Archive for the ‘Gene Therapy Research’ Category

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

Contact: Phyllis Edelman pedelman@genetics-gsa.org 301-634-7302 Genetics Society of America

BETHESDA, MD October 2, 2012 -- Cancerous tumors contain hundreds of mutations, and finding these mutations that result in uncontrollable cell growth is like finding the proverbial needle in a haystack. As difficult as this task is, it's exactly what a team of scientists from Cornell University, the University of North Carolina, and Memorial Sloan-Kettering Cancer Center in New York have done for one type of breast cancer. In a report appearing in the journal GENETICS, researchers show that mutations in a gene called NF1 are prevalent in more than one-fourth of all noninheritable or spontaneous breast cancers.

In mice, NF1 mutations are associated with hyper-activation of a known cancer-driving protein called Ras. While researchers have found earlier evidence that NF1 plays a role in other types of cancer, this latest finding implicates it in breast cancer. This suggests that the drugs that inhibit Ras activity might prove useful against breast cancers with NF1 mutations.

"As we enter the era of personalized medicine, genomic technologies will be able to determine the molecular causes of a woman's breast cancer," said John Schimenti, Ph.D., a researcher involved in the work from the Center for Vertebrate Genomics at Cornell University College of Veterinary Medicine in Ithaca, New York. "Our results indicate that attention should be paid to NF1 status in breast cancer patients, and that drug treatment be adjusted accordingly both to reduce the cancer and to avoid less effective treatments."

To make this discovery, scientists analyzed the genome of mammary tumors that arise in a mouse strain prone to genetic instability -- whose activity closely resembles the activity in human breast cancer cells -- looking for common mutations that drive tumors. The gene NF1 was missing in 59 out of 60 tumors, with most missing both copies. NF1 is a suppressor of the oncogene Ras, and Ras activity was extremely elevated in these tumors as a consequence of the missing NF1 gene. Researchers then examined The Cancer Genome Atlas (TCGA) data, finding that NF1 was missing in more than 25 percent of all human breast cancers, and this was associated with a decrease in NF1 gene product levels, which in turn is known to increase Ras activity.

"This research is compelling because it helps us understand why some breast cancers are more likely to respond to only certain types of treatment," said Mark Johnston, Editor-in-Chief of the journal GENETICS. "The findings reported in this article may guide clinicians to better treatments specific to the needs of each patient."

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FUNDING: This study was supported by NIH training grants IT32HDO57854 and 5T32GM007617 that supported M.D.W.; Empire State Stem Cell Fund contract numbers C026442 and C024174 to J.C.S.; and C.M.P. and A.D.P. were supported by NCI Breast SPORE program (P50-CA58223-09A1), by U24-CA143848, and by the Breast Cancer Research Foundation.

CITATION: Marsha D. Wallace, Adam D. Pfefferle, Lishuang Shen, Adrian J. McNairn, Ethan G. Cerami, Barbara L. Fallon, Vera D. Rinaldi, Teresa L. Southard, Charles M. Perou, and John C. Schimenti Comparative Oncogenomics Implicates the Neurofibromin 1 Gene (NF1) as a Breast Cancer Driver Genetics October 2012 192:385-396

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Gene responsible for many spontaneous breast cancers identified

ScienceDaily (Oct. 2, 2012) A research team at Aarhus University reveals a surprising interplay between the ends of human genes: If a protein-coding gene is too short it becomes inactive! The findings also explain how some short genes have adapted to circumvent this handicap.

Gene ends communicate

Human genomes harbour thousands of genes, each of which gives rise to proteins when it is active. But which inherent features of a gene determine its activity? Postdoctoral Scholar Pia Kjlhede Andersen and Senior Researcher Sren Lykke-Andersen from the Danish National Research Foundation's Centre for mRNP Biogenesis and Metabolism have now found that the distance between the gene start, termed the 'promoter', and the gene end, the 'terminator', is crucial for the activity of a protein-coding gene. If the distance is too short, the gene is transcriptionally repressed and the output is therefore severely decreased. This finding outlines a completely new functional interplay between gene ends.

Small genes utilise specialised terminators

Fortunately, most human protein-coding genes are long and are therefore not repressed by this mechanism. However, some genes, e.g. 'replication-dependent histone genes', are very short. How do such genes express their information at all? Interestingly, many of these differ from the longer protein-coding genes by containing specialised terminators. And in fact, if such a specialised terminator replaces a normal terminator in a short gene context, the short gene is no longer transcriptionally repressed. It therefore appears that naturally occurring short genes have evolved 'their own' terminators to achieve high expression levels.

The new findings add to a complex molecular network of intragenic communication and help us to understand the basic function of genes.

The researchers behind the results that have just been published in the international journal Genes & Development are affiliated with the Danish National Research Foundation's Centre for mRNP Biogenesis and Metabolism at the Department of Molecular Biology and Genetics, Aarhus University.

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Length matters in gene expression

Sequencing, splicing and expressing DNA may seem to be the quintessence of cutting-edge scienceindeed DNA manipulation has revolutionized fields such as biofuels, chemicals and medicine. But in fact, the actual process can still be tedious and labor-intensive, something Lawrence Berkeley National Laboratory (Berkeley Lab) scientist Nathan Hillson learned the hard way.

After struggling for two days to design a protocol to put together a genetic circuit with 10 pieces of DNAusing a spreadsheet as his primary toolhe was dismayed to discover that an outside company could have done the whole thing, including parts and labor, for lower cost than him ordering the oligonucleotides himself. "I learned two things: one, I never wanted to go through that process again, and two, it's extremely important to do the cost-effectiveness calculation," said Hillson, a biochemist who also directs the synthetic biology program at the Berkeley Lab-led Joint BioEnergy Institute (JBEI). "So that was the genesis of the j5 software. This is the perfect thing to teach a computer to do."

The j5 software package, which has attracted users from more than 250 institutions worldwide since it was made available last year, is now the basis for the latest startup to emerge from JBEI, a Department of Energy research center established in 2007 to pursue breakthroughs in the production of cellulosic biofuels. By building on j5 and adding modules for commercial users, TeselaGen Biotechnology, founded by Hillson and two partners, says it will significantly reduce the time and cost involved with DNA synthesis and cloning, a multibillion-dollar market.

"It's like AutoCAD for biology," said TeselaGen co-founder and CEO Mike Fero. "Modern cloning is a computational problem. We are the missing informatic piece to making modern scarless DNA assembly methods a reality for the majority of biologists. Otherwise it's a small cadre of people who can do it."

Recombinant DNA assemblies are critical tools in a number of scientific pursuits: for understanding how cells are altered in diseases such as cancer, for building better antibiotics, for converting plant biomass to biofuels and for basic scientific understanding of cellular pathways. Standard cloning techniques have been in use for 40 years and are still the industry standard.

"Our biggest competition is traditional cloning, or inertia," said Fero, who was a particle physicist for 10 years before pursuing a career in biotech. "We have to make it so easy people will happily switch to the newer methods."

TeselaGen licensed j5 from Berkeley Lab, the lead institutional partner of JBEI, and currently has more than 100 scientists and engineers from large industrial and pharmaceutical companies in private beta.

"We are so pleased with the startup of TeselaGen, based on a deceptively simple idea but clearly providing a solution to a very difficult problem," said Cheryl Fragiadakis, director of technology transfer at Berkeley Lab. "It is a great example as well of a company coming out of our JBEI activities.The Lab's Tech Transfer encourages and supports entrepreneurial ventures, providing education and networking for our scientists, as a great way to get technologies out for the benefit of society."

The cloud-based software not only designs DNA construction protocols, it will compare methods to determine the one that is most cost-effective, weighing, for example, if it is cheaper to outsource a portion of the DNA construction.The more complex the task, the more time and money the program can save. The greatest savings are with combinatorial libraries, collections of hundreds to millions of related DNA assemblies, each with a different combination of genes or parts that perform similar functions in different organisms.

For example, simple construction of a metabolic pathway that takes two and a half weeks and costs $1,400 using traditional cloning can be cut down to two weeks and less than half the cost with j5. A more complex task of constructing a combinatorial protein library (with 243 constructs) would drop from $122,000 and 11 months with traditional cloning to under $30,000 and 1.5 months with j5. The same task using direct DNA synthesis would cost $538,000 and take 2.3 months.

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DNA construction technology makes genetic engineering cheaper, faster

Allergen-free cows milk and pigs with hardened arteries illustrate how the accuracy of genetic engineering has improved.

Two unsuspecting farm animals have helped to demonstrate the increasing accuracy of genetic engineering techniques. The first is a cow that produced hypoallergenic milk after researchers used RNA interference to block the production of an allergy-inducing protein, as reported this week (October 2) in PNAS. The second, reported in another paper in the same issue, is a pig that could be a model for atherosclerosis after researchers used an enzyme called a TALEN to silence a gene that helps to remove cholesterol.

Researchers have long struggled to remove cow milks allergy-inducing protein, beta-lactoglobulin, which can cause diarrhoea and vomiting in children. They were previously unable to introduce foreign genes precisely enough, however, so they could never quite successfully replace the gene that codes for beta-lactoglobulin with a defective form.

But scientists at AgResearch in Hamilton, New Zealand, worked with molecules that interfere with messenger RNA (mRNA), which helps translate genes into proteins. They found microRNA (miRNA) in mice that targeted beta-lactoglobulin mRNA, so they inserted DNA encoding a version of this miRNA into the genomes of cow embryos. Out of 100 embryos, one calf produced beta-globulin-free milk. This isnt a quick process, Stefan Wagner, a molecular biologist at AgResearch, told Nature. One problem is that RNA interference cant eliminate the protein completely because some mRNA slips through.

Another technique could speed up the process. TALENs are enzymes that target and cut out a specific DNA sequence from the genome. As the break is repaired, mutations are introduced that scramble the targeted gene, leaving it unable to function.

The TALEN technology is staggeringly easy, quick, and leaves no mark in the genome, researcher Bruce Whitelaw, told Nature. Whitelaw, a molecular biologist at the Roslin Institute near Edinburgh, UK, used TALENs to disrupt genes encoding low-density lipoprotein (LDL) receptors in pigs. Without those receptors, which remove LDL from the blood, Whitelaws pigs develop atherosclerotic arteries. Such pigs could be reliable models for biomedical researchers studying human atherosclerosis.

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The GM Barnyard

PARIS--(BUSINESS WIRE)--

Regulatory News:

Cellectis (ALCLS.PA), the French genome engineering specialist, announces in the Journal of Biological Chemistry, one of the most respected scientific journals in the world, the publication of a new approach regarding the targeted modification of DNA2. The manuscript unmasks novel perspectives and broadens the scope of TALENsTM technology to new therapeutic approaches to fight against cancer and genetic diseases. Until now, TALENsTM, the molecular scissors created by Cellectis Group, were only able to target certain parts of the genome. A team of the Groups researchers, led by Julien Valton and Philippe Duchateau, was able to overcome this constraint, opening the way to a wider range of applications, especially in the therapeutic field.

This study, the first to be published on TALENsTM, was awarded by the selection committee of the JBC as Paper of the Week.

Since their identification in 2009, TALEs have quickly emerged as the new generation of DNA-binding domain with programmable specificity and have been successfully used to generate the molecular scissors known as TALENsTM. However, their sensitivity to methylation, a ubiquitous modification of DNA, represents a major bottleneck for their widespread utilization in the genome engineering and therapeutic fields. Using a combination of biochemical, structural and cellular approaches, the R&D department of Cellectis was able to identify the basis of such sensitivity and more importantly, to propose an efficient and universal method to overcome it.

These results are proof of the scientific creativity and quality of our research teams, as well as the power of our genome engineering tools. This new publication strengthens the relevance of our investment in TALEstechnology, and confirms our strategy within the therapeutic field, declared Andr Choulika, Chief Executive Officer of Cellectis Group.

2) Overcoming TALE DNA Binding Domain Sensitivity to Cytosine Methylation Julien Valton, Aurelie Dupuy, Fayza Daboussi, Severine Thomas, Alan Marechal, Rachel Macmaster, Kevin Melliand, Alexandre Juillerat and Philippe Duchateau J. Biol. Chem. jbc.C112.408864. First Published on September 26, 2012, doi:10.1074/jbc.C112.408864

About Cellectis

Founded in France in 1999, the Cellectis Group is based on a highly specific DNA engineering technology. Its application sectors are human health, agriculture and bio-energies. Co-created by Andr Choulika, its Chief Executive Officer, Cellectis is today one of the world leading companies in the field of genome engineering. The Group has a workforce of 230 employees working on 5 sites worldwide: Paris & Evry in France, Gothenburg in Sweden, St Paul (Minnesota) & Cambridge (Massachusetts) in the United States. Cellectis achieved in 2011 16M revenues and has signed more than 80 industrial agreements with pharmaceutical laboratories, agrochemical and biotechnology companies since its inception. AFM, Dupont, BASF, Bayer, Total, Limagrain, Novo Nordisk are some of the Groups clients and partners.

Since 2007, Cellectis has been listed on NYSE-Euronext Alternext market (ALCLS.PA) in Paris.

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Cellectis Publishes Results Paving the Way for New Therapeutic Approaches against Cancer and Genetic Diseases

FIVE POINTS, Calif., Oct. 2, 2012 /PRNewswire/ --S&W Seed Company (Nasdaq:SANW) today announced that it has signed agreements with Monsantoand Forage Genetics International, LLC. (FGI) that enables the company to produce and sell seed of approved varieties containing Monsanto's Roundup Ready trait. FGI has also agreed to incorporate the Roundup Ready trait into specific S&W-developed varieties previously selected for high yield and salt tolerance.

Using classical plant breeding methods, S&W has worked hard for 30 years to become a leader in developing high yielding, non-dormant, salt tolerant varieties of alfalfa. Adoption of biotechnology has been slower to occur in Europe and other areas compared to the United States, and most of S&W's existing export customers require verification that alfalfa seed does not have biotech traits. Demand for S&W's proprietary non-biotech varieties continues to grow at a strong pace in warm climate zones where non-dormant alfalfa is the market standard. Accordingly, non-biotech varieties of alfalfa seed will continue to be a primary focus for S&W in international markets where biotech varieties have not been approved. S&W will also continue to ensure its classically bred varieties are grown under strict guidelines requiring isolation and quality control in processing.

In order to meet the needs of its many domestic customers that want Roundup Ready alfalfa, S&W has been working in collaboration with FGI to pursue the development of S&W varieties that incorporate Monsanto and FGI's advanced traits, such as the Roundup Ready trait, into S&W's own seed varieties. Any biotech varieties ultimately produced by S&W will be targeted for sale to the U.S. domestic customers, at least initially.

Mark Grewal, chief executive officer of S&W Seed Company commented, "We are pleased and honored that Monsanto and Forage Genetics chose S&W as a business partner in the alfalfa seed market. FGI and Monsanto are the undisputed world leaders in pioneering biotech research for alfalfa. We have always taken pride in our classically bred, high yielding, non-dormant, salt tolerant varieties. New S&W varieties that fill the demand for Roundup Ready technology can only expand our market within the United States. We look forward to completing the lab and field work necessary to have an S&W seed variety ready for the biotech market. In addition to the ability to sell the newly developed alfalfa seed varieties, S&W hopes to access future alfalfa seed technology developed by FGI and Monsanto under future licensing agreements."

"We are pleased S&W will introduce our Roundup Ready technology into some of their upcoming product offerings," Stephen Welker, Alfalfa Lead, Monsanto, commented. "Roundup Ready technology will provide the ability to produce higher quality and higher yielding crops that are largely free of weeds and other plants that compete for nutrients from the soil. S&W has a reputation for developing alfalfa seed varieties with a strong performance, including some of the most salt-tolerant varieties available."

About S&W Seed Company

Founded in1980 and headquartered in the Central Valley of California, S&W Seed Company is a leading producer of warm climate, high yield alfalfa seed varieties, including varieties that can thrive in poor, saline soils, as verified over decades of university-sponsoredtrials.S&WSeedalso offers seed cleaning and processing at its 40-acre facility in Five Points, Californiaand, in 2011,began the commercial launch of its California-basedsteviabusinessin response tothe growing global demand for the all-natural, zero calorie sweetenerfrom the food and beverage industry. For more information, please visitwww.swseedco.com.

Safe Harbor Statement

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may," "future," "plan" or "planned," "will" or "should," "expected," "anticipates," "draft," "eventually" or "projected." You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors and other risks identified in the Company's 10-K for thefiscal year ended June 30, 2012, and other filings made by the Company with the Securities and Exchange Commission.

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S&W Seed Announces Agreement with Monsanto and Forage Genetics

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

Contact: Hilary Glover hilary.glover@biomedcentral.com 44-020-319-22370 BioMed Central

Drug resistance is a major problem when treating infections. This problem is multiplied when the infection, like HIV-1, is chronic. New research published in BioMed Central's open access journal Retrovirology has examined the genetic footprint that drug resistance causes in HIV and found compensatory polymorphisms that help the resistant virus to survive.

Currently the strategy used to treat HIV-1 infection is to prevent viral replication, measured by the number of viral particles in the blood, and to repair the immune system, assessed using CD4 count. Over the past 20 years treatment and life expectancy have vastly improved. However, due to drug resistance, complete viral suppression requires an array of drugs.

For the virus drug resistance comes at a cost. In the absence of the drug the virus carrying drug resistance mutations is less 'fit' than the wild-type virus and so should not be able to replicate as efficiently. During interruptions to treatment wild-type viruses quickly predominate. However newly infected people can be drug resistant even before they have received any treatment.

Researchers from the SPREAD project have been monitoring HIV infections across Europe. This multinational team has looked at 1600 people, newly infected with HIV-1 subtype B. Almost 10% of these patients had HIV-1 harbouring transmitted drug resistance (TDR) and worryingly, when they measured virus production and CD4 count, there was no indication that these strains of HIV-1 were weaker.

In recent years there has been much talk about polymorphisms, naturally occurring differences in the genes that are responsible for the differences between animals of the same species, for example blood groups or the ability to digest lactose in milk. They may also increase propensity for certain diseases including cancer and type 2 diabetes. But animals are not the only organisms that harbour polymorphisms they are present in viruses as well.

By examining polymorphisms in these strains of HIV-1 the researchers discovered that certain polymorphisms in the gene coding for protease (essential for viral replication) known to act as compensatory mechanisms, improve the 'fitness' of resistant strains, even in the absence of the drug. Kristof Theys, one of the researchers involved in the project commented, "Our worry is that over time we will be seeing more people presenting with TDR HIV-1."

Prof Anne-Mieke Vandamme, who led this study, fears "Contrary to what was expected, transmission of TDR virus may also contribute to a 'fitter' and more virulent HIV, which has important clinical implications in how we best treat these people."

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The genetics of HIV-1 resistance

ALISO VIEJO, Calif.--(BUSINESS WIRE)--

Ambry Genetics (Ambry), a global leader in genetic services with a focus on clinical diagnostics and genomics, announces that chairman and CEO Charles Dunlop was honored with a RARE Champion award at Global Genes Projects Champions of Hope Gala Event on the evening of Thursday, September 27 in Newport Beach, Calif. Mr. Dunlop and Ambry Genetics were honored for Ambrys ongoing support of the Mauli Ola Foundation (Mauli Ola), a non-profit organization dedicated to alleviating symptoms of cystic fibrosis (CF) in children afflicted with the disorder through surfing.

This was truly amazing. I was both honored and humbled to accept this award on behalf of the incredible team at Ambry and Mauli Ola, commented Charles Dunlop. This honor means so much to us, but the greatest reward is still seeing the joy its bringing to kids with cystic fibrosis and their families. The surfing community has really rallied around this cause. Nothing could be more remarkable.

The award was issued in the Biotech/Industry category at the gala event held at Newport Beachs Balboa Bay Club & Resort, as part of the first annual RARE Tribute To Champions of Hope from the Global Genes Project. Ambrys award was presented by Rob Montelone, a schoolteacher from Yorba Linda, Calif. Rob and his wife are the parents of five children, three of whom have CF.

I was over the moon when I was invited by the Global Genes Project to present this award to Charles and Ambry, because Mauli Ola has been such an amazingly positive force for my family, having truly changed our lives, said Mr. Montelone. Mauli Olas good work has not only brought joy to my three children with cystic fibrosis; their symptoms have actually been alleviated.

Corporate sponsors of the event included Pfizer, Shire, Genzyme, Bayer, Goldman Sachs, Genzyme, and Burrill & Company.

This gala event was such a gratifying experience for a lot of us at Ambry, said Kelly Gonzalez, M.S., CGG, senior manager of clinical genomics at Ambry, who attended. We were particularly touched by some of the speeches from parents of children with rare diseases. It is just these types of diseases that we are successfully diagnosing with the Clinical Diagnostic Exome and other proprietary diagnostic tests, and it is very gratifying to be able to make a difference in the lives of such children and their parents.

About Ambry Genetics

Ambry Genetics is a College of American Pathologists (CAP)-accredited and Clinical Laboratory Improvement Amendments (CLIA)-certified commercial clinical laboratory with headquarters in Aliso Viejo, Orange County, Calif. Since its founding in 1999, it has become a leader in providing genetic services focused on clinical diagnostics and genomic services, particularly in sequencing and array services. Ambry has established a reputation for unparalleled service and has been at the forefront of applying new technologies to the clinical molecular diagnostics market and to the advancement of disease research. To learn more about testing and services available through Ambry Genetics, visit http://www.ambrygen.com.

About the Mauli Ola Foundation

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Ambry Genetics Chairman and CEO Charles Dunlop Honored with “RARE Champion” Award at Global Genes Project’s Champions ...

SILVER SPRING, Md. and SINGAPORE, Oct. 2, 2012 (GLOBE NEWSWIRE) -- Nuvilex, Inc. (NVLX), an international biotechnology provider of cell and gene therapy solutions, announced today its wholly-owned subsidiary, Austrianova Singapore Pte Ltd (ASPL) will attend this year's AusBiotech event.

The annual AusBiotech event this year will be held from October 30 - November 2 at the Melbourne Convention and Exhibition Centre, Melbourne, Australia. It has earned a reputation as the industry's premier biotechnology conference for the Asia Pacific region and has successfully expanded its relevance to the Australian and International Biotechnology industries by attracting more than 1100 participants from over 20 countries.

Dr Brian Salmons, CEO of ASPL said, "AusBiotech has grown in stature over the past several years. In prior years, we entered agreements with companies and found it to be one of the most valuable events for networking with new contacts. We anticipate meeting with companies with proprietary therapeutic cells, such as stem cells, that can leverage their technology with our Cell-in-a-Box(R) delivery system. We believe the new contacts we make will expand our customer base and increase the use of cell and gene therapy for making therapeutic products and treating diseases. We will also be promoting our Bac-in-a-Box(R) technology for the first time at this meeting and anticipate generating interest around its potential."

The Chief Executive of Nuvilex, Dr. Robert Ryan, stated "Attendance at this important biotech event in Australia and within easy reach of Southeast Asia will enable us to have increased exposure for our Cell-in-a-Box(R) and Bac-in-a-Box(R) live cell encapsulation technology and to showcase its immense versatility, thus providing our companies greater visibility at a time that such capabilities are becoming more important in the marketplace. It is our goal to bring more projects to fruition from this meeting as more companies today are looking to bring cellular-based therapy and product creation from the drawing board to reality and into regular use."

About Nuvilex

Nuvilex, Inc. (NVLX) is an international biotechnology provider of live therapeutically valuable, encapsulated cells and services for research and medicine. A great deal of work is ongoing to move Nuvilex and its Austrianova Singapore subsidiary forward. This was clearly apparent during Dr. Ryan's trip to Singapore and the advent of new developments in the company as a whole. Our company's own offerings will include cancer, diabetes, other treatments and capabilities using the company's cell and gene therapy expertise and live-cell encapsulation technology.

The Nuvilex, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=13494

Safe Harbor Statement

This press release contains forward-looking statements described within the 1995 Private Securities Litigation Reform Act involving risks and uncertainties including product demand, market competition, and meeting current or future plans which may cause actual results, events, and performances, expressed or implied, to vary and/or differ from those contemplated or predicted. Investors should study and understand all risks before making an investment decision. Readers are recommended not to place undue reliance on forward-looking statements or information. Nuvilex is not obliged to publicly release revisions to any forward-looking statement, reflect events or circumstances afterward, or disclose unanticipated occurrences, except as required under applicable laws.

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Nuvilex Subsidiary Austrianova Singapore to Participate in AusBiotech 2012

SEATTLE, Oct. 2, 2012 /PRNewswire/ --Cell Therapeutics, Inc. ("CTI") (NASDAQ and MTA: CTIC) announced that OPAXIO (paclitaxel poliglumex) has been granted orphan-drug designation by the U.S Food and Drug Administration ("FDA") for the treatment of glioblastoma multiforme ("GBM"), a malignant brain cancer.

Orphan designation was granted based on preliminary activity seen from phase 2 results of OPAXIO when added to standard therapy (temozolamide ("TMZ") plus radiation). In this study, progression-free and overall survival was encouraging among patients with GBM, including patients whose tumors expressed unmethylated MGMT. Current standard therapy is less effective in patients with tumors that have unmethylated MGMT, an important DNA repair enzyme. A randomized trial is now underway for patients with GBM with unmethylated MGMT comparing standard TMZ and radiation to OPAXIO and radiation.

According to theNational Cancer Institute, GBM is the most common and deadliest type of primary brain tumor in adults. It is estimated that there will be 10,000-12,000 new cases of GBM diagnosed in the US this year alone. The standard of care for patients with GBM is a surgical resection, if possible, followed by radiation given with concurrent TMZ. The prognosis for the majority of patients with GBM is poor with less than 25% of patients surviving two years with current therapies. Survival is shorter for patients whose tumors have active (unmethylated) MGMT.

Orphan-drug designation is granted by the FDA to novel drugs that seek to treat a rare disease or condition. Orphan-drug designation provides substantial potential benefits to the drug developer, including seven years of market exclusivity for the product upon regulatory approval, fee waivers and tax incentives.

Under the leadership of Dr. Howard Safran at Brown University Medical Center, a multicenter Phase 2 study (BrUOG 244) has been initiated comparing the efficacy of OPAXIO plus radiation with that of TMZ plus radiation in newly-diagnosed patients with GBM and unmethylated MGMT. In approximately 55% of patients with GBM, MGMT is unmethylated, thereby decreasing the efficacy of standard therapy with TMZ plus radiation therapy ("RT"). The randomized study seeks to determine whether OPAXIO plus radiation will improve progression free survival and overall survival when compared to TMZ plus radiation, the current treatment standard in this disease.

"The current randomized trial is based on the encouraging results previously demonstrated with OPAXIO and radiation in patients with newly diagnosed malignant brain cancer and specifically targets GBM patients with a genomic marker, unmethylated MGMT, who are less likely to benefit from the current standard of care TMZ and radiation," stated Howard Safran, M.D., Medical Director of the Brown University Oncology Group. "We are pleased OPAXIO has been granted orphan-drug designation as patients with this disease have a serious unmet medical need for improved long-term survival particularly when MGMT is unmethylated."

About the Study

The study is expected to enroll up to 120 patients. Patients in the OPAXIO arm will receive OPAXIO once every week plus RT for six weeks. Patients in the TMZ arm will receive daily oral TMZ plus RT for six weeks. After completion of initial therapy, both arms will receive maintenance TMZ on day 1-5 and then every 28 days for up to 12 cycles for a total of 48 weeks. Participating institutions include Rhode Island Hospital of Brown University, Memorial Hospital of University of Massachusetts, Maine Medical Center, Upstate Medical Center of the State University of New York (Syracuse), the University of Washington, the University of California at San Diego and Penn State Hershey Medical Center.

About OPAXIO

OPAXIO (paclitaxel poliglumex, CT-2103), is an investigational, biologically enhanced, chemotherapeutic that links paclitaxel, the active ingredient in Taxol, to a biodegradable polyglutamate polymer, which results in a new chemical entity. When bound to the polymer, paclitaxel is inactive, potentially sparing normal tissue's exposure to high levels of paclitaxel and its associated toxicities. Blood vessels in tumor tissue, unlike blood vessels in normal tissue, are porous to macromolecules such as OPAXIO. Based on preclinical studies, it appears that OPAXIO is preferentially distributed to tumors due to their leaky blood vessels and trapped in the tumor bed, allowing significantly more of the dose of chemotherapy to localize in the tumor than with standard paclitaxel. Once inside the tumor cell, enzymes metabolize the protein polymer, releasing active paclitaxel.Unlike standard radiosensitizing agents, OPAXIO appears tumor selective and does not appear to enhance radiation toxicity to normal tissues.

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Cell Therapeutics OPAXIOTM Receives Orphan Drug Designation for Malignant Brain Cancer from FDA

ScienceDaily (Oct. 1, 2012) A novel therapy in the early stages of development at Virginia Commonwealth University Massey Cancer Center shows promise in providing lasting protection against the progression of multiple myeloma following a stem cell transplant by making the cancer cells easier targets for the immune system.

Outlined in the British Journal of Hematology, the Phase II clinical trial was led by Amir Toor, M.D., hematologist-oncologist in the Bone Marrow Transplant Program and research member of the Developmental Therapeutics program at VCU Massey Cancer Center. The multi-phased therapy first treats patients with a combination of the drugs azacitidine and lenalidomide. Azacitidine forces the cancer cells to express proteins called cancer testis antigens (CTA) that immune system cells called T-cell lymphocytes recognize as foreign. The lenalidomide then boosts the production of T-cell lymphocytes. Using a process called autologous lymphocyte infusion (ALI), the T-cell lymphocytes are then extracted from the patient and given back to them after they undergo a stem cell transplant to restore the stem cells' normal function. Now able to recognize the cancer cells as foreign, the T-cell lymphocytes can potentially protect against a recurrence of multiple myeloma following the stem cell transplant.

"Every cell in the body expresses proteins on their surface that immune system cells scan like a barcode in order to determine whether the cells are normal or if they are foreign. Because multiple myeloma cells are spawned from bone marrow, immune system cells cannot distinguish them from normal healthy cells," says Toor. "Azacitidine essentially changes the barcode on the multiple myeloma cells, causing the immune system cells to attack them," says Toor.

The goal of the trial was to determine whether it was safe, and even possible, to administer the two drugs in combination with an ALI. In total, 14 patients successfully completed the investigational drug therapy. Thirteen of the participants successfully completed the investigational therapy and underwent a stem cell transplant. Four patients had a complete response, meaning no trace of multiple myeloma was detected, and five patients had a very good partial response in which the level of abnormal proteins in their blood decreased by 90 percent.

In order to determine whether the azacitidine caused an increased expression of CTA in the multiple myeloma cells, Toor collaborated with Masoud Manjili, D.V.M., Ph.D., assistant professor of microbiology and immunology at VCU Massey, to conduct laboratory analyses on bone marrow biopsies taken from trial participants before and after treatments. Each patient tested showed an over-expression of multiple CTA, indicating the treatment was successful at forcing the cancer cells to produce these "targets" for the immune system.

"We designed this therapy in a way that could be replicated, fairly inexpensively, at any facility equipped to perform a stem cell transplant," says Toor. "We plan to continue to explore the possibilities of immunotherapies in multiple myeloma patients in search for more effective therapies for this very hard-to-treat disease."

In addition to Manjili, Toor collaborated with John McCarty, M.D., director of the Bone Marrow Transplant Program at VCU Massey, and Harold Chung, M.D., William Clark, M.D., Catherine Roberts, Ph.D., and Allison Hazlett, also all from Massey's Bone Marrow Transplant Program; Kyle Payne, Maciej Kmieciak, Ph.D., from Massey and the Department of Microbiology and Immunology at VCU School of Medicine; Roy Sabo, Ph.D., from VCU Department of Biostatistics and the Developmental Therapeutics program at Massey; and David Williams, M.D., Ph.D., from the Department of Pathology at VCU School of Medicine, co-director of the Tissue and Data Acquisition and Analysis Core and research member of the Developmental Therapeutics program at Massey.

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Immune system harnessed to improve stem cell transplant outcomes

A new cancer research centre has been opened in Adelaide.

The $5 million centre has been funded by the Australian Cancer Research Foundation and the South Australian Government.

Its focus will be gene research and development of individually-tailored treatments.

SA Health Minister John Hill said the centre, in Frome Road in the city, would benefit many people for years to come.

"The end result of this research will contribute to the eventual development of personalised cancer treatment with fewer long-term side effects and better patient outcomes," he said.

A new unit also has been opened at a cancer research facility in Adelaide's southern suburbs, with a focus on cancer prevention.

The unit at the Flinders Centre for Innovation in Cancer cost about $4 million to establish.

Researcher Professor Graeme Young said behavioural, dietary and environmental factors would be studied, and there would be a focus on early detection and screening programs.

"We need better solutions for cancer prevention because we're still battling to control this problem, especially as people age," he said.

Read the original:
Cancer research facilities boosted

By Elizabeth Lopatto - 2012-10-01T19:53:38Z

Scientists have altered the genes of a dairy cow to produce milk thats rich in a protein used in numerous food products and lacking in a component that causes allergies in humans.

Using a process called RNA-interference that turns certain genes on or off, scientists from New Zealand produced a cow whose milk had increased casein, a protein used to make cheese and other foods, and almost no beta-lactoglobulin, a component in milk whey protein that causes allergies. The female calf was also born without a tail, according to the report today in the journal Proceedings of the National Academy of Sciences.

The study can be seen as a proof-of-concept that tinkering with nutritional content genetically is possible, said William Hallman, director of the food policy institute at Rutgers University. More testing will be needed to determine the milks full dietary content, and scientists must consider the effects of breeding gene-altered animals, he said. The field has been controversial because of safety and environmental concerns.

Could you clone a breeding stock that would allow for a herd with milk of this type? Hallman, who wasnt involved in the study, said in a telephone interview. There are lots of issues about what might happen in the next generation.

Todays research represents a road map for other groups that may wish to knock out proteins, and not just in milk, he said.

Farmers in the U.S. earned about $35 billion in 2011 from dairy sales, said Christopher Galen, a spokesman for the National Milk Producers Federation.

Aside from the hypoallergenic qualities, the genetically modified cows milk may also be valuable for its higher content of casein. The milk protein is used in a range of food products, including cheese, thickening agents in soups, salad dressings and whipped toppings. Its also used in adhesives, cosmetics and some pharmaceuticals, Hallman said.

In terms of dairy economics, casein is the most profitable part of the milk, he said.

Ordinarily, the proportion of whey to casein in the milk from dairy cows is 21-to-79, according to the paper. The milk from the genetically altered cow had a ratio of 4-to-96, according to a paper. Thats probably due to a 96 percent reduction in beta-lactoglobulin, or BGL, the paper said.

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Gene-Modified Cow Makes Milk Rich in Protein, Study Finds

The U.S. National Institutes of Health (NIH) today announced that researchers investigating a gene known to increase the risk of Alzheimers have discovered the gene is also associated with lower levels of beta amyloid in healthy older people. This finding throws into question the current hypothesis in Alzheimers research that holds increasing beta amyloid levels are a large part of the disease. Beta amyloid is a brain protein that is a main component of deposits found in the brains of Alzheimers patients.

The prevailing hypothesis has implicated factors increasing beta amyloid in the brain as an integral element of Alzheimers disease pathology, said Dr. Richard Hodes, National Institute on Aging (NIA) director. This study indicates the importance of exploring and understanding other distinct mechanisms that may be at work in this disease.

Researchers used brain scans to measure brain amyloid in 57 cognitively normal older people from the Baltimore Longitudinal Study of Aging (BLSA) and 22 cognitively normal people from the Alzheimers Disease Neuroimaging Initiative (ADNI). The participants were age 78.5 on average. They found that 17 of the BLSA participants and four of the ADNI participants carried the Alzheimers risk variant of the complement receptor-1 (CR1) gene.

We found that brain amyloid burden in the group with the CR1 risk variant was lower than in the group without it, said Dr. Madhav Thambisetty, lead author of the study and chief of the Clinical and Translational Neuroscience Unit in the Laboratory of Behavioral Neuroscience of the NIAs Intramural Research Program. This difference in brain amyloid between the two groups is statistically significant in several brain regions. That suggests to us that the CR1 risk factor gene, if it contributes to Alzheimers disease, does it in a way unrelated to increasing amyloid burden.

The findings suggest that the increased risk of Alzheimers associated with CR1 is not driven by an increase in amyloid in the brain and that we may also need to consider multiple genetic risk factors in combination. It may be possible that CR1 acts through other mechanisms, distinct from those that increase amyloid deposition in the brain. These may include influencing inflammation in the brain, but further research is needed to identify what these other mechanisms might be.

The study was a joint effort by the NIH and NIA. It was recently published in the journal Biological Psychiatry.

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Alzheimer’s Gene Linked to Lower Brain Amyloid

FRIDAY, Sept. 28 (HealthDay News) -- Lower back pain that occurs when discs in the spine deteriorate over time may be linked to a specific gene, according to a new study.

Researchers at King's College London said their findings could lead to the development of new treatments for this common type of back pain, known as lumbar disc degeneration.

Vertebrates develop bony growths called osteophytes when the discs next to them become dehydrated and lose height. These growths can lead to lower back pain. More than one-third of middle-aged women have at least one degenerate disc, the researchers said, and between 65 and 80 percent of the people with this condition inherited it.

By comparing the spines of 4,600 people using MRI images and mapping their genes, the study found the PARK2 gene, in particular, was linked to those with degenerating discs. This gene, they explained, may be turned off in people with lumbar disc degeneration. The researchers also said this gene could affect how quickly discs in the spine deteriorate.

It's still unclear how the PARK2 gene may get turned off in people with lumbar disc degeneration. The researchers suggested environmental factors, such as lifestyle or diet, could play a role.

"Further work by disc researchers to define the role of this gene will, we hope, shed light on one of the most important causes of lower back pain," said Dr. Frances Williams, senior lecturer from the department of twin research and genetic epidemiology at King's College London, in a college news release.

The study authors added that more research is needed to fully explain how this condition is triggered. Their research uncovered an association between the gene and disc problems, not a cause and effect.

"It is feasible that if we can build on this finding and improve our knowledge of the condition, we may one day be able to develop new, more effective treatments for back pain caused by this common condition," Williams added.

The study was published online Sept. 19 in the Annals of Rheumatic Diseases.

-- Mary Elizabeth Dallas

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Study Ties Common Back Ailment to Faulty Gene

The announcement about scientists producing GE milk will damage New Zealands brand on which our more than $10 billion a year dairy exports rely, the Green Party said today.

Today AgResearch scientists announced they have used genetic engineering (GE) technology to breed the first cow in the world that produces high protein milk that may be hypo-allergenic.

"This is just another GE mirage; another announcement from GE proponents about a potential product which we do not need and has no market," Green Party GE Steffan Browning said today.

"Its not right for these scientists to be touting their finding as a solution to milk allergies in babies as some sort of justification for the huge amount of resources that have been invested into GE research.

"We see this over and over again with GE scientists; this new product or that new product that will have apparent amazing results but it never actually meets our real needs for a safe, healthy food supply.

"The Royal Commission into GE recommended that wherever possible animals that are a common source of food should not be used for GE but that recommendation has been ignored.

"Field trials in New Zealand need to be closed down and GE research needs to be kept in the lab.

"Our export markets want safe food grown in a natural environment but the production of GE milk puts those markets at risk.

"Putting at $10 billion a year industry at risk for half a glass of milk is not something to be celebrating," said Mr Browning.

Original post:
Just another GE mirage

A cow in New Zealand has been genetically modified to produce hypoallergenic milk.

AgResearch

Two genetically engineered farm animals reported today illustrate how far from Frankensteins stitched-together monster animal biotechnology has come. One of those animals, a cow, secretes milk that lacks an allergy-inducing protein because researchers accurately blocked its production using the technique of RNA interference1. And in pigs, scientists have used an enzyme called a TALEN2 to scramble a gene that would normally help remove cholesterol.

RNA interference (RNAi) and TALENs are more accurate at targeting the gene in question than are earlier genetic engineering techniques. For years, researchers tried to remove the allergy-inducing milk protein beta-lactoglobulin from cow's milk, which can cause diarrhea and vomiting in some toddlers. They tried replacing the gene encoding beta-lactoglobulin with a defective form, but this proved nearly impossible because the techniques available to introduce foreign genes into animal genomes were not precise, and misplaced genes failed to express themselves correctly.

In 2006, scientists at AgResearch in Hamilton, New Zealand began to experiment with molecules that interfere with the messenger RNA go-between that enables translation of a gene into protein. In mice, they discovered a short chunk of RNA, called a microRNA, that targeted beta-lactoglobulin messenger RNA directly to prevent its translation. They inserted DNA encoding a version of this microRNA into the genome to create genetically modified cow embryos that they hoped would grow into cows without the allergen in their milk. Out of 100 embryos, one calf yielded beta-globulin-free milk. This isnt a quick process, says Stefan Wagner, a molecular biologist at AgResearch. That's why it has taken so long to succeed in making an allergen-free cow, he says.

Wagner says that TALENs, which were not readily available when he began his research, might speed up the process, and that the team plans to use them to eliminate beta-lactoglobulin. RNAi cannot eliminate the protein completely because some messenger RNA slips past the blockade, but each TALEN targets a specific DNA sequence in the genome and cuts it. As the body repairs the break, mutations are often introduced that render the targeted gene non-functional. The TALEN technology is staggeringly easy, quick, and leaves no mark in the genome, says Bruce Whitelaw, a molecular biologist at the Roslin Institute near Edinburgh in the United Kingdom, who contributed to the work in pigs. In essence, we are just mimicking an evolutionary process with precise, man-made editors.

His team used TALENs to disrupt genes encoding low-density lipoprotein (LDL) receptors. Without these receptor proteins to remove cholesterol-containing LDLs from the blood, LDLs build-up and lead to atherosclerosis. Pigs with this condition may be reliable models of human atheroscelerosis in biomedical research.

The TALEN-modified pig is not the first model of human heart disease (see Model pigs face a messy path), but the technique makes genetic engineering less costly and more efficient. Id be exaggerating if I said that pigs and cows can now be thought of as big mice, but we are moving in that direction, says Heiner Niemann, a bioengineer at the Institute of Farm Animal Genetics in Neustadt, Germany.

The excitement surrounding these technological advances is bittersweet, however. Originally, engineered animals were produced with the aim of making food safer, healthier and more abundant. Yet despite years of investment, almost no animal has been approved by regulatory agencies around the world. Wagner says he has not tasted the milk from his special cow because hes not permitted to under New Zealand law. We must restrict our research to scientific analysis, he says. The current climate for animal biotech is not very good, and therefore, we are nowhere near getting this to the consumer."

Originally posted here:
Animals engineered with pinpoint accuracy

Smoking is not the only cause of lung cancer.

"It's a heterogeneous group of people who get lung cancer these days," said Dr. Linda Garland, director of clinical research in thoracic oncology at The University of Arizona Cancer Center in Tucson.

Garland says she's seen a stigma attached to lung cancer, where people blame the patient and think it's something they brought on themselves by smoking. But that is not an accurate picture.

Secondhand smoke exposure, and exposure to uranium and radon, are all associated with causing lung cancer in nonsmokers, she said. And nonsmokers who live in very polluted cities get lung cancer at a higher rate than people who live in a very clean environment.

Other cases are attributed to genetics.

The good news for people whose lung cancer is related to genetic abnormalities is that there are oral drugs that have proven very effective in shrinking tumors.

"The interesting thing is we've come to find a lot of very important information about 'never smokers' ' lung cancer in that we now have defined three very specific genetic abnormalities and those drive the lung cancer," Garland said.

"These driver mutations or fusion genes create in a very simplistic way a very activated pathway that leads to proliferation, ability to metastasize, replication potential - all the things that define a cancer cell."

When a tumor is removed, it can be tested for the three genetic abnormalities and if one is found, the patient can be treated with one of two cancer medications that became available in the last 15 years. When the gene mutation is targeted with the right medication, very rapid, very significant tumor shrinkage can occur, Garland said.

"Sometimes it looks like you've got the electrical circuit to Los Angeles County and you take the main breaker and turn it off. It's very dramatic," she said. "People can get some very prolonged, excellent benefit from these pills."

Read this article:
Genetics, pollution contribute to lung cancer

LOS ANGELES, Oct.1, 2012 /PRNewswire/ --Response Genetics, Inc. (RGDX), a company focused on the development and commercialization of molecular diagnostic tests for cancer, announced today the addition of James Wynne as the company's Vice President, Sales & Marketing. In this newly created position, Mr. Wynne will be responsible for leading the company's ResponseDX strategic and operational sales and marketing activities including the expansion of the company's direct sales efforts and identifying new service offerings.

"With Jim's strong background in providing results driven and advanced skills in sales management, marketing and communications in the clinical laboratory diagnostics space, we believe he is ideally suited to lead our ResponseDX sales and marketing efforts," said Thomas A. Bologna, Chairman & Chief Executive Officer of Response Genetics. "Jim's track record of building a performance driven sales team along with his true appreciation for how to market clinical testing services to pathologists and oncologists will be a tremendous asset to us. We are looking forward to his contributions."

Mr. Wynne has spent the last 20 plus years in cancer diagnostics. He spent the first six years in pharmaceutical sales moving into leadership roles. He joined Impath in 1995 as a Territory Sales Manager before being promoted to Director of Sales for the western region. Under his leadership revenue grew exponentially. After nine successful years, Mr. Wynne was recruited to UCLA Medical Center as the Director of Sales for UCLA's pathology outreach program. Mr. Wynne joins Response Genetics from Clarient, Inc. where he started in 2004 when the company was known as Chromavision Medical Systems. Mr. Wynne recruited and developed the original Clarient sales team and worked with the marketing group to prioritize product development. During his tenure, he directly helped the sales team grow to an approximately $115 million revenue generating organization at the time of its acquisition by GE Healthcare. Mr. Wynne received a BA in Sociology from the University of California, Los Angeles and an MBA from Pepperdine University.

Mr. Wynne, commenting on his appointment, "I am extremely excited about joining Response Genetics for a number of reasons. It's a perfect fit for my background, interests and skills, and I am happy to be a part of the leadership team that Tom has assembled to transition Response Genetics into a top line, sales driven, high growth company."

About Response Genetics, Inc.

Response Genetics, Inc. (the "Company") is a CLIA-certified clinical laboratory focused on the development and sale of molecular diagnostic testing services for cancer. The Company's technologies enable extraction and analysis of genetic information from genes derived from tumor samples stored as formalin-fixed and paraffin-embedded specimens. The Company's principal customers include oncologists and pathologists. In addition to diagnostic testing services, the Company generates revenue from the sale of its proprietary analytical pharmacogenomic testing services of clinical trial specimens to the pharmaceutical industry. The Company's headquarters is located in Los Angeles, California. For more information, please visit http://www.responsegenetics.com.

Forward-Looking Statement Notice

Except for the historical information contained herein, this press release and the statements of representatives of the Company related thereto contain or may contain, among other things, certain forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995.

Such forward-looking statements involve significant risks and uncertainties. Such statements may include, without limitation, statements with respect to the Company's plans, objectives, projections, expectations and intentions, such as the ability of the Company, to provide clinical testing services to the medical community, to continue to expand its sales force, to continue to build its digital pathology initiative, to attract and retain qualified management, to continue to provide clinical trial support to pharmaceutical clients, to enter into new collaborations with pharmaceutical clients, to enter into areas of companion diagnostics, to continue to execute on its business strategy and operations, to continue to analyze cancer samples and the potential for using the results of this research to develop diagnostic tests for cancer, the usefulness of genetic information to tailor treatment to patients, or to successfully file a registration statement with the Securities Exchange Commission ("SEC"), and other statements identified by words such as "project," "may," "could," "would," "should," "believe," "expect," "anticipate," "estimate," "intend," "plan" or similar expressions.

These statements are based upon the current beliefs and expectations of the Company's management and are subject to significant risks and uncertainties, including those detailed in the Company's filings with the SEC. Actual results, including, without limitation, actual sales results, if any, or the application of funds, may differ from those set forth in the forward-looking statements. These forward-looking statements involve certain risks and uncertainties that are subject to change based on various factors (many of which are beyond the Company's control). The Company undertakes no obligation to publicly update forward-looking statements, whether because of new information, future events or otherwise, except as required by law.

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Response Genetics, Inc. Announces Appointment of James Wynne as Vice President, Sales & Marketing

HOUSTON, Oct. 1, 2012 /PRNewswire/ --The Houston Stem Cell Summit will host an extraordinary lineup of keynote speakers who represent the most accomplished stem cell scientists, clinicians and entrepreneurs in the United States. Joining these distinguished speakers will be Governor of Texas, Rick Perry, consistent champion of adult stem cell therapies.

(Logo: http://photos.prnewswire.com/prnh/20120831/NY66463LOGO )

The Houston Stem Cell Summit will be held October 26 27 in its namesake city and will highlight the latest therapeutic research regarding the use of adult stem and progenitor cell therapies. The Summit will also provide a forum for entrepreneurs to discuss their latest efforts to commercialize stem cell therapies, and to debate and discuss FDA and other legal and regulatory issues impacting stem cell research and commercialization.

Opening Keynote Address October 26, 2012 Arnold I. Caplan, PhD, Professor of Biology and Professor of General Medical Sciences (Oncology) Case Western Reserve University

Dr. Caplan has helped shape the direction and focus of adult stem cell research and commercialization. Virtually every adult stem cell company and literally tens of thousands of research papers are based on Dr. Caplan's original and ground breaking research. Professor Caplan is considered to be the "father" of the mesenchymal stem cell and first described this progenitor cell in his landmark paper; "Mesenchymal stem cells", Journal of Orthopaedic Research 1991;9(5):641-650. Since that foundational study, Dr. Caplan has published over 360 manuscripts and articles in peer reviewed journals. Dr. Caplan has been Chief Scientific Officer at OrthoCyte Corporation since 2010. In addition, Dr. Caplan co-founded Cell Targeting Inc. and has served as President of Skeletech, Inc. as its founder. He is the recipient of several honors and awards from the orthopedic research community. Dr. Caplan holds a Ph. D. from Johns Hopkins University Medical School and a B.S. in chemistry from the Illinois Institute of Technology.

Summit Keynote Address October 26, 2012 Texas Governor Rick Perry

Governor Perry is the 47th and current Governor of Texas. Governor Perry has long championed the role of medical technologies in building the future of not only Texas, but also the United States. In many ways, his strong advocacy on behalf of research and advanced medical technologies is one of his strongest and as yet underappreciated legacies. In addition to his service to the state of Texas, Governor Perry has also served as Chairman of the Republican Governors Association in 2008 and again in 2011. Despite a rigorous schedule, particularly in the teeth of this election season, Governor Perry has graciously made time to speak and encourage the researchers, patients, companies and physicians who form the fabric and future of the stem cell therapy community.

Texas Medical Center Keynote Address, October 27, 2012 James T. Willerson, MD

Over the course of his career, Dr. James T. Willerson has served as a medical, scientific and administrative leader for each of the major institutions that are the foundation of the Texas Medical Center. Dr. Willerson is currently President and Medical Director, Director of Cardiology Research, and Co-Director of the Cullen Cardiovascular Research Laboratories at Texas Heart Institute (THI). Dr. Willerson was appointed President-Elect of THI in 2004 and became President and Medical Director in 2008. He is also an adjunct professor of Medicine at Baylor College of Medicine and at The University of Texas MD Anderson Cancer Center. He is the former chief of Cardiology at St. Luke's Episcopal Hospital and the former chief of Medical Services at Memorial Hermann Hospital.

Dr. Willerson has served as a visiting professor and invited lecturer at more than 170 institutions.

View post:
Houston Stem Cell Summit Announces Extraordinary Lineup of Keynote Speakers

For more than 40 years, Lesley Kelly of Glasgow, Scotland, lived with third-degree burns that stretched over 60 percent of her body.

Kelly was 2 years old when she fell into a bathtub filled with hot water that scorched most of the right side of her body. She lost full range of motion around many of her joints.

"When you have bad scarring, the buildup is very thick and has no elasticity," said Kelly, 45, whose right elbow was most affected by the buildup of scar tissue. "The problem with thermal burn scarring [is that] it's hard to get the range of motion."

Kelly underwent numerous reparative surgeries through the years, but the scar tissue continued to grow back. The procedures did not lessen the look of her scars.

In 2011, Kelly underwent a new, experimental procedure that used stem cells from her own fat tissue to repair the buildup around her right elbow.

Surgeons cleaned the scar buildup around the elbow and used liposuction to pull fat from off Kelly's waist. They separated the fat cells from the stem and regenerative cells, which were then injected into the wound on Kelly's arm. The procedure took less than two hours.

Within months, Kelly was able to regain 40 degrees of motion that she had lost more than 40 years ago.

"If this technology was available earlier in my life, my scars would not have been as bad," said Kelly.

There are an estimated 50,000 to 70,000 burn cases each year in the U.S., according to the American Burn Association.

The stem cell therapy, approved in the U.K. to treat soft tissue wounds, is now gaining traction in the U.S.

Here is the original post:
Experimental Stem Cell Therapy May Help Burn Victims

Lesley Kelly, 45, underwent stem cell therapy to repair scar tissue buildup in her right arm. (Cytori Therapeutics, Inc.)

By Lara Salahi, ABC News For more than 40 years, Lesley Kelly of Glasgow, Scotland, lived with third-degree burns that stretched over 60 percent of her body.

Kelly was 2 years old when she fell into a bathtub filled with hot water that scorched most of the right side of her body. She lost full range of motion around many of her joints.

"When you have bad scarring, the buildup is very thick and has no elasticity," said Kelly, 45, whose right elbow was most affected by the buildup of scar tissue. "The problem with thermal burn scarring [is that] it's hard to get the range of motion."

Kelly underwent numerous reparative surgeries through the years, but the scar tissue continued to grow back. The procedures did not lessen the look of her scars.

In 2011, Kelly underwent a new, experimental procedure that used stem cells from her own fat tissue to repair the buildup around her right elbow.

Surgeons cleaned the scar buildup around the elbow and used liposuction to pull fat from off Kelly's waist. They separated the fat cells from the stem and regenerative cells, which were then injected into the wound on Kelly's arm. The procedure took less than two hours.

Within months, Kelly was able to regain 40 degrees of motion that she had lost more than 40 years ago.

"If this technology was available earlier in my life, my scars would not have been as bad," said Kelly.

There are an estimated 50,000 to 70,000 burn cases each year in the U.S., according to the American Burn Association.

See the original post:
New Therapy May Help Burn Victims

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

Contact: Stephanie Desmon sdesmon1@jhmi.edu 410-955-8665 Johns Hopkins Medicine

Working with mice, Johns Hopkins researchers say they have developed a gene-based therapy to stop the rodent equivalent of the autoimmune disease myasthenia gravis by specifically targeting the destructive immune response the disorder triggers in the body.

The technique, the result of more than 10 years of work, holds promise for a highly specific therapy for the progressively debilitating muscle-weakening human disorder, one that avoids the need for long-term, systemic immunosuppressant drugs that control the disease but may create unwanted side effects.

The research, if replicated in humans, could be a big leap in treating not only myasthenia gravis, but also other autoimmune disorders, the researchers say.

"To treat autoimmune diseases, we normally give drugs that suppress not only the specific antibodies and cells we want to inhibit, but that also broadly interfere with other functions of the immune system," says Daniel B. Drachman, M.D., a professor of neurology and neuroscience at the Johns Hopkins University School of Medicine and leader of the study published this month in the Journal of Neuroimmunology. "Our goal was to suppress only the abnormal response, without damaging the remainder of the immune system, and that's what we did in these mice."

A healthy immune system has the amazing ability to distinguish between the body's own cells, recognized as "self," and foreign proteins and other substances including germs and tumors to fight infections, cancer and other diseases. The body's immune defenses normally coexist peacefully with cells that carry distinctive "self" marker molecules. But when immune defenders encounter foreign molecules, they quickly launch an attack. Autoimmune disorders occur when the immune system makes a mistake, in which it confuses "self" with something foreign, and then launches an attack by immune cells and/or antibodies to seek out and damage the body's own cells.

Drachman, one of the world's leading authorities on myasthenia gravis and other neurologic autoimmune disorders, and his colleagues say they have found a way to create a "guided missile" approach as opposed to the "carpet bombing" of overall immunosuppression. Essentially, Drachman says, the method eliminates the cells of the immune system that are involved in the attack against self and leaves other cells alone.

The research team created the guided missiles by genetically engineering dendritic cells, which are the immune cells that specialize in presenting antigens to the immune system's T-cells. They extracted dendritic cells from mice with myasthenia gravis, purified them and inserted genes which direct these dendritic cells to target the auto-aggressive immune cells, and destroy them using a "warhead" known as Fas ligand. Then they injected back into the mice the genetically engineered cells, which homed in on the immune system's faulty T-cells. The newly introduced "guided missiles" then sought out and bound themselves to those T-cells, causing apoptosis, or cellular suicide, which halted the autoimmune attack before it could gain traction.

"This way, the autoantibodies were specifically reduced, a key step in treating myasthenia gravis," Drachman says.

Continued here:
Researchers halt autoimmune disease myasthenia gravis in mice

October 1, 2012

redOrbit Staff & Wire Reports Your Universe Online

A team of researchers, led by members of the University of Cincinnati (UC) and Cincinnati Childrens Hospital Medical Center, have reportedly discovered a new genetic mutation that leads to deafness and hearing loss associated with a relatively rare condition.

In a September 30 press release, lead investigator and UC assistant professor of ophthalmology Zubair Ahmed explain that he and his colleagues were able to pinpoint the gene which caused deafness in Usher syndrome type 1 as well as deafness that is not associated with the syndrome through the genetic analysis of 57 humans from Pakistan and Turkey.

The culprit, according to Ahmed, is a protein known as CIB2. Mutations in the protein, which binds to calcium inside cells, has been discovered to be linked to deafness both in Usher syndrome and cases of non-syndromic hearing loss.

He noted that these mutations were found to be the primary genetic cause of non-syndromic hearing loss in Pakistan, and that during their research, he and his colleagues discovered a second CIB2 mutation that has been linked to deafness among people of Turkish heritage.

In animal models, CIB2 is found in the mechanosensory stereocilia of the inner ear hair cells, which respond to fluid motion and allow hearing and balance, and in retinal photoreceptor cells, which convert light into electrical signals in the eye, making it possible to see, Saima Riazuddin, assistant professor in UCs department of otolaryngology and co-lead investigator on the study, added in a statement.

Ahmed, Riazuddin, and company found that CIB2 tended to be stained brighter at the tips of shorter rows of the cellular apical modifications known as stereocilia than nearby longer rows, where it could be involved in the calcium signaling process which regulates how the ear converts mechanical energy into the type of energy recognizable by a persons brain as sound.

With this knowledge, we are one step closer to understanding the mechanism of mechano-electrical transduction and possibly finding a genetic target to prevent non-syndromic deafness as well as that associated with Usher syndrome type 1, Ahmed explained.

Their work appears in the Sept. 30 advance online edition of the journal Nature Genetics. Researchers from the National Institute on Deafness and other Communication Disorders (NIDCD), the Baylor College of Medicine and the University of Kentucky were also involved in the study, which was funded by the NIDCD, the National Science Foundation (NSF), and the Research to Prevent Blindness Foundation.

See the article here:
New Gene Associated With Hearing Loss Discovered

Washington, October 1 (ANI): Researchers have discovered a new genetic mutation responsible for deafness and hearing loss associated with Usher syndrome type 1.

These findings could help researchers develop new therapeutic targets for those at risk for this syndrome.

For the study, researchers at the University of Cincinnati (UC) and Cincinnati Children's Hospital Medical Center work together with the National Institute on Deafness and other Communication Disorders (NIDCD), Baylor College of Medicine and the University of Kentucky.

Usher syndrome is a genetic defect that causes deafness, night-blindness and a loss of peripheral vision through the progressive degeneration of the retina.

"In this study, researchers were able to pinpoint the gene which caused deafness in Usher syndrome type 1 as well as deafness that is not associated with the syndrome through the genetic analysis of 57 humans from Pakistan and Turkey," said Zubair Ahmed, PhD, assistant professor of ophthalmology who conducts research at Cincinnati Children's and is the lead investigator on this study.

Ahmed stated that a protein, called CIB2, which binds to calcium within a cell, is associated with deafness in Usher syndrome type 1 and non-syndromic hearing loss.

"To date, mutations affecting CIB2 are the most common and prevalent genetic cause of non-syndromic hearing loss in Pakistan. However, we have also found another mutation of the protein that contributes to deafness in Turkish populations," he said.

"In animal models, CIB2 is found in the mechanosensory stereocilia of the inner ear-hair cells, which respond to fluid motion and allow hearing and balance, and in retinal photoreceptor cells, which convert light into electrical signals in the eye, making it possible to see," explained Saima Riazuddin, PhD, assistant professor in UC's department of otolaryngology who conducts research at Cincinnati Children's and is co-lead investigator on the study.

Researchers found that CIB2 staining is often brighter at shorter row stereocilia tips than the neighboring stereocilia of a longer row, where it may be involved in calcium signaling that regulates mechano-electrical transduction, a process by which the ear converts mechanical energy-or energy of motion-into a form of energy that the brain can recognize as sound.

"With this knowledge, we are one step closer to understanding the mechanism of mechano-electrical transduction and possibly finding a genetic target to prevent non-syndromic deafness as well as that associated with Usher syndrome type 1," Ahmed said.

Originally posted here:
Gene that causes deafness pinpointed