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The PMO is talking the GMO language but people will fight it out

India, July 29 -- Four major committees of the Government of India, including the Parliamentary Standing Committee on Agriculture, have called for a moratorium on testing Genetically Modified (GM) food. Now, we also have the Technical Expert Committee (TEC) report submitted to the Supreme Court which is strong in its recommendations. Both democracy and science support a ban on GMOs.

Earlier, Jairam Ramesh as environment minister had decided to stop the testing of Bt Brinjal. Ramesh was compelled, after the series of public hearings which he thankfully attended. Why? It was because he realised that the government's position was not backed by science; instead, science was on the side of those who called for it to not be introduced.

What we know from science is that the complexity of the DNA and gene has not been completely understood. Genetic engineering is based on the orthodox genetic reductionism, genetic determinism and the false assumption that one gene contributes to one trait. Science teaches us that a trait has contributions from many genes and the context in which the organism is. Disciplines like gene ecology and epigenetics have made genetic reductionism obsolete. Yet, the GM industry and pseudo-scientists are pushing ahead with genetic modification.

Every claim that the GMO industry made in the last 20 years has proved to be false. The first was that GM foods will be a solution to hunger and give us food security. Nothing could be farther from the truth. Vidarbha's Bt Cotton story proves this. Monsanto had promised 1500 kg per acre when it started trials; the yield today is 400 kg per acre. The second was that pesticide use will reduce; there has been a 13-fold rise, the bollworm became resistant to pesticides and new pests emerged.

Even in the US, the use of herbicides has gone up by 15 per cent with GM crops. Pests have become super-pests, weeds have become super-weeds. There's evidence that GMOs are killing beneficial species both in the soil and our gut. Finally, it's seed monopoly. GMOs mean patents, patents mean royalties. That's why GMOs are being pushed. It's a fact that 95 per cent of cotton seed in India is now owned by Monsanto.

On purely scientific basis, India should not have GMOs or GM foods but there seem to be extraneous reasons.

I would say that the PMO is bulldozing us, as a nation, into doing what's not backed by evidence and what should never be done. The PMO is pushing the GMO agenda, especially GM foods, because it's a commitment he made, under the Indo-US Agriculture Knowledge Initiative, to the US government. So, the PMO is talking the language of the GMO industry.

There are others like Sharad Pawar who pushes for GMO even though he has been totally countered by data. He is too close to the GM lobby, to Monsanto, to take an independent view. There are industry-led lobby groups who are paid to peddle the industry's version. And, the government does not listen to independent voices and look at scientific evidence.

On no count is genetic engineering delivering on its promises. GMO technology has proved to be inefficient compared to all available alternatives. If we allow this technology to wipe out our biodiversity, farmers, food security, society, it would be a crime.

Now that Monsanto and Blackwater, a private military, have joined forces it's even more dangerous. They will use all undemocratic means to mobilise the market here. The BRAI Bill is an example. They have made seed their weapon of control.

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The PMO is talking the GMO language but people will fight it out

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Genetic Engineering: A New Use for Algae in Biofuel Production

Lawrence, KS (PRWEB) July 29, 2013

Phycologia In recent years major companies from all over the globe, including Exxon Mobile and Itochu Corporation, have merged with research facilities to research algae as a renewable energy source. However, it has been determined that far more research is needed before movement to the commercial production phase of algae biofuel can truly take place. A recent research study took up this challenge and examined a promising freshwater algal strain for possible genetic engineering applications that could make it a viable biofuel.

An article in the journal Phycologia takes an in-depth look at the genetic structure of a unicellular green alga, Botryococcus braunii, and explores its unique ability to be utilized in the genetic engineering of biofuel development. Botryococcus braunii was initially selected for large-scale biofuel production because of its extraordinary ability to synthesize large amounts of hydrocarbon oils. Several difficulties were encountered in the initial production and harvesting processes, leaving it by the wayside. However, this latest research reintroduces B. braunii as the perfect vehicle for genetic engineering applications when compared with three other species of green algae, five species of land plants, and eight other phyla species, including bacteria, archaea, fungi, and mammals.

The research focused on the codon usage, or DNA compatibility, of B. braunii with the other organisms. Codon usage for this particular alga is one of the fundamental genetic markers that had not been explored. Codons are greatly affected by the vast amount of guanines (G) and cytosines (C), two of the four nucleotides that make up a DNA molecule. Many green algal species having high GC content, which causes codon usage bias, or poor compatibility, with other organisms. Surprisingly, B. braunii had comparatively low GC content and its codon usage was similar to that of bacteria, mammals, and land plants.

Although further study is necessary, the ability of B. braunii to synthesize hydrocarbons, combined with the newly discovered codon usage and GC content data, could lead to new genetic engineering techniques that could hasten biofuel development and production.

Full text of the article, Codon usage of Botryococcus braunii (Trebouxiophyceae, Chlorophyta): implications for genetic engineering applications, Phycologia, Vol. 52, No. 4, 2013, is available at http://www.phycologia.org/doi/full/10.2216/12-041.1

About Phycologia Phycologia is the official publication of the International Phycological Society and publishes papers on any aspect of algal research. For more information, visit http://www.intphycsoc.org.

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Genetic Engineering: A New Use for Algae in Biofuel Production

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We’re Crazy Close To A Cure For Allergies – Video


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Unraveling genetic networks

Public release date: 29-Jul-2013 [ | E-mail | Share ]

Contact: Jason Socrates Bardi jbardi@aip.org 240-535-4954 American Institute of Physics

WASHINGTON D.C., July 29, 2013 -- If genes are the currency of life, then the whole economies are genetic networks, which include genes as well as the complex webs of interactions and interconnections between them. Genetic networks are integrally important to the proper development and functioning of an organism, just as genes are, but they tend to be far more complex and difficult to understand.

Because of their complexity, the field has been slow to unravel genetic networks, said Leon Glass, the Isadore Rosenfeld Chair in Cardiology and a professor of physiology at McGill University.

Now a special issue of the journal CHAOS, produced by AIP Publishing, explores new experimental and theoretical techniques for unraveling genetic networks.

"Most emphasis has been on the properties of individual genes, and mutations in individual genes have been identified that lead to diseases, such as the cystic fibrosis gene," said Glass. But the proper expression of individual genes is regulated by both environmental factors, metabolic factors and the expression of other genes in the body, he added.

"To understand these interactions," Glass said, "it is essential to consider genetic networks."

Because of the convoluted interconnectedness of gene networks, researchers have realized that they might best be understood using nonlinear dynamics -- an analytical method to understand systems (such as the weather) in which simple changes can have complex, cascading and even chaotic effects.

Just such methods are the subject of the special issue of CHAOS. Included among the collection of articles are two papers describing powerful new methods to understand very large genetic networks and a paper combining experimental and theoretical work to unravel the genetic networks controlling development in fruit flies.

"The rapid expansion of biologic data concerning structure and dynamics of genetic networks makes it essential to develop appropriate computer and analytical methods to deal with these problems," said Glass, who was an early pioneer in the development of theoretical methods for understanding gene network models. Glass is the author of the introduction to the new issue along with James Collins, an HHMI Investigator and a bioengineering professor at Boston University and Harvard's Wyss Institute, and Rka Albert, a professor of physics and biology at Pennsylvania State University.

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Evolution of diverse sex-determining mechanisms in mammals

Public release date: 29-Jul-2013 [ | E-mail | Share ]

Contact: Amanda Petrak amanda.petrak@case.edu 216-368-0345 Case Western Reserve University

Scientists historically have argued that evolution proceeds through gradual development of traits. But how can incremental changes apply to the binary switch between two sexes, male or female? Researchers at Case Western Reserve University's School of Medicine have found that a genetic process among the many species of rodents could have significant implications regarding our assumptions about sex determination and the pace of evolution.

"What we addressed is a long-standing puzzle in natural history: why different types of rodents can exhibit profound differences in how male sex is determined in the embryo," said Michael Weiss, MD, PhD, chairman of the Department of Biochemistry, the Cowan-Blum Professor of Cancer Research and a professor of biochemistry and medicine. "Some rodent populations have both XY males and XY females, and in other populations the Y chromosome has disappeared entirely."

In a study published in Proceedings of the National Academy of Sciences, Weiss and his research team analyzed the Sry gene, which is part of the Y chromosome. This mammalian gene, which steers differentiation in the embryonic gonad toward the development of testes, begins the process leading to the birth of males. For most mammals, including primates, Sry is a conserved feature of the Y chromosome, ultimately giving rise to male anatomy; females generally have two X chromosomes and no Y.

But within anomalous families of rodents, common in South America, activation of the Sry gene may have uncertain consequences. Some of these groups have both XY males and XY females as normal components of the population. Other related species have even lost their Y chromosomes altogether. Without the emergence of compensating ways of specifying sex, the species could not produce malesand would become extinct. For such rodents, therefore, evolution meant inventing entirely different methods of sex determination. These mammals have in essence evolved other ways to play nature's mating game.

The CWRU team attributed the rapid evolvability of sex determination in rodents to a novel protein domain added to the SRY protein. Scientists knew that this domain existed, but Weiss and his team wanted to understand more about its function in gene regulation and its role in evolution. The team determined that the new protein domain acts as a "genetic capacitor," providing a protective buffer to the Sry gene. This buffer allowed male development even when a mutation occurs elsewhere in the gene that might otherwise cause sex reversalbut the buffer is unstable over generations. Slippage of DNA during the production of sperm can lead to sudden changes in the length of the buffer and the degree of protection. By analogy to a capacitor in an electric circuit, the team suggested that this domain can "discharge" to accelerate the pace of evolutionary change. The idea of a genetic capacitor was pioneered by MIT Professor Susan Lindquist in studies of heat-shock proteins in fruit flies in (Nature, "Hsp90 as a capacitor for morphological evolution") and the present paper extended this idea to the pace of mammalian evolution.

How did the Sry buffer arise? "We discovered that a genetic accident 20 million years ago in an ancestral rodent holds the key to solving this puzzle. A simple DNA repeat sequence (called a 'micro-satellite') invaded the Y chromosome and was incorporated into the Sry gene. This invasion accelerated the evolvability of Sry and probably the Y chromosome in general, enabling this subgroup of rodents to explore new molecular mechanisms of sex determination," Weiss said.

Weiss and his team will continue this research, but believe these initial results may have additional implications for our understanding of human evolution and genetics. Because rodents have higher mutation rates and shorter life spans, they also evolve more rapidly and so provide a natural laboratory for studies of mammalian evolution.

Research last year at MIT has shown that in humans and other primates the Y chromosome has been stable for at least the past 25 million years (Nature, Strict evolutionary conservation followed rapid gene loss on human and rhesus Y chromosomes), which Weiss suggests may reflect the absence of micro-satellite-related slippage in the Sry gene. Yet the transcriptional strengths of the murine and human Sry factors are similar. The research suggests that human SRY and its specification of male development has evolved to be just above a genetic threshold of activity, which may in turn enable human communities to benefit from a diversity of male characteristics and behaviors.

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Evolution of diverse sex-determining mechanisms in mammals

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Parents don't fully understand biobank research, study finds

Public release date: 29-Jul-2013 [ | E-mail | Share ]

Contact: Gina Bericchia Gina.Bericchia@NationwideChildrens.org 614-355-0495 Nationwide Children's Hospital

Researchers who collect genetic samples from children for medical research need to explain the process more clearly to parents, according to a new study that suggests many parents don't fully understand the finer details about how these samples will be used and stored. The study was published in June in Genetics in Medicine.

Kim McBride, MD, MS, principal investigator in the Center for Cardiovascular and Pulmonary Research in The Research Institute at Nationwide Children's Hospital, and colleagues followed up with families enrolled in a genetic biobanka storage facility for DNA, genetic data, and tissue samplesafter their initial consent, to find out whether they were fully aware of the nature of their agreement. The results were alarming: more than half of all parents misunderstood key concepts of the study.

Ensuring parental understanding of the consent process is a crucial element of ethical genetics research, says Dr. McBride. For the study, he and his colleagues collected anonymous questionnaires from families that had consented between 2004 and 2008 to the storage and analysis of their children's DNA samples in a genetic biobank. The biobank was created to study the genetics of congenital malformations of the heart's left ventricular outflow tract.

The researchers found that, while parents had a good grasp of some consent concepts, other important information was poorly understood. For example, parents understood that their consent was voluntary and the samples would be used for research on the causes of heart defects. However, they overestimated how the research might benefit their child and undervalued the risks of enrolling in the study.

"Although the primary purpose of the study was to obtain biologic samples for future testing, parents did not understand that their child's samples would be stored indefinitely," Dr. McBride says. When both parents were involved in the decision, they displayed a better overall understanding of the true nature of the consent agreement than parents making the decision alone.

Most families participating in research are involved with clinical trials, which involve a different set of expectations and agreements during the consent process than genetic sampling. "Participants in a clinical trial are enrolled in a treatment and are followed over time with the expectation that they may receive a new and better therapy," Dr. McBride says. In a biobank study, however, participants cannot typically expect any personal benefit or even any follow-up.

This is starting to change, however. Some new models for biobank studies are more inclusive of the research subject, offering on-going contact and return of results that may impact their health, says Dr. McBride, who also is an assistant professor in pediatrics at The Ohio State University College of Medicine. "To provide individuals and families with adequate knowledge to participate in genetic research, informed consent delivery must evolve, especially as the demand for genomic data increases."

Interventions to improve understandingoften incorporating visual aids and video consentingprovide promising results, but few studies demonstrate their effectiveness for biobanking consent. Furthermore, there is currently no movement to adopt these techniques widely, something Dr. McBride says warrants consideration. "The focus of researchers should shift to how to improve the informed consent process through alternative methods of consent delivery, so that consenting families are truly informed partners in genetic research."

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Genetic glitch potent player in many types of allergies

Washington, July 29 (ANI): Scientists have discovered that a faulty genetic pathway could be responsible for a range of allergies.

The research by the Johns Hopkins Children's Center and the Johns Hopkins Institute of Genetic Medicine has found that aberrant signalling by a protein called transforming growth factor-beta, or TGF-beta, may be responsible for disrupting the way immune cells respond to common foods and environmental allergens, leading to a wide range of allergic disorders.

It was found that mutations in the genes that lead to abnormal TGF-beta signaling are also keys to Marfan and Loeys-Dietz syndromes, genetic conditions marked by blood vessel laxity and dangerous stretching of the aorta, the body's largest blood vessel.

"Disruption in TGF-beta signalling does not simply nudge immune cells to misbehave but appears to singlehandedly unlock the very chain reaction that eventually leads to allergic disease," senior investigator Harry "Hal" Dietz, M.D. said.

The study involved 58 children with LDS, ages 7 to 20, followed at Johns Hopkins. Most of them had either a history of allergic disease or active allergies, like allergic rhinitis, eczema, food allergies, asthma, and gastrointestinal and esophageal allergic disease.

These patients also had abnormally high levels of several traditional markers of allergic disease, including IgE - an antibody that drives life-hreatening allergic responses - and high numbers of eosinophils, white blood cells involved in allergic reactions.

Because TGF-beta is known to control immune cell maturation, the researchers homed in on a group of cells known as regulatory T cells, which keep tabs on other immune cells to ensure that they don't go into overdrive.

To identify precisely how TGF-beta affects immune cell behavior, the researchers next obtained undifferentiated, or naive, immune cells from LDS patients. Immersed in TGF-beta, these "pre-specialized" cells quickly transformed into allergy-promoting immune cells known for their ability to recognize and attack pathogens, as well as otherwise innocent substances, like food proteins.

In a final, critical discovery, the researchers found that the immune cells of children with LDS had abnormally high levels of a protein called SMAD, a known transmitter of TGF-beta signaling.

The study was published on July 24 in journal Science Translational Medicine. (ANI)

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Genetics, Epigenetics, Biology and the Emotions. Chris Astill-Smith – Chapter 1 of 9 – Video


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Study unravels genetics behind debilitating inflammatory disease Takayasu arteritis

Public release date: 29-Jul-2013 [ | E-mail | Share ]

Contact: Beata Mostafavi bmostafa@umich.edu 734-764-2220 University of Michigan Health System

ANN ARBOR, Mich. Researchers have uncovered the genetics behind what makes some people susceptible to Takayasu arteritis, a debilitating disease that can lead to poor circulation, easy tiredness in the legs and arms, organ damage and stroke.

A study led by the University of Michigan has identified five genes tied to Takayasu arteritis, an inflammation that damages the aorta and can lead to narrowed arteries, aneurysms, high blood pressure, and heart failure. The findings appear in the August issue of The American Journal of Human Genetics.

"Discovering the genetic makeup of Takayasu arteritis is a pivotal step that will lead to fundamental understanding of the disease mechanisms and developing therapies to more effectively treat it," says senior author Amr Sawalha, M.D., associate professor of internal medicine in the division of rheumatology at the U-M Medical School. "This disease can be devastating but is understudied and poorly understood."

Takayasu arteritis mainly causes inflammation in the aorta the large artery that carries blood from the heart to body and other major blood vessels. This inflammation can also affect the heart valves, reduce blood flow to the legs and arms, and cause a stroke. Other symptoms include weight loss, fever, night sweats, fatigue and joint and muscle pain.

The disease is most common among women and typically occurs between the ages 20 and 40.

The new findings increase the number of genes linked to susceptibility to the disease to five risk areas both in the HLA (an inherited group of genes known as human leukocyte antigen) and outside the HLA. In addition to the previously established genetic association in HLA-B for Takayasu arteritis, researchers discovered and carefully localized novel genetic risk areas in HLA-DQB1/HLA-DRB1, FCGR2A/FCGR3A, and PSMG1.

"We have established and localized the genetic association with IL12B, which encodes the P40 subunit of the interleukin-12 (IL-12) and IL-23," says Gher Saruhan-Direskeneli, M.D., professor of physiology at Istanbul University and co-author of the study.

"Therapies to inhibit the IL12/IL23 pathway have been successful in other inflammatory diseases, and these recent findings support investigating this pathway closer in Takayasu arteritis as a potential therapeutic target," Sawalha adds.

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Study unravels genetics behind debilitating inflammatory disease Takayasu arteritis

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Oxford Genetics secures funding for DNA cloning system

(PRWEB UK) 29 July 2013

Venture capital fund manager Mercia Fund Management (MFM) has announced 150,000 of funding in Oxford Genetics Ltd. The new biotechnology company is behind SnapFast - a system that simplifies the purchase of synthetic DNA molecules (plasmids).

CEO of Oxford Genetics, Dr Ryan Cawood, invented the SnapFast system and likens it to Lego for DNA. Historically, most genetic engineering has been performed by combining pieces of DNA, gathered from a variety of sources that were never intended to fit together. This can make clinical research and drug development time-consuming, costly and often frustrating. Oxford Genetics provides all of the standard DNA components that researchers use on a regular basis in a compatible and easy to clone format.

The funding will be used for the companys e-commerce website, http://www.oxfordgenetics.com, and new product development. Currently Oxford Genetics provides human, mouse and bacterial DNA and through the funding will soon be able to offer insect, plant and yeast DNA.

Oxford Genetics is yet another company to benefit from SEIS (Seed Enterprise Investment Scheme) funding from the Mercia Growth Fund suite of funds (MFMs hybrid EIS/SEIS funds).

Dr Cawood commented on the companys decision to work with MFM: We were looking for a venture capital fund with experience of biotech start-ups and MFMs name kept coming up. The investment team has a good reputation in our industry sector and is already adding value.

Dr Mark Payton, Managing Director of MFM, added: Our team is actively identifying start-ups with early commercial traction and high growth potential. As part of the MFM SEIS investment portfolio, Oxford Genetics receives an injection of capital and support. The company clearly stood out for us - the SnapFast system and the online business model meet an untapped need in the market. Coupled with a strong product roadmap and an exceptional founder, we predict a promising future for this start-up.

The Mercia Growth Fund suite of funds, (hybrid funds offering EIS and SEIS investment capital), comprise approximately 4 million. Collectively with Mercia Growth Funds 1 and 2 (hybrid EIS and SEIS funds), MFM oversees arguably the largest SEIS discretionary funds in the UK.

About Mercia Fund Management Mercia Fund Management (MFM) is a venture capital fund manager with over 19 million under active management.

MFM provides investment growth capital for businesses ranging from early commercial traction through to those seeking expansion or working capital that are trading profitably. Furthermore, MFM can also provide cost-effective incubator accommodation at Forward House in Henley-in-Arden coupled with centralised support services in keeping with its model as a hands-on, supportive investor.

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Oxford Genetics secures funding for DNA cloning system

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Statement on New Dense Breast Notification Law in North Carolina From Atossa Genetics

SEATTLE, WA--(Marketwired - Jul 29, 2013) - Atossa Genetics, Inc. (NASDAQ: ATOS), The Breast Health Company, today announces its support for the fact that North Carolina has become the 12th state to pass a dense breast notification law, HB 467, the Breast Density Notification & Awareness bill. The bill was signed by Gov. Pat McRoy on July 23, 2013, and will become effective on January 1, 2014.

Dense breast notification legislation requires that women who are found through mammography to have dense breasts must be notified of this fact. Twelve states including Alabama, California, Connecticut, Hawaii, Maryland, Nevada, New York, North Carolina, Oregon, Tennessee, Texas and Virginia have enacted such legislation. Similar dense breast measures have been introduced or advanced in many other states. At the federal level, the Breast Density and Reporting Act of 2011 (HR 3102) was introduced in the previous Congress in October 2011 by Representative Rosa DeLauro (D-CT) and Representative Steve Israel (D-NY).

"Are You Dense Advocacy, Inc. and its supporters are working tirelessly on behalf of women to advance dense breast legislative initiatives at the state and federal levels," said Dr. Steven Quay, Chairman, CEO & President of Atossa Genetics and inventor of theForeCYTEBreast Health Test for breast cancer risk assessment. "This latest legislative victory is an important step forward in protecting women's health. The need for dense breast notification laws is strongly supported by data indicating that mammograms have greater difficulty spotting tumors in dense breast tissue and that women with dense breasts are at higher risk for breast cancer. We continue to support Are You Dense Advocacy and its efforts to enact dense breast notification laws in the remaining 38 states and at the national level."

In January 2013, Atossa launched the ForeCYTE Breast Health Test. This test analyzes cells from the linings of the milk ducts and lobules, where approximately 95 percent of breast cancers arise, in order to identify pre-cancerous changes that confer an increased risk of breast cancer. Atossa believes that its test is advantageous for women in general, and particularly for women with dense breasts, since dense breast tissue does not affect ForeCYTE's ability to collect and analyze cells from the breast.

"We believe that women ages 18 to 73, including women with dense breasts, should take the ForeCYTE Breast Health Test in order to gain valuable and potentially life-saving information about their breast health status. By identifying women at high risk for breast cancer through scientific means and intervening with lifestyle changes or therapeutic interventions, we believe the incidence of breast cancer can be significantly reduced, just as cervical cancer rates have fallen dramatically since the introduction of the Pap smear," Dr. Quay added.

A peer-reviewed study of 2,712 women age 25 to 65 years showed a strong association between increased breast density and cytological atypia of nipple aspirate fluid, the specimen collected by the ForeCYTE Test. The authors concluded, "Particularly in premenopausal women, the identification of women at increased risk of breast cancer might be enhanced using nipple aspiration." M M Lee, N L Petrakis, M R Wrensch, et al. "Association of abnormal nipple aspirate cytology and mammographic pattern and density." Cancer Epidemiol Biomarkers Prev 3:33-36.

Are You Dense Advocacy, Inc. is the government relations affiliate of Are You Dense, Inc., an independent, national breast health advocacy organization dedicated to informing the public about dense breast tissue and its significance for the early detection of breast cancer. Are You Dense, Inc. was founded by Nancy M. Capello, Ph.D., who was diagnosed with advanced stage breast cancer in February 2004 after receiving "normal" mammography reports for a decade.

For additional information on breast cancer risk, including the risks associated with dense breasts, please visit the Breast Cancer Overview page on the American Cancer Society's website at http://www.cancer.org/cancer/breastcancer/overviewguide/.

About the ForeCYTE Breast Health Test

The ForeCYTE Breast Health Test, intended for the 110 million women in the U.S. ages 18 to 73, is a painless, quick and non-invasive procedure that can be done in a physician's office. A small sample of fluid, aspirated from the nipple of each breast with the Company's modified breast pump, can provide vital early detection of cancer or pre-cancerous conditions that may progress to cancer over an approximately eight year period and before cancer can be detected by mammography or other means and without the risks of radiation, especially in women younger than age 50. No invasive biopsy needles or open surgical incisions are used in the Atossa test and the test is painless.

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Statement on New Dense Breast Notification Law in North Carolina From Atossa Genetics

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uniQure Announces Start of Parkinson's Disease Gene Therapy Phase I Trial by UCSF/NIH to Test Its GDNF Gene in Patients

AMSTERDAM, THE NETHERLANDS--(Marketwired - Jul 29, 2013) - uniQure B.V., a leader in human gene therapy, today announced the start of a human trial in Parkinson's disease with glial cell line-derived neurotrophic factor (GDNF). The gene therapy developed by researchers at the University of California, San Francisco (UCSF), uses the GDNF gene, which uniQure licensed from Amgen. This trial is part of a wide-ranging collaboration between uniQure and UCSF and represents one of uniQure's suite of GDNF-based gene therapies in development targeting disorders of the central nervous system.

uniQure and UCSF's joint program is an investigator initiated clinical trial in Parkinson's disease, led by Dr. Krystof Bankiewicz MD, PhD, at UCSF, and Dr. John D. Heiss, MD, at the National Institute for Neurological Disorders and Stroke, part of the National Institutes of Health (NIH). Using the ClearPoint Neuro Interventional System, Dr. Bankiewicz's team administered the GDNF gene packaged in an AAV vector to the brain of a first Parkinson's patient. The ClearPoint system has been developed to allow real-time observation of brain surgery, allowing unmatched precision of gene therapy delivery. The hypothesis is that GDNF's neuro-regenerative and protective properties may protect and strengthen brain cells that produce dopamine. Dopamine is a chemical that affects brain function. People with Parkinson's disease have problems producing dopamine in a specific area in the brain responsible for the control of body movements. The study is a Phase I open-label dose escalation safety study that will include 24 patients divided over 4 cohorts. The first patient was dosed on May 20, and so far there have been no safety issues. The study is fully funded by the NIH.

"The start of this trial is the culmination of over 10 years of preclinical work performed at UCSF," says Dr. Krys Bankiewicz of UCSF. "We are extremely proud to have optimized the delivery technologies together with our partners so that we can now safely deliver a gene therapy product into a minuscule space in the brain, including the putamen, observe the surgery in real-time and guarantee the highest possible level of quality and precision of gene delivery. uniQure is our partner of choice for the development of the therapy as they are first in having mastered the vector design, as well as the development and manufacturing challenges gene therapy developers faced in the past."

Under the terms of the collaboration, uniQure will have the rights to the results of the study as well as the IND. In exchange uniQure will manufacture the GDNF-AAV construct using its baculovirus platform for the next phases of the study.

In addition, uniQure is collaborating on a range of GDNF-based CNS disorders, such as multiple system atrophy (MSA), Huntington's disease, and hearing loss together with Universit de Toulouse (France), University of Cambridge (UK), and UCSF. Using AAV5, which has a strong tropism for the CNS, the partners will evaluate these programs up to preclinical proof of concept. As part of this work, uniQure and UCSF are currently developing administration of AAV5 to the cerebrospinal fluid to facilitate broad distribution of substance in the brain.

uniQure has appointed Dr. Bankiewicz chair of its CNS Scientific Advisory Board. Dr. Bankiewicz is the Kinetics Foundation Chair in Translational Research and Professor in Residence of Neurological Surgery and Neurology at UCSF. His research focus is restorative interventions, specifically drug delivery and gene transfer, in models of Parkinson's disease and brain tumors. His laboratory studies delivery of therapeutic agents into the central nervous system through cell-implantation, convection-enhanced and gene transfer-based delivery methods, as well as in vivo applications of MRI and PET to detect in-vivo changes in the brain and their correlation with functional outcome.

"Success in CNS gene therapy is to a large extent dependent on cutting edge delivery technologies. Capitalizing on the unique and broad collaboration with UCSF, in particular the outstanding group in Dr. Bankiewicz' laboratory, we are accessing and developing leading CNS gene delivery technologies. This Parkinson trial also demonstrates the potential of the Company's gene therapy technologies to reach beyond orphan diseases. The modular approach of our manufacturing platform and the use of AAV5 will allow us to rapidly advance a portfolio of promising programs targeting CNS disorders," said Jrn Aldag, CEO of uniQure.

About uniQure uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. Our approach is validated by multiple partnerships and the regulatory approval of our lead product Glybera. http://www.uniqure.com.

DisclaimerThis press release contains forward-looking statements based on uniQure's current expectations. These forward-looking statements include statements regarding the development of a suite of CNS therapies, partnerships in the CNS area, and the development of additional gene therapies. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties regarding further regulatory requirements, the success of further clinical trials, and competitive pressures. uniQure assumes no responsibility to update such forward-looking statements.

Press release (PDF): http://hugin.info/157414/R/1719343/572213.pdf

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uniQure Announces Start of Parkinson's Disease Gene Therapy Phase I Trial by UCSF/NIH to Test Its GDNF Gene in Patients

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BrainStorm’s NurOwn Cell Therapy Receives Orphan Drug Designation in the European Union for ALS

NEW YORK, NY and PETAH TIKVA, ISRAEL--(Marketwired - Jul 29, 2013) - BrainStorm Cell Therapeutics (OTCQB: BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, today announced that the European Commission has granted Orphan Drug Designation for NurOwn, the Company's stem cell therapy consisting of autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors, for the treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's Disease.NurOwn received Orphan Drug Designation from the Food and Drug Administration (FDA) in 2011.

Orphan drugs benefit from 10 years market exclusivity in the European Union (EU) after marketing approval. Additional benefits for sponsor companies include reduced fees for various centralized activities including applications for marketing authorization, inspections and protocol assistance, as well as possible eligibility for EU grants and other R&D-supporting initiatives.

BrainStorm is currently conducting a Phase IIa dose-escalating trial with 12 ALS patients at the Hadassah Medical Center in Jerusalem, Israel. The company anticipates launching a Phase II multi-center trial at three leading institutions in the United States towards the end of 2013, pending FDA approval.

About BrainStorm Cell Therapeutics, Inc.BrainStorm Cell Therapeutics Inc. is a biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. The Company holds the rights to develop and commercialize its NurOwn technology through an exclusive, worldwide licensing agreement with Ramot, the technology transfer company of Tel Aviv University. For more information, visit the company's website at http://www.brainstorm-cell.com.

Safe Harbor Statement - Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements.The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov.These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements.The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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