. The low-expression version of the MAOA gene promotes higher levels of monoamine, which allows larger amounts of these neurotransmitters to stay in the brain and boost mood.

The researchers analyzed data from a population-based sample of 345 individuals 193 women and 152 men participating in Children in the Community, a longitudinal mental health study. The DNA of study subjects had been analyzed for MAOA gene variation and their self-reported happiness was scored by a widely used and validated scale.

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Study finds gene that predicts happiness in women

SAN JOSE, Calif., Aug. 27, 2012 /PRNewswire/ --A study published in The Journal of Maternal-Fetal and Neonatal Medicine demonstrates that the fraction of fetal cell-free DNA (cfDNA) in maternal blood is unaffected by the mother's presumed risk for trisomy, offering support for the use of non-invasive prenatal testing (NIPT) for detecting genetic conditions such as Down syndrome in a broad patient population. Lead and senior authors of the study were Dr. Herb Brar, Director of Riverside Perinatal Diagnostics Center, and Dr. Mary Norton, Professor of Obstetrics and Gynecology, Lucile Packard Children's Hospital at Stanford University, respectively.

(Photo: http://photos.prnewswire.com/prnh/20120827/NE62428LOGO )

Results of the study, a post-hoc comparative analysis of the previously published "Non-invasive Chromosomal Evaluation" (NICE) study, showed that there were no significant differences in the fraction of fetal cfDNA in maternal blood in women who were stratified into three different trisomy risk groups based on maternal age, prenatal screening results or nuchal translucency measurement. The amount of fetal cfDNA in maternal blood is the principal factor in successfully detecting trisomies with NIPT. Trisomy refers to the presence of three chromosomes rather than two. Certain trisomies are known to cause genetic conditions. The study is available at: http://informahealthcare.com/doi/abs/10.3109%2F14767058.2012.722731

"The results of this study were particularly significant because they showed that fetal fraction of cfDNA does not vary significantly among pregnant women regardless of their predetermined trisomy risk," said Dr. Herb Brar. "This adds to the growing amount of research that suggests NIPT can offer an effective prenatal screening option in the general pregnant population."

NIPT is a new screening option that analyzes cell-free fetal DNA circulating in maternal blood to evaluate the risk of having a baby with Down syndrome and other common genetic conditions. It involves a single blood draw as early as 10 weeks' gestation and delivers a greater than 99 percent detection rate for trisomy 21, which causes Down syndrome. NIPT, using the Harmony Prenatal Test, also has shown to have fewer false positive test results; up to 50 times less than traditional prenatal screening options such as maternal serum screening.

Previous studies of the entire cohort of the NICE study demonstrated that fetal fraction did not vary with race, ethnicity, maternal age, or trisomy type. It also showed that the fraction of fetal cfDNA was similar in pregnancies of gestational ages between 10 and 22 weeks. The NICE study was a prospective cohort study of more than 4,000 pregnant women of at least 10 weeks' gestational age across 50 clinical sites internationally. The study evaluated the performance of Ariosa Diagnostics' Harmony Prenatal Test in detecting fetal trisomy 21 and 18, which cause Down syndrome and Edwards syndrome, respectively. The NICE study evaluated any patient undergoing invasive prenatal testing, such as chorionic villus sampling (CVS) or amniocentesis, not just those who were determined to be at higher risk of having a baby with a genetic condition.

"We believe strongly that scientific research supports NIPT as a screening option for any pregnant woman, empowering them and their physicians to make the best decisions for individual circumstances. This is simply good patient care," said Ariosa Diagnostics CEO Ken Song, M.D. "NIPT has proven effective in thousands of patients, with a high accuracy and low false positive rate of less than 0.1% for each trisomy tested. It offers several advantages over traditional screening tests and can more appropriately triage those women who should undergo invasive procedures, namely amniocentesis and CVS, which carry a small risk of miscarriage."

About Ariosa Diagnostics (formerly Aria Diagnostics)

Ariosa Diagnostics, Inc. is a molecular diagnostics company committed to providing safe, highly accurate and affordable prenatal tests for maternal and fetal health. Led by an experienced team, Ariosa is using its proprietary technology to perform a directed analysis of cell-free DNA in blood. Ariosa's simple blood test equips pregnant women and their healthcare providers with reliable information to make decisions regarding their health, without creating unnecessary stress or anxiety.

The company began operations in 2010 and is headquartered in San Jose, Calif. For more information, visitwww.ariosadx.com.

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New Study in The Journal of Maternal-Fetal and Neonatal Medicine Shows Fetal Cell-Free DNA in Maternal Blood ...

SAN DIEGO, Aug.28, 2012 /PRNewswire/ -- Sequenom, Inc. (SQNM), a life sciences company providing innovative diagnostic testing and genetic analysis solutions, today announced that its wholly-owned subsidiary, the Sequenom Center for Molecular Medicine (Sequenom CMM), has completed several international distribution agreements that will expand access to the MaterniT21 PLUS testing service outside the United States. Agreements in Asia cover Japan and Hong Kong; in Europe cover the Czech Republic and Slovakia, and the Netherlands; and in the Middle East, cover Israel.

The announcement of these agreements follows recent news that Sequenom's current licensee in Europe, LifeCodexx, gained CE Marking for their trisomy 21 test, the PraenaTest, using the Sequenom-licensed technology and has made it available as a testing service at prenatal clinics and hospitals in Germany, Austria, Liechtenstein and Switzerland.

"We believe the completion of these international agreements represents an important advance in access to and future adoption of our technology in the international prenatal care market," said Harry F. Hixson, Jr., Ph.D., Chairman and CEO, Sequenom, Inc. "We look forward to working closely with each of our partners in these countries and to continuing our efforts to further expand access for expectant parents around the world."

The distribution agreements further support total volume increases and goals for the company for 2012. As of August 18, 2012, Sequenom CMM had accessioned nearly 27,000 MaterniT21 PLUS test samples since the beginning of the year, and the company increased its 52-week run rate from 65,000 to 70,000 total samples. The company has also completed its previously announced sales force expansion, with more than 70 field representatives now active across the United States.

Sequenom CMM has also completed a number of steps designed to improve functionality and enhance capacity of the MaterniT21 PLUS test. The test now includes a report on the presence of "Y" chromosomal material, which was validated in the same original clinical cohort as trisomies 21, 18 and13. Other new features of the MaterniT21 PLUS test include increased multiplexing - the new 12-plex process should more than double sequencing capacity per instrument - and the use of upgraded bioinformatics and automated library preparation processes. To ensure maintenance of the high precision of the MaterniT21 PLUS test, all modifications have been rigorously validated in an equivalency study. In addition, Sequenom CMM now employs new reagents made available through Sequenom's agreement with Illumina, all contributing to a more streamlined, efficient testing process.

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. Estimates suggest there are an estimated 750,000 pregnancies at risk for fetal aneuploidy each year in the United States. The test detects an increased amount of chromosomal material for trisomies 21, 18 and 13, as well as fetal sex. The MaterniT21 PLUS test is available exclusively through Sequenom CMM as a testing service to physicians. To learn more about the test, please visit Sequenomcmm.com.

About Sequenom

Sequenom, Inc. (SQNM) 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.

Sequenom CMM, LLC

Sequenom 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.

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Sequenom Completes International Distribution Agreements To Expand Access To MaterniT21 PLUS Prenatal Testing Service

Calcutta News.Net Monday 27th August, 2012

LONDON - The Medical Research Council (MRC) will be providing Edinburgh University with 60 million pounds of funding over the next five years to enable scientists carry out genetic research with the aim of learning more about conditions like schizophrenia, cystic fibrosis and genetic eye disorders.

The funding is being provided to the MRC Human Genetics Unit and the MRC Institute of Genetics and Molecular Medicine (IGMM) at the University of Edinburgh.

It is hoped the research will also help doctors at the IGMM, one of the largest centres of its kind in Europe, develop new tests and therapies for patients with cancer and osteoarthritis.

Director Professor Nick Hastie said, "The challenge we face is to work out how human genes work together to build a human. We also want to find out how subtle DNA differences help shape human diversity and influence susceptibility to a wide range of common diseases."

The IGMM is a partnership between the MRC, University of Edinburgh's Centre for Molecular Medicine and Cancer Research UK.

The funding will help IGMM "to turn the potential of the genetic revolution into reality", stated Hastie.

The funding, to be made over the next five years, will confirm the centre's position as a world leader in genetics research.

Dr Wendy Ewart of the MRC, said: "The Human Genetics Unit and IGMM are a shining example of the kind of partnership working needed to address the challenges of 21st century research. The MRC is proud to continue its support for these establishments and their drive to transform discoveries about the human genome into benefits for human health."

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Genetic research department of Endinburg University to receive 60mn pounds

The technique would retrain cells that typically don't respond to light.

Kiji McCafferty

One biotech startup wants to restore vision in blind patients with a gene therapy that gives light sensitivity to neurons that don't normally possess it.

The attempt, by Ann Arbor, Michigan-based Retrosense Therapeutics, will use so-called optogenetics. Scientists have used the technique over the last few years as a research tool to study brain circuits and the neural control of behavior by directing neuron activity with flashes of light. But Retrosense and others groups are pushing to bring the technique to patients in clinical trials.

The idea behind Retrosense's experimental therapy is to use optogenetics to treat patients who have lost their vision due to retinal degenerative diseases such as retinitis pigmentosa. Patients with retinitis pigmentosa experience progressive and irreversible vision loss because the rods and cones of their eyes die due to an inherited condition. If the company is successful, the treatment could also help patients with the most common form of macular degeneration, which affects nearly a million people in the United States. The U.S. Food and Drug Administration hasn't approved any therapies for either condition.

Retrosense is developing a treatment in which other cells in the retina could take the place of the rods and cones, cells which convert light into electrical signals. The company is targeting a group of neurons in the eye called ganglion cells. Normally, ganglion cells don't respond to light. Instead, they act as a conduit for electrical information sent from the retina's rods and cones. The ganglion cells then transmit visual information directly to the brain.

Doctors would inject a non-disease causing virus into a patient's eye. The virus would carry the genetic information needed to produce the light-sensitive channel proteins in the ganglion cells. Normally, rods, cones, and other cells translate light information into a code of neuron-firing patterns that is then transmitted via the ganglion cells into the brain. Since Retrosense's therapy would bypass that information processing, it may require the brain to learn how to interpret the signals.

So far, Retrosense and its academic collaborators have shown that the treatment can restore some vision-evoked behaviors in rodents. The treatment also seems safe in nonhuman primates. The optogenetically modified ganglion cells of these primates are light-responsive, but behavioral tests aren't possible, as there are no nonhuman primate models of retinal degeneration, says Retrosense CEO Sean Ainsworth.

Retrosense plans to begin its first clinical trial in 2013 with nine blind retinitis pigmentosa patients.

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Company Aims to Cure Blindness with Optogenetics

Millions of people around the world are living with HIV, thanks to drug regimens that suppress the virus. Now there's a new push to eliminate HIV from patients' bodies altogether. That would be a true cure.

We're not there yet. But a report in Science Translational Medicine is an encouraging signpost that scientists may be headed in the right direction.

Forty-three patients got immune cells designed to attack and kill cells infected with HIV. As long as 16 years later, these genetically engineered T cells are still circulating in their bloodstreams. And there's been no sign the gene therapy caused any cancers, or is likely to.

That may seem like a modest victory. After all, there's no evidence yet that the gene therapy did what it's supposed to eliminate the reservoir of HIV hiding in the patients' cells, waiting to emerge as soon as patients stop taking their antiviral drugs.

But to scientists in HIV and gene therapy research, it's a highly encouraging indicator. "We're not hitting a home run. This is a single," AIDS researcher Pablo Tebas of the University of Pennsylvania tells Shots.

"It looks like if you do this, it's going to be safe because we have not seen any toxicity in 16 years," he says. "And two, the genetically modified cells are still circulating. They perpetuate. Those are two important things this study is telling us."

Tebas is not an author of the study, but he works with the Penn researchers who did the work. They were unavailable for comment.

Previous attempts at this kind of gene therapy, called adoptive T cell transfer, have been plagued by cancers that can arise when the genes introduced into engineered cells insert themselves next to growth-promoting genes. In other cases the engineered cells have died out before they have a sustained positive effect.

Another hopeful sign that engineered T cells can actually work came from the same Penn group last summer. They reported on a single patient with advanced chronic lymphocytic leukemia who had failed a succession of chemotherapy treatments.

The Penn researchers injected the patient with some of his own T cells that had been engineered to home in on leukemia cells, which bear a distinctive surface protein. Within a month, doctors could find no leukemia cells in the patient's bone marrow.

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A Step Forward For Gene Therapy To Treat HIV

Japanese scientists from the NIMS International Center for Materials Nanoarchitectonics (MANA) have developed a nanostructured sheets film capable of introducing designated genes into animal cells. The scientists also demonstrated the safety and efficacy of the new nanosheet film as a substrate for reverse gene transfection.

The methods of introducing genes into cells can be performed in liquids (solution-based) or on the surface of a solid substrate (solid phase gene transfection). In the solid phase gene transfection, DNA is fixed on the solid surface and then cells adhere on the DNA-bearing surface. The objective of the present research is to explore new solid substrates for the reverse gene transfection. This solid-mediated transfection has attracted attention due to the higher delivery efficiency of DNA than liquid phase transfection method. Different types of DNA are possible to arrange on a solid surface and introduce into cells. This technology is also effective in systematic analysis and profiling of the effects of genes.

Until now, an extracellular matrix called fibronectin, which is an animal-derived protein, or other similar substances, had been used as an accelerant in solid phase gene transfection. However, this approach had been considered problematic in clinical application situations, where the gene transferred cells are returned to the patient's body. Thus, the use of animal-derived substances has a serious concern from the viewpoint of safety, etc.

In the present research, the MANA researchers prepared a nanosheets film through a near-infinite number of nanoscale walls protruding from the surface. The film is composed of only inorganic silica without any animal sources. The MANA team found that genes can be introduced into cells with extremely high efficiency when fixing DNA on the nanostructured silica film and contacting with cells. Since an animal-derived supplements is not necessary, this should be a safe and simple solid phase transfection system.

This research result is applicable to gene therapy and offers a revolutionary gene introduction method. It is expected to make a valued contribution to gene therapy for hereditary diseases such as diseases of inborn error of metabolism, hemophilia, etc., and for intractable diseases such as diabetes and the like.

More information: pubs.rsc.org/en/Content/ArticleLanding/2012/CC/c2cc34289h

Provided by National Institute for Materials Science

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Development of nanosheets film has potential for safe, effective gene transfection into cells