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Archive for the ‘Gene Therapy Research’ Category

Takeda and Seattle Genetics Highlight Post-Hoc Analysis Examining Progression-free Survival with ADCETRIS® …

LUGANO, Switzerland--(BUSINESS WIRE)--

Takeda Pharmaceutical Company Limited (TSE:4502) and Seattle Genetics, Inc. (SGEN) today announced data from a post-hoc analysis examining progression-free survival (PFS) following treatment with ADCETRIS (brentuximab vedotin) versus last prior therapy in patients diagnosed with relapsed or refractory Hodgkin lymphoma (HL) post-autologous stem cell transplant (ASCT) or relapsed or refractory systemic anaplastic large cell lymphoma (sALCL). The data were highlighted during a presentation at the 12th International Conference on Malignant Lymphoma (ICML) being held June 1922, 2013 in Lugano, Switzerland.

ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of classical HL and sALCL.

The post-hoc analysis compared investigator assessed PFS following ADCETRIS single-agent treatment to the last prior systemic therapy in patients taking part in two pivotal Phase 2 studies. The post-hoc analysis was conducted in patients with relapsed or refractory HL post-ASCT or relapsed or refractory sALCL in the intent-to-treat (ITT) population. It also included prior systemic treatment histories and post-ADCETRIS stem cell transplant experience for each patient in the ITT populations.

These encouraging data suggest that ADCETRIS may delay disease progression compared to prior therapies used in this heavily pretreated patient population, said John Radford, M.D., Professor of Medical Oncology, University of Manchester, Manchester, UK. ADCETRIS is a CD30-targeted treatment option for patients with relapsed or refractory HL or relapsed or refractory sALCL that has shown a high overall response rate, including durable complete responses in both of its approved indications.

Progression-free survival analyses of two pivotal phase 2 studies of brentuximab vedotin in patients with relapsed or refractory Hodgkin lymphoma or systemic anaplastic large-cell lymphoma (Poster #303)

The analysis, presented by Dr. Radford, included:

Relapsed or Refractory HL post-ASCT

Relapsed or Refractory sALCL

Details of the poster presentation are as follows:

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Takeda and Seattle Genetics Highlight Post-Hoc Analysis Examining Progression-free Survival with ADCETRIS® ...

No danger of cancer through gene therapy virus, study suggests

June 19, 2013 In fall 2012, the European Medicines Agency (EMA) approved the modified adeno-associated virus AAV-LPL S447X as the first ever gene therapy for clinical use in the Western world. uniQure, a Dutch biotech company, had developed AAV-LPL S447X for the treatment of a rare inherited metabolic disease called lipoprotein lipase deficiency (LPLD) which affects approximately one or two out of one million people. The disease causes severe, life-threatening inflammations of the pancreas. Afflicted individuals carry a defect in the gene coding for the lipoprotein lipase enzyme which is necessary for breakdown of fatty acids. AAV-LPLS447X shall be used as a viral vector to deliver an intact gene copy to affected cells.

The viruses modified for gene therapy cannot integrate their DNA into the host cell genome, because they lack a particular enzyme needed for this. Nevertheless, integration may happen occasionally. "We had to exclude that AAV-LPLS447X tends to integrate at sites in the genome where this integration might activate cancer-promoting genes. This is exactly what had been observed with a virus used for gene therapy," says Dr. Manfred Schmidt, a molecular biologist. Schmidt leads a research group at NCT Heidelberg and DKFZ that studies the safety of gene-therapeutic methods.

In collaboration with scientists from uniQure, the Heidelberg researchers analyzed the genome of five LPLD patients who had been treated with AAV-LPLS447X . In addition, they also studied mice following intramuscular or intravenous administration of the therapeutic virus.

The analysis of 15 million individual genomes of five treated patients showed, as expected, that AAV-LPLS447X rarely integrates into the genome of the host cells (fewer than 1 out of 1,000 AAV-LPLS447X particles). In most cases, the viral genome persists in the cytoplasm as a separate structure. If it is integrated, this happens at random sites. The researchers did not find any tendency for integration at particular sites in the genome.

Christine Kaeppel and Raffaele Fronza, first authors of the article, were very surprised to discover the AAV-LPLS447X genome in the so-called mitochondrial genome. Mitochondria are tiny membrane-enclosed structures that generate energy for the cell. They are the only cellular component aside from the nucleus containing DNA. "An adeno-associated virus has never before been observed to integrate into the mitochondrial genome on its own," reported the scientists.

"For the first time, we have thoroughly analyzed in AAV-treated patients whether and where the viral genome integrates. Now we can regard AAV-LPLS447X as safe. Those few cases where we have observed integration of viral DNA in muscle cells are barely relevant in view of all the reconstructions and rearrangements that are permanently taking place in our DNA anyway," says study director Schmidt.

AAV-LPLS447X is considered to be a prototype vector for gene therapy. "If AAV-LPLS447X stands the test, other gene therapies against more common diseases such as Huntington's disease or Parkinson's might also become possible," says Schmidt. In addition, a growing number of diseases have been found to be linked to alterations in mitochondrial genes. The newly discovered property of the AAV vector might also prove useful for correcting genetic defects in human mitochondrial DNA.

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No danger of cancer through gene therapy virus, study suggests

No danger of cancer through gene therapy virus

Public release date: 19-Jun-2013 [ | E-mail | Share ]

Contact: Dr. Sibylle Kohlstdt s.kohlstaedt@dkfz.de Helmholtz Association of German Research Centres

In fall 2012, the European Medicines Agency (EMA) approved the modified adeno-associated virus AAV-LPL S447X as the first ever gene therapy for clinical use in the Western world. uniQure, a Dutch biotech company, had developed AAV-LPL S447X for the treatment of a rare inherited metabolic disease called lipoprotein lipase deficiency (LPLD) which affects approximately one or two out of one million people. The disease causes severe, life-threatening inflammations of the pancreas. Afflicted individuals carry a defect in the gene coding for the lipoprotein lipase enzyme which is necessary for breakdown of fatty acids. AAV-LPLS447X shall be used as a viral vector to deliver an intact gene copy to affected cells.

The viruses modified for gene therapy cannot integrate their DNA into the host cell genome, because they lack a particular enzyme needed for this. Nevertheless, integration may happen occasionally. "We had to exclude that AAV-LPLS447X tends to integrate at sites in the genome where this integration might activate cancer-promoting genes. This is exactly what had been observed with a virus used for gene therapy," says Dr. Manfred Schmidt, a molecular biologist. Schmidt leads a research group at NCT Heidelberg and DKFZ that studies the safety of gene-therapeutic methods.

In collaboration with scientists from uniQure, the Heidelberg researchers analyzed the genome of five LPLD patients who had been treated with AAV-LPLS447X . In addition, they also studied mice following intramuscular or intravenous administration of the therapeutic virus.

The analysis of 15 million individual genomes of five treated patients showed, as expected, that AAV-LPLS447X rarely integrates into the genome of the host cells (fewer than 1 out of 1,000 AAV-LPLS447X particles). In most cases, the viral genome persists in the cytoplasm as a separate structure. If it is integrated, this happens at random sites. The researchers did not find any tendency for integration at particular sites in the genome.

Christine Kaeppel and Raffaele Fronza, first authors of the article, were very surprised to discover the AAV-LPLS447X genome in the so-called mitochondrial genome. Mitochondria are tiny membrane-enclosed structures that generate energy for the cell. They are the only cellular component aside from the nucleus containing DNA. "An adeno-associated virus has never before been observed to integrate into the mitochondrial genome on its own," reported the scientists.

"For the first time, we have thoroughly analyzed in AAV-treated patients whether and where the viral genome integrates. Now we can regard AAV-LPLS447X as safe. Those few cases where we have observed integration of viral DNA in muscle cells are barely relevant in view of all the reconstructions and rearrangements that are permanently taking place in our DNA anyway," says study director Schmidt.

AAV-LPLS447X is considered to be a prototype vector for gene therapy. "If AAV-LPLS447X stands the test, other gene therapies against more common diseases such as Huntington's disease or Parkinson's might also become possible," says Schmidt. In addition, a growing number of diseases have been found to be linked to alterations in mitochondrial genes. The newly discovered property of the AAV vector might also prove useful for correcting genetic defects in human mitochondrial DNA.

###

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No danger of cancer through gene therapy virus

Cell Therapy for Cardiovascular Disease – Video


Cell Therapy for Cardiovascular Disease
Thomas J. Povsic, MD, PhD Assistant Professor of Medicine Division of Cardiology.

By: DukeClinicalResearch

Continued here:
Cell Therapy for Cardiovascular Disease - Video

Wellington Chen, M.D. of Advanced Rejuvenation Introduces Stem Cell Therapy For OsteoArthritis & COPD in Sarasota …

Sarasota, Florida (PRWEB) June 18, 2013

After almost 20 years of performing regenerative treatments in the field of non surgical orthopedics, Wellington Chen, M.D., will begin conducting clinical trials for many degenerative diseases using adipose-derived stem cell therapy in Sarasota, Florida. The independent review board of the International cell medicine society is responsible for overseeing these trials.

Advanced Rejuvenation will treat patients suffering from chronic obstructive pulmonary disease (COPD) and osteoarthritis following the IRB-approved protocols. Advanced Rejuvenation will be using adult autologous stem cells, harvested from the patients own adipose (fat) tissue or bone marrow if fat is not available. Because patients are receiving their own cells, there is no risk of rejection. As of 2007, over 9,000 studies have shown the safety using these cell lines.

Autologous stem cell therapy are your bodies repair men. They are circulated throughout your body and as soon as there is a need for them, chemical messages trigger them to migrate to the area and do their magic. They are both immune modulating and also regenerative which makes them a great therapeutic agent for osteoarthritis and COPD. Numerous studies have shown them to have the capacity to grow new cartilage, muscle, ligaments, glands and even organs. We believe stem cell treatments will become the future of care for most orthopedic problems avoiding the need for surgery. With COPD, when stem cells are run into the blood stream through an IV they will mostly pass through the lungs. We are excited to be apart of these research studies.

Advanced Rejuvenation trained under scientist Kristin Comella, CEO of Stemlogix. She was recently named in the Wall Street Journal as one of the 50 most influential people on stem cell research. Advanced Rejuvenation will implement Stemlogixs patented extraction process, allowing for an exceptionally high yield and viability of stem cells from fat.

During the in-office and same day procedure, a mini liposuction is performed. A half of a cup of fat in harvested from around the abdominal region which produces approximately 8 million stem cells. The stem cells are isolated put back into the patients joints or with COPD via an IV infusion. Local anesthesia is all that is needed and pain medication can be prescribed but is rarely necessary.

Advanced Rejuvenation has treated various orthopedic conditions for 4 years using fat transfer and now offers these treatments to patients ranging from NFL players to retired golfers. If you would like more information, e-mail Advanced Rejuveantion at AskDrGecko(at)Gmail(dot)com or call our office.

About Advanced Rejuvenation

Advanced Rejuvenation is a multi specialty practice in Sarasota, Florida, specializing in regenerative treatments such as Stem Cell Treatments, Prolotherapy, Ozone Therapy, Naturopathic, Acupuncture, Chiropractic Functional Neurology, Osteopathy, Functional Medicine, Active Isolated Stretching (AIS)

Contact: Advanced Rejuvenation Phone: (941) 330-8553 E-mail: AskDrGecko(at)Gmail(dot)com Website: http://www.SarasotaStemCell.com Office address: 2033 Wood Street #210 Sarasota, Florida 34237

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Wellington Chen, M.D. of Advanced Rejuvenation Introduces Stem Cell Therapy For OsteoArthritis & COPD in Sarasota ...

Biotechs following Supreme Court gene ruling: What, us worry?

Some biotechnology companies have grumbled about the legal challenge to their right to patent genes, arguing that taking away that right could impede innovation. But now that the US Supreme Court has ruled companies cant patent genes, the decision has been largely met with a collective shrug from the biopharmaceutical industry.

Thats because the high court ruling continues to allow drug companies to patent the technologies that enable them to turn genetic research into medicines. The latest industry executive to weigh in is Millennium Pharmaceuticals new president, Anna Protopapas, who said in an interview that the court decision wont affect her Cambridge companys pipeline of cancer drugs in late-stage clinical trials nor its early-stage research on new experimental therapies.

Its my understanding that the decision was quite narrow, Protopapas said. The focus of Millennium has always been on understanding how to use biological information in genes to develop meaningful drugs. And that wont change.

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Biotechs following Supreme Court gene ruling: What, us worry?

VGTI Florida Scientists to Receive $3.6 Million in NIH Research Grants

PORT ST. LUCIE, Fla.--(BUSINESS WIRE)--

Scientists at the Vaccine & Gene Therapy Institute of Florida (VGTI Florida) have been awarded three new federal research grants that will further support the Institutes mission of Translating Research Into Health.

Dr. Elias Haddad, Ph.D., Associate Member and part of the HIV Vaccine Group at VGTI Florida has been awarded a 5-year, $3.17 million R01 grant from the National Institutes of Health (NIH) for work on Boosting anti-HIV immunity through manipulation of Tfh (follicular helper T cells) Function.

This is VGTI Floridas first prime R01 award from the NIH. Dr. Haddads findings on a major defect in a particular T cell subset, the follicular helper T cells, that is a component in the response to vaccines, were published earlier this year in the March 10 issue of Nature Medicine.

We are getting close to the understanding of the pathology contributing to persistent HIV/AIDS infection, said Dr. Haddad. This grant will support further research into understanding of the dysfunction of the immune system in HIV infection.

In addition, Dr. Ted Ross, Ph.D., Full Member and Program Director of Vaccine Development and Viral Pathogenesis at VGTI Florida, has been awarded two research grants. A two-year, $129 thousand subcontract to a NIH grant to the University of Texas Medical Branch (Prime Awardee) on Mucosal Vaccine against Ebola and Marburg Viruses. These pathogens require Biosafety Level 4 facilities and so we will not be doing any work at VGTI Florida on the viruses themselves, said Dr. Ross. My laboratory will engineer vaccines on the computer to optimize the antigen sequences to allow development of a safe vaccine.

His second award is a two-year, $198 thousand subcontract to a NIH grant awarded to Vanderbilt University (Prime Awardee) on Human Neutralizing Monoclonal Antibodies for Rift Valley Fever Virus. At VGTI Florida, my research group will screen human antibodies for the ability to neutralize this highly pathogenic virus, which is a threat to not only humans as a biodefense agent, but also livestock in the United States, said Dr. Ross. An outbreak of Rift Valley fever virus would be devastating to the cattle and would have a high mortality rate in infected people. Identifying these neutralizing antibodies is the first step to designing a safe and effective vaccine, he added.

These awards demonstrate the ability of VGTI Florida scientists to garner federal grant monies and bring them to the State of Florida, said Dr. Jay Nelson, CEO and Founder of VGTI Florida. We made a commitment to the State to recruit top scientists and develop robust research programs to serve the public good and we are achieving these objectives.

Our recruiting efforts are in full swing and these substantial grant awards validate our strategies and follow-through in growing a sustainable institute in Port St. Lucie and expanding the life science industry in the state of Florida, said Mel Rothberg, Chief Operating Officer of VGTI Florida.

About VGTI Florida:

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VGTI Florida Scientists to Receive $3.6 Million in NIH Research Grants

‘Mountain Astrologer’ Magazine Publishes Investigative Feature on History of Monsanto and Genetic Engineering of Food

KINGSTON, N.Y., June 18, 2013 /PRNewswire/ --How did Monsanto become so powerful? How is such a relatively small company so influential?

These are among the questions asked in "In Through the Out Door: The Cosmic Signature of Monsanto and GMOs," an investigative feature written by Eric Francis Coppolino, editor of Planet Waves.

Journalists and writers may request the article by emailing press@planetwaves.net. You'll receive a reply with a link to the PDF.

Published in the August/September edition ofThe Mountain Astrologer (on stands July 1), the article tracks the history of Monsanto from its founding in 1901 to the present, including the recent discovery of illegal GMO wheat in Oregon and the recent March Against Monsanto.

Coppolino has covered Monsanto since 1991, publishing his work in Sierra magazine, The Las Vegas Sun andThe Village Voice. The New York Times oncesaid that Coppolino"has a hunger for government documents that rivals that of Seymour Hersh." His astrology has appeared throughout the English-speaking world from the UK's The Daily Mail to Australia's Harper's Bazaar.

One of the world's best-known astrologers covering politics and government, Coppolino makes no predictions about what might happen to Monsanto or its GMO enterprise. Rather he uses astrology to analyze the company's history, influence and staying power.

Quoting documents obtained through the legal discovery process, the article documents Monsanto's history as a chemical firm in prior decades, including alleged manipulation of cancer research.

It then tracks the many twists of Monsanto's rise as the leading purveyor of genetically modified crops, including the patents for the New Leaf Potato, Bovine Growth Hormone and Bt corn.

Carol van Strum, author of A Bitter Fog, helped research and fact-check the article.

"This is an unusual perspective on Monsanto that's never been written before," said Tem Tarriktar, publisher of The Mountain Astrologer. "Due to his history covering the company and his track record as a news astrologer, Eric was the one writer to take on this story."

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'Mountain Astrologer' Magazine Publishes Investigative Feature on History of Monsanto and Genetic Engineering of Food

Genetic Testing Company Should Free Data

Now that the Supreme Court has ruled that merely isolating a DNA sequence does not make it eligible to patent, the question arises, What will happen to the crucially important data accumulated by an overly broad monopoly?

The answer to this question has implications for people who may have an inherited risk for breast and ovarian cancer and to the scientists who hope to use that data for life-saving decisions about cancer surgery.

For the past 15 years, Myriad Genetics of Salt Lake City, Utah, has performed more than a million diagnostic tests for mutations in two genes -- BRCA1 and BRCA2 -- that are associated with inherited risk of breast and ovarian cancer. The Supreme Court ruling means Myriad has enjoyed a 15-year monopoly far broader than it should have been. The company now faces a fateful decision about what to do with that data it collected as a monopolist.

Will Myriads data be kept as proprietary assets to give Myriad a leg up on the competition that has already arisen, with four companies announcing after the Courts decision they will introduce BRCA genetic testing? Or will Myriad share the data so others outside Myriad can know the basis for interpreting the tests?

Interpreting test results, regardless of what laboratory does the testing, depends crucially on access to data and objectively verifiable interpretation of test results. Data about test results and their clinical significance should be publicly available and objectively verifiable, not secreted in a proprietary database.

Women (and some men) sending their samples to Myriad were not told their data would be kept as trade secrets. When there was a legal presumption of patent-enforceable monopoly, it would not have mattered. But now it does.

Accumulating the data was perfectly legal; and exclusive rights are the very purpose of patents. Moreoever, there is no law against hoarding data of commercial value; indeed, state laws protect trade secrets.

But data about peoples cancer risk are not the same as the secret formula for Coca-Cola. If someone wants a cola, she can buy Pepsi or Diet Rite. But for the past 15 years, Myriad has offered the only commercial test for BRCA mutations in the United States. That means the data about BRCA mutations flowed to Myriad and there they stopped.

Now the Court has ruled that the data are not protected by patent. But the data remain in private hands, and that is wrong.

Just to be clear, Myriad does a good job of testing, and its prices are in line with other tests of similar type. Myriad reports results quickly and clearly. It has committed resources to educating women at risk and their doctors about its tests. It works with its customers to secure payment, so that the vast majority have at least the initial test covered by insurance or a health plan. And Myriad will even do free testing for families harboring mutations whose clinical significance is unknown and for some customers without health coverage (.5 percent of its tests have been done at no cost to customers).

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Genetic Testing Company Should Free Data

Similar genetic variation found in overweight newborns and adults

Public release date: 18-Jun-2013 [ | E-mail | Share ]

Contact: Aaron Lohr alohr@endocrine.org 240-482-1380 The Endocrine Society

Similar genetic variations occur in both overweight newborns and obese adults, a large study finds. The results will be presented Tuesday at The Endocrine Society's 95th Annual Meeting in San Francisco.

"Our data suggest that adult obesity and newborn adiposity share, in part, a common genetic background," said study lead author Reeti Chawla, MD, fellow in pediatric endocrinology at Ann & Robert H. Lurie Children's Hospital of Chicago and the Northwestern University Feinberg School of Medicine, in Chicago, IL. "Allowing earlier identification of high-risk newborns may allow for earlier interventions to take place to possibly prevent obesity later in life. "

Obesity has become an epidemic worldwide. In the United States alone, more than one-third of adults are obese, according to the Centers for Disease Control and Prevention. Excess weight and obesity are related to numerous health problems, including heart disease, type 2 diabetes, stroke and some cancers. Since being obese in childhood increases the risk of adult obesity, medical researchers are interested in identifying early risk factors, or genetic markers, to help predict who is at greater risk for weight gain.

One of these genetic markers is called a single nucleotide polymorphism, or SNP, which is a naturally occurring genetic variant within the general population. In this case, investigators used SNPs related to adult obesity to identify genetic markers associated with higher newborn weight and skinfold thickness.

Investigators were able to identify 144 SNPs associated with birth weight or skinfold thickness. Since some of these 144 SNPs are closely linked and inherited together, they then narrowed the group down to 45 SNPs that are related to higher fat among newborns.

Investigators obtained the genetic data of 4,465 newborns from a large, multi-ethnic study examining the association between maternal blood-sugar levels and risk of poor pregnancy outcome. The study, called the Hyperglycemia and Adverse Pregnancy Outcomes, or HAPO, comprised mothers and infants from diverse ethnic backgrounds, including 1,095 Afro-Caribbean, 1,363 European, 616 Mexican-American and 1,207 of Thai descent.

Chawla said that she and her team now are using the 45 SNPs identified in this study to develop a genetic risk score "to determine whether bearing a large number of these SNPs predicts which newborns are at risk for increased fat at birth and, potentially, obesity later in life."

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Similar genetic variation found in overweight newborns and adults

‘Genetic Score’ for Newborns May Predict Adult Obesity

Findings may lead to the ability to give newborns a "genetic score" on obesity risk factors.

A new study suggests that overweight newborns may share the same genetic marker for a higher risk for obesity later in life, which may eventually allow doctors to identify those predisposed to obesity as soon as they're born.

[READ: Gene Research May Help Spot Baby's Obesity Risk]

Reeti Chawla, a pediatric endocrinologist with the Northwestern University Feinberg School of Medicine, says that she and her team were able to identify 45 genetic markers that are linked to both obesity in adults and are commonly found in babies with large birth weights.

In the United States, babies that are 8 pounds, 13 ounces or more are generally considered to be "large for gestational age."

"Obesity is such a complex trait that obviously has a lot of environmental components, but it appears babies born large have an increased risk for obesity later in life," Chawla says.

The study was based on the Hyperglycemia and Adverse Pregnancy Outcomes, a study that looked at more than 4,400 newborns and their mothers' blood sugar levels. Subsequent studies will follow these children as they grow up.

[ALSO:More Evidence Links BPA to Childhood Obesity]

She says that the findings, which she discussed Tuesday at The Endocrine Society's annual meeting in San Francisco, may lead to the ability to give newborns a "genetic score" on obesity risk factors. Though Chawla says it's going to be tough to find a doctor who is overly concerned about a newborn gaining weight, she says that if parents know their child is at risk for obesity, they can make earlier interventions to make sure they remain healthy.

"I think we'll be able to say 'Maybe we need to follow these kids more closely,' or 'Maybe the weight guidelines should be different for those newborns,'" she says.

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'Genetic Score' for Newborns May Predict Adult Obesity

The Zacks Analyst Blog Highlights: Myriad Genetics, Anika Therapeutics, Biogen Idec , Alexion Pharmaceuticals and …

For Immediate Release

Chicago, IL June 18, 2013 Zacks.com announces the list of stocks featured in the Analyst Blog. Every day the Zacks Equity Research analysts discuss the latest news and events impacting stocks and the financial markets. Stocks recently featured in the blog include Myriad Genetics Inc. (MYGN-Free Report), Anika Therapeutics Inc. (ANIK-Free Report), Biogen Idec Inc. (BIIB-Free Report), Alexion Pharmaceuticals, Inc. (ALXN-Free Report) and Cabot Oil & Gas Corporation (COG-Free Report).

Today, Zacks is promoting its ''Buy'' stock recommendations. Get #1Stock of the Day pick for free.

Here are highlights from Mondays Analyst Blog:

Myriad: End to Legal Battle Over Patents

The much hyped Supreme Court ruling regarding legal claims that challenge its patent landscape is out for Myriad Genetics Inc. (MYGN-Free Report) and it seems like a halfway triumph for this molecular diagnostic company.

The Issue

In Nov 2012, Myriad revealed that the U.S. Supreme Court would review the decision of U.S. Court of Appeals for the Federal Circuit. It applies to the companys composition of matter claims that isolated DNA of the BRCA1 and BRCA2 genes are patentable. Earlier, in Apr 2012, U.S. Court of Appeals for the Federal Circuit declared that Myriads composition of matter claims covering isolated DNA of the BRCA 1 and BRCA 2 genes were patentable.

Following the Supreme Court review, earlier ruling by the Federal Circuit Court of Appeals to declare the patentability of Myriads composition of matter claims covering isolated DNA of the BRCA 1 and BRCA 2 genes were subject to legal scrutiny once again. The legal claims pertain to the companys flagship Bracanalysis test. The decision was anticipated in Jun 2013.

The Verdict

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The Zacks Analyst Blog Highlights: Myriad Genetics, Anika Therapeutics, Biogen Idec , Alexion Pharmaceuticals and ...

Fixing Muscle Imbalances and Muscle Attachment (Genetics) – Video


Fixing Muscle Imbalances and Muscle Attachment (Genetics)
I cover four points in this video. 1. Muscle Attachment and tendon length are predetermined by your genetics. 2. Fixing Muscle Imbalances is possible by lean...

By: Fortress

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Fixing Muscle Imbalances and Muscle Attachment (Genetics) - Video

Dog Genetics Spur Scientific Spat

The issue of when or where canines were domesticated has geneticists in a tug of war. Image: Les Hirondelles Photography/Flickr/Getty Images

How does a Venus flytrap know when to snap shut? Can it actually feel an insects tiny, spindly legs? And how do cherry blossoms know when to bloom? Can they...

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Scientists investigating the transformation of wolves into dogs are behaving a bit like the animals they study, as disputes roil among those using genetics to understand dog domestication.

In recent months, three international teams have published papers comparing the genomes of dogs and wolves. On some matters such as the types of genetic changes that make the two differ the researchers are more or less in agreement. Yet the teams have all arrived at wildly different conclusions about the timing, location and basis for the reinvention of ferocious wolves as placid pooches. Its a sexy field, says Greger Larson, an archeogeneticist at the University of Durham, UK. He has won a 950,000 (US$1.5-million) grant to study dog domestication starting in October. Youve got a lot of big personalities, a lot of money, and people who want to get their Nature paper first.

In January, Erik Axelsson and Kerstin Lindblad-Toh, geneticists at Uppsala University in Sweden, and their colleagues reported in Nature that genes involved in the breaking down of starch seemed to set domestic dogs apart from wild wolves. In the paper and in media interviews, the researchers argued that dog domestication was catalyzed by the dawn of agriculture around 10,000years ago in the Middle East, as wolves began to loiter around human settlements and rubbish heaps (see Nature http://doi.org/mv4; 2013).

But Larson, who has worked with Lindblad-Toh on other projects, says that their claim is dubious. He notes that bones that look similar to those of domestic dogs predate the Neolithic revolution by at least several thousand years, so domestication must have occurred before then. Why waste space [in a paper] saying something that is patently untrue? he says.

Axelsson concedes that the changes in starch digestion in dogs could have occurred after they were domesticated. But he also counters that the Neolithic era lasted for thousands of years, and that dogs may have been domesticated during the earliest steps towards agrarian life when human hunter-gatherers settled down and began eating more starch-rich wild plants.

A second study, published last month in Nature Communications, argues that dogs were domesticated 32,000years ago when they began scavenging with Palaeolithic humans in southern China. A team led by Ya-ping Zhang at the Kunming Institute of Zoology in China drew that conclusion from studying the whole genomes of several grey wolves, modern European dog breeds and indigenous Chinese dogs.

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Dog Genetics Spur Scientific Spat

Mutant virus used to treat blindness

Scientists have discovered and amplified an evolved adeno-associated virus (AAV) to target and deliver gene therapy to retinal cells in mice. This discovery could lead to less invasive and more precise gene therapies for retinal degeneration diseases such as retinoschisis and Lebers congenital amaurosis, as well as disease in other organs that normally couldnt be treated with gene therapy because of the lack of tissue specific drug delivery systems.

This study, which was published in Science Translational Medicine and reported by TheScientist.com, is still in animal trials but has already been praised because of its potential to improve the outcome of gene therapy as well as to expand the use of gene therapy to other diseases.

For disease with genetic causes, gene therapy can be very effective because it treats the root of the problem, defective of missing genes. But one of the biggest problems with gene therapy is the delivery system: our bodies naturally digest DNA or RNA, and oftentimes the gene used for treatment breaks down before it reaches the target cells or tissues.

AAV vectors or delivery systems are very effective because it takes advantage of the virus life cycle. Most of us are aware that when viruses infect cells, as in the case of the common cold or even HIV, they destroy the cells in the process of creating more copies.

But some viruses dont destroy the cells they infect. Instead, they leave behind genes and insert them in the host DNA. Some viruses that cause cancer make use of this mechanism in order to shut down a cells natural apoptotic or self-destruct mechanism, causing the cells to grow uncontrollably and develop into cancer. Viruses like AAV often leave harmless genes and can even be manipulated to insert useful or therapeutic genes, making them great drug delivery systems.

But the problem with AAV is it often prefers to infect lung cells, making them hard to use for diseases that target other organs.

What Prof David Shaffer, research lead, and his team at the University of California in Berkley did was to isolate and propagate a version of the AAV virus that could infect other cells, in this case, retinal cells. AAV is a respiratory virus and so it evolved to infect lung epithelial cells, explained Schaffer to TheScientist.com. It never evolved to penetrate deep into tissue.

To test if it would be possible for AAV to evolve, they first injected regular AAV into the vitreous of the mouse eyes and a week later harvested cells from deep within the retina. As expected, most of the AAV didnt infect the retinal cells but a small portion eventually evolved and became capable of infecting photoreceptor cells. They then harvested these virus particles, repackaged them, and reinfected into another mouses eye. They did this six times in order to harvest enough virus variants to study and test, which they identified variant 7m8.

They then tested the effectiveness of this variant as a gene therapy vector by testing on mouse models with retinoschisis and Lebers congenital amaurosis. Both diseases are caused by missing genes, which made them ideal candidates for gene therapy through simple gene replacement. Those treated with the new AAV variant showed improved retinal function while those who were treated with regular AAV did not.

Lastly, they tested the variant on a macaque eye to see the variant would perform just as well in primates. Primates, which includes humans, have thicker retinas and the researchers had concerns that the AAV vector may not be able to penetrate deep enough into the retinal cells. The designed the AAV vector to express a fluorescent protein when it infects retinal cells and their results showed that the vector was able to target the photoreceptors up to the thinnest part of the retina or the fovea.

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Mutant virus used to treat blindness

‘Gene signature’ test diagnoses benign thyroid growths

Public release date: 15-Jun-2013 [ | E-mail | Share ]

Contact: Aaron Lohr alohr@endocrine.org 240-482-1380 The Endocrine Society

A new genetic test accurately and consistently diagnoses benign growths, or nodules, on the thyroid gland, according to a study from Chile. The results will be presented Saturday at The Endocrine Society's 95th Annual Meeting in San Francisco.

"We have developed a 'gene signature' that effectively identifies benign thyroid nodules," said Hernan Gonzalez, MD, PhD, associate professor at the Pontifical Catholic University of Chile in Santiago. "This test is potentially useful to identify patients who do not require surgery."

The thyroid gland, located in the front of the neck, secretes thyroid hormone. This hormone is involved in many bodily processes, including turning food into energy. For unknown reasons, the thyroid gland sometimes develops small nodules. Although these nodules are extremely common and usually benign, a small number are cancerous, which makes screening tests essential.

In the United States this year, slightly more than 60,000 cases of thyroid cancer will be diagnosed and about 1,850 related deaths will occur, according to estimates from the American Cancer Society. Of these diagnosed cases, more than 45,000 will occur in women, who develop the disease more often than men.

Fortunately, thyroid cancer usually is curable. Screening for thyroid cancer involves using a thin needle to take a biopsy of the growth, called a fine needle aspirate biopsy, which is then analyzed for cancer cells.

The problem with the currently available screening test is that it often yields inconclusive results. In one-fifth to one-fourth of these tests, the results are unclear. In addition to causing anxiety among patients, inconclusive tests lead to repeated tests to rule out cancer, or even to surgery, given that cancer is present in about 25 percent of inconclusive cases. As a result, three out of four patients have unnecessary surgery, leading to rising treatment costs and the risk of possible complications related to unnecessary medical procedures.

The genetic test developed by the current study accurately identified benign nodules in nearly all samples analyzed. Specifically, the test differentiated between cancerous and non-cancerous tissue in 96 percent of thyroid samples.

"For the general public, this is important since it will offer a diagnostic tool that will avoid thousands of surgeries, with a major impact in health costs, eliminating potential surgical complications and the need for permanent thyroid hormone supplementation," Gonzalez said. "In addition, it should be widely available to local labs and hospitals and at a reasonable cost for the health system."

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'Gene signature' test diagnoses benign thyroid growths

$8.7 million grant supports ‘Gene-Environment Interaction’ research

Public release date: 17-Jun-2013 [ | E-mail | Share ]

Contact: Keith Herrell keith.herrell@uc.edu 513-558-4559 University of Cincinnati Academic Health Center

CINCINNATIThe University of Cincinnati's (UC) environmental health department has received an $8.7 million federal grant to continue operating its Center for Environmental Genetics (CEG).

Led by Shuk-mei Ho, PhD, director, the CEG supports state-of-the-art core facilities and technologies needed to conduct innovative research that focuses on how environmental agents interact with genetic and epigenetic factors to influence disease risk and outcome.

This funding renewal, from the National Institutes of Health's National Institute of Environmental Health Sciences (NIEHS), will be dispersed in annual increments of about $1.74 million through March 31, 2018. The center is one of 20 Environmental Health Sciences (EHS) Core Centers funded by the NIEHS, designed to build scientific collaboration to identify toxicants in the environment, learn how these toxicants affect people's health and provide insights into preventing environmentally induced illnesses.

Additional missions of the center are to attract new talents to EHS research and empower communities to impact public health policies.

"This grant validates the important work of the CEG and will allow us to continue to conduct state-of-the-art environmental research at UC's Academic Health Center and Cincinnati Children's Hospital Medical Center," says Ho, Jacob G. Schmidlapp professor and chair of UC's environmental health department, a University System of Ohio (USO) Center of Excellence.

Founded in 1992 by Daniel Nebert, MD, now a UC professor emeritus of environmental health, the CEG encourages research collaboration between basic and applied scientists, epidemiologists and clinicians seeking to understand the complex relationships between genetic predisposition to disease and environmental exposures.

Susan Pinney, PhD, professor of environmental health, serves as deputy director of the CEG. Alvaro Puga, PhD, an associate professor of environmental health, and Daniel Woo, MD, a professor in UC's department of neurology and rehabilitation medicine, are associate directors.

"Environmental chemicals and their potential health effects are increasingly part of the public consciousness," Pinney says. "This funding will allow us to respond to questions from the public with cutting-edge science."

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$8.7 million grant supports 'Gene-Environment Interaction' research

The Jolie Gene: What does it all cost?

Last month, Angelina Jolie stunned the world with a New York Times op/ed titled, My Medical Choice. Angelina confessed she had undergone a preventative double mastectomy and breast reconstruction as a result of learning that she carries the BRCA1 gene mutation and therefore had an 87% risk of breast cancer.

Her goal of going public with this news was to encourage public discussion, awareness and acceptance around genetic screening and the preventative measures available to women (and men) at high risk of developing cancer.

Near the end of Angelinas editorial, she writes: It has got to be a priority to ensure that more women can access gene testing and lifesaving preventive treatment, whatever their means and background, wherever they live. The cost of testing for BRCA1 and BRCA2, at more than $3,000 in the United States, remains an obstacle for many women.

Which raises a critical point: Even if you can afford to get the testing done, would you be able to afford the preventative surgeries and treatments? How much does it all cost anyway?

Can you put a price on a stay-at-home mom?

Genetic screening

The actual cost for the BRCA gene mutation tests (called BRACAnalysis) depends on how extensive the testing is and can range between $400 and $4,000. The costs are the same everywhere, since all BRACAnalysis tests are conducted by one company: Myriad Genetics (MYGN) in Salt Lake City, Utah. The publicly listed biotech company has patents on the BRCA1 and BRCA2 genes and the tests for mutations in those genes is their main line of business. After Angelinas op/ed, Myriads stock rose to a three-year high.

How can a company patent genes that exist in all our bodies, you ask? Great question. In fact, this is a question currently being considered by the US Supreme Court. Opponents argue that Myriads monopoly on the tests hampers scientific research, keeps costs high and restricts testing accessibility.

In Myriads defense, the company claims that over 95% of patients are reimbursed for the cost of the tests through insurance and that if a patient is not insured and in financial need, they offer an assistance program to provide testing at a reduced cost or even free.

Unbreak my heart: The crippling effect of health struggles on your finances

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The Jolie Gene: What does it all cost?

Personalized Medicine Diagnostics (Flow Cytometry, Sepsis Immunos, Routine Coagulation, Psychiatric Disorders, Tumor …

NEW YORK, June 17, 2013 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

Personalized Medicine Diagnostics (Flow Cytometry, Sepsis Immunos, Routine Coagulation, Psychiatric Disorders, Tumor Markers, Molecular Blood Typing and Other Testing)

http://www.reportlinker.com/p01360928/Personalized-Medicine-Diagnostics-Flow-Cytometry-Sepsis-Immunos-Routine-Coagulation-Psychiatric-Disorders-Tumor-Markers-Molecular-Blood-Typing-and-Other-Testing.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=In_Vitro_Diagnostic

Personalized Medicine Diagnostics - Market Overview

Personalized Medicine is often defined as "the right treatment for the right person at the right time." Personalized medicine is becoming the place to be in clinical diagnostics as well and slowly becoming the reality of future in the diagnostics industry

By Technology Segment - Personalized Medicine diagnostics market is expected to grow with a double digit CAGR for the period of 2013 to 2018. It is expected that personalized medicine diagnostics market by technology is going to double by 2018 from its current market size in 2012. In this segment, Point of Care Testing and Molecular Diagnostics segments control the #1 and #2 positions in 2012.

By Diseases Segment - Personalized Medicine diagnostics market is expected to be more than US$ 30 Billion by 2018. Diabetes management test and Cancer management test are the leading market in this segment.

Renub Research report entitled "Personalized Medicine Diagnostics (Flow Cytometry, Sepsis Immunos, Routine Coagulation, Psychiatric Disorders, Tumor Markers, Molecular Blood Typing and Other Testing)" report provides a comprehensive analysis of the emerging personalized medicine diagnostic market segments, including their dynamics, size, market share, key investors, clinical trials statement, technological trends, company analysis and a realistic future potential for personalized medicine in clinical testing. The report also entails major drivers and challenges of personalized medicine diagnostic market. This 173 page report contains 94 Figures and 16 Tables studies the Personalized Medicine Diagnostics Market. This report contains 12 chapters

Chapter 1 is Executive Summary

Chapter 2 contains worldwide personalized medicine diagnostic market & forecast (2011 2018). The market includes technological & disease-wise personalized medicine diagnostics market

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Personalized Medicine Diagnostics (Flow Cytometry, Sepsis Immunos, Routine Coagulation, Psychiatric Disorders, Tumor ...

Genetics Shop – Video


Genetics Shop
Science is the cool and stuff.

By: FicocelliMr

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Genetics Shop - Video

Human Genetics – Blood Type Video – Video


Human Genetics - Blood Type Video
I had some trouble with iMovie but the articles referenced are as follows: cassiopaea.org https://cassiopaea.org/forum/index.ph... http://en.wikipedia.org/wi...

By: Nomad1217

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Human Genetics - Blood Type Video - Video

Atossa Genetics Announces Nationwide Agreement With Network Provider HealthSmart

SEATTLE, WA--(Marketwired - Jun 17, 2013) - Atossa Genetics, Inc. (NASDAQ: ATOS), The Breast Health Company, has entered into a contractual agreement with HealthSmart, a Preferred Provider Organization (PPO) network serving clients in all 50 states.

The agreement with HealthSmart affords preferred providers and their patients with greater access to Atossa'sForeCYTE Breast Health Test by ensuring timely reimbursement for the laboratory costs associated with the test.

"Our science-based approach to breast cancer risk assessment provides doctors and their patients with vital information concerning a woman's breast health status and risk for breast cancer," stated Dr. Steven Quay, Chairman CEO & President of Atossa Genetics. "The agreement with HealthSmart, our third agreement with a PPO organization, will help more doctors and patients access the ForeCYTE test."

About the ForeCYTE Breast Health Test

TheForeCYTE Breast Health Test 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 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. The ForeCYTE test is targeted to the 110 million women in the U.S. ages 18 to 73.

Just as the Pap smear has reduced cervical cancer rates by over 70 percent in women who receive regular testing, becoming the most successful screening test in medicine, the goal of Atossa Genetics is to reduce the stubbornly high rate of breast cancer through the early detection of the precursor changes that can lead to breast cancer and the treatment of those early changes.

About Atossa Genetics, Inc.

Atossa Genetics, Inc., The Breast Health Company, based in Seattle, WA, is focused on preventing breast cancer through the commercialization of patented, FDA-designated Class II diagnostic medical devices and patented, laboratory developed tests that can detect precursors to breast cancer up to eight years before mammography.

The National Reference Laboratory for Breast Health (NRLBH), a wholly owned subsidiary of Atossa Genetics, Inc., is a CLIA-certified high-complexity molecular diagnostic laboratory located in Seattle, Washington.

For additional information on Atossa Genetics, please visit http://www.atossagenetics.com. To see a video about Atossa and the ForeCYTE test, click here: http://vimeo.com/62214008

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Atossa Genetics Announces Nationwide Agreement With Network Provider HealthSmart

Plastic Surgery – Is Gene Therapy the Future?

Los Angeles, California (PRWEB) June 16, 2013

According to a May 29, 2013 NBC News article, titled Gene Therapies for Regenerative Surgery Are Getting Closer, Says Review in Plastic and Reconstructive Surgery, researchers have made significant progress in the development of gene therapy techniques that can grow skin, bone and tissue for reconstructive surgery. Researchers from Padua University Hospital in Italy who conducted the review claim the potential benefits of using gene therapy in reconstructive surgery are numerous. (Go to goo.gl/kYjFa)

According to the article, gene therapy may be instrumental in solving a problem that most plastic and reconstructive surgeons face, which is the lack of sufficient tissue to correct deformities that patients have in certain areas of their body. For example, while most surgeons can effectively treat small burns, they often face major challenges while trying to treat patients with large burns, because such patients generally lack sufficient tissue to develop skin flaps that can adequately cover the affected area(s).

What this means, says Dr. Simon Ourian, Medical Director of Epione Beverly Hills, is that despite undergoing plastic surgery, some patients still need to use cosmetics to improve the appearance of their burns. However, by using gene therapy to promote the growth of specific tissue, reconstructive surgeons can, in future, improve the quality of plastic surgery treatments.

Further according to the report, in addition to improving the effectiveness of regenerative surgery through growth of different tissue, gene therapy can be used to control the growth factors that aid in skin healing, in the bone formation process, and the regeneration of injured nerves.

I look forward to seeing the results of future research and hope that the day we have practical ways to apply this technology isnt too far off, says Dr. Ourian.

Dr. Ourian has been a pioneer in laser technology and non-invasive aesthetic procedures including Restylane, Juvderm, Radiesse and Sculptra. These treatments are used for the correction or reversal of a variety of conditions such as acne, acne scars, skin discoloration, wrinkles, stretch marks, varicose veins, cellulite, and others. More information about gene therapy can be found on Epiones website.

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Plastic Surgery – Is Gene Therapy the Future?

Clinical Grade RetroNectin® Reagent Available To Support Gene Therapy Clinical Research

MOUNTAIN VIEW, Calif., June 17, 2013 /PRNewswire/ --In an effort to aid progress in gene therapy clinical research, representatives of Clontech laboratories, Inc. and its parent company Takara Bio Inc. announce the availability of clinical grade RetroNectin reagent for direct supply to biomedical researchers.

RetroNectin reagent is designed to enable efficient retroviral transduction of genes into hematopoietic stem cells as well as lymphocytes and other blood cells. The RetroNectin method has been recognized as a standard gene transduction method in ex vivo gene therapy around the world. In addition, RetroNectin reagent has another remarkable feature that can also be useful for cell therapies: during the expansion culture of human T lymphocytes, RetroNectin reagent helps to increase proportion of nave T cells. This RetroNectin induced T cell method has already become available as a cancer therapy in three Japanese clinics under technical support from Takara Bio.

Takara Bio is the exclusive supplier of RetroNectin reagent, a recombinant human fibronectin fragment developed in 1995 by Takara Bio in collaboration with Indiana University. It has been used in 68 gene therapy clinical trials in 44 institutes and hospitals in 10 countries to date.

Previously, access to clinical-grade RetroNectin reagent required a Material Transfer Agreement (MTA) between a research institution and Takara Bio. Researchers may now submit direct orders to Clontech or local Takara Bio subsidiaries for RetroNectin (GMP), which is manufactured as a quality-assured product according to guidelines for Good Manufacturing Practice (GMP). The Drug Master File for RetroNectin (GMP) has been filed with the U.S. Food and Drug Administration. In a recent study published in Science Translational Medicine in March 2013, scientists at Memorial Sloan-Kettering Cancer Center reported an immunotherapy strategy for the treatment of five adult patients with acute lymphoblastic leukemia. Each patient's T cells were extracted, altered by introduction of DNA that would cause the cells to attack tumor cells, and infused back into the patient's bloodstream. According to researchers, all patients achieved tumor eradication and complete remission. RetroNectin reagent was used during T cell transduction.

Corresponding author Dr. Renier J. Brentjens said, "It was very clear to us even 10 years ago that the use of RetroNectin coated plates markedly, massively improved gene transfer." Dr. Brentjens continued, "The methodologies that many of us now use have been developed over a number of years. Once you have a system that works, you become very reliant and dependent on those reagents."

"RetroNectin reagent has become a standard reagent for many gene transfer protocols worldwide," said Carol Lou, General Manager of Clontech. "We are sure that such direct access to RetroNectin (GMP) without MTA execution will make this reagent available much more easily to any scientists or clinicians interested in RetroNectin clinical applications, which aligns with Takara Bio's mission of contributing to the health of mankind through gene therapy."

About Clontech Laboratories, Inc.Clontech Laboratories, Inc., a wholly-owned subsidiary of Takara Bio Inc., develops, manufactures, and distributes a wide range of life science research reagents under the Clontech and Takara brands. Key products include the Living Colors fluorescent proteins; high-performance qPCR and PCR reagents (including theTaKaRa Ex Taq,TaKaRa LA Taq, Titanium, and Advantage enzymes); RT enzymes and SMART library construction kits; the innovative In-Fusion cloning system; Tet-based inducible gene expression systems; and a range of Macherey-Nagel nucleic acid purification tools. These and other products support applications including gene discovery, regulation, and function; protein expression and purification; RNAi and stem cell studies; and plant and food research. For more information, visit http://www.clontech.com.

About Takara Bio Inc.Takara Bio Inc. is an innovative biotechnology company based in Shiga, Japan. As a world leader in biotechnology research and development, Takara Bio was the first company to market PCR technology in Japan and is also the developer of the RetroNectin reagent, which is used as a world-standard in gene therapy protocols. In addition to providing research reagents and equipment to the life science research market, Takara Bio has active research and product development activities in the fields of gene and cell-based therapy, and agricultural biotechnology; and is committed to preventing disease and improving the quality of life for all people through the use of biotechnology. Through strategic alliances with other industry leaders, the Company aims to extend its reach around the world. More information is available athttp://www.takara-bio.com.

For more information, contact:

Lorna Neilson, Ph.D. Director, Business Development Clontech Laboratories, Inc. A TakaraBio Company lorna_neilson@clontech.com (650) 919-7372

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Clinical Grade RetroNectin® Reagent Available To Support Gene Therapy Clinical Research

Gene Therapy – Technologies, Markets and Companies 2013

DUBLIN, June 17, 2013 /PRNewswire/ --

Research and Markets (http://www.researchandmarkets.com/research/dfcq6z/gene_therapy) has announced a new report "Gene Therapy - Technologies, Markets and Companies" to its offering.

(Logo: http://photos.prnewswire.com/prnh/20130307/600769 )

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now be combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes a broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy reports written by him during the past decade, including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic.

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration, as well as targeted gene therapy, are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2012, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide. A breakdown of these trials is shown according to the areas of application.

The voluminous literature on gene therapy was reviewed and selected 750 references are appended in the bibliography. The references are constantly updated. The text is supplemented with 73 tables and 15 figures.

Profiles of 181 companies involved in developing gene therapy are presented, along with 204 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

Benefits of this report

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Gene Therapy - Technologies, Markets and Companies 2013

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