Archive for the ‘Gene Therapy Research’ Category

Public release date: 12-Feb-2013 [ | E-mail | Share ]

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

New Rochelle, NY, February 12, 2013About 16,000 women on active duty in the U.S. military give birth each year. Most return to service after 6 weeks and can be deployed after 4 months. The potential for an increased risk of depression among new mothers who deploy and are exposed to combat experience is presented in a study published in Journal of Women's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women's Health website at http://www.liebertpub.com/jwh.

Stacie Nguyen, MPH and coauthors from the Millennium Study Team, San Diego State University and Naval Hospital Camp Pendleton, California, and Uniformed Services University of the Health Sciences, Bethesda, MD, analyzed data collected as part of a large study to assess the long-term health of U.S. military personnel. The article "Is Military Deployment a Risk Factor for Maternal Depression?" describes the findings for 1,660 women who gave birth during active duty service.

Women who deployed and reported combat experience after giving birth were at increased risk for depression compared to women who did not deploy after childbirth. Among women who deployed with combat exposure, those who had given birth did not have a significantly increased risk for depression compared to those who had not, suggesting that the risk was related more to combat than to factors related to childbirth.

"With increasing numbers of women in the military and being exposed to combat experiences, it is critical for us to better understand maternal depression among our female service members," says Susan G. Kornstein, MD, Editor-in-Chief of Journal of Women's Health, Executive Director of the Virginia Commonwealth University Institute for Women's Health, Richmond, VA, and President of the Academy of Women's Health.

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About the Journal

Journal of Women's Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women's healthcare issues. Complete tables of content and a sample issue may be viewed on the Journal of Women's Health website at http://www.liebertpub.com/jwh. Journal of Women's Health is the Official Journal of the Academy of Women's Health and the Society for Women's Health Research.

About the Society

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Depression risk among women in the military deployed after childbirth

Feb. 12, 2013 Scientists at Washington University School of Medicine in St. Louis have identified a genetic mutation that may underlie common behaviors seen in some people with autism, such as difficulty communicating and resistance to change.

An error in the gene, CELF6, leads to disturbances in serotonin, a chemical that relays messages in the brain and has long been suspected to be involved in autism.

The researchers identified the error in a child with autism and then, working in mice, showed that the same genetic alteration results in autism-related behaviors and a sharp drop in the level of serotonin circulating in the brain.

While the newly discovered mutation appears to be rare, it provides some of the first clues to the biological basis of the disease, the scientists report Feb. 13 in the Journal of Neuroscience.

"Genetically, autism looks very complicated, with many different genetic routes that lead to the disease," says lead author Joseph D. Dougherty, PhD, an assistant professor of genetics at Washington University. "But it's not possible to design a different drug for every child. The real key is to find the common biological pathways that link these different genetic routes and target those pathways for treatment."

Autism is known to have a strong genetic component, but the handful of genes implicated in the condition so far explain only a small number of cases or make a small contribution to symptoms.

This led Dougherty and senior author Nathaniel Heintz, PhD, a Howard Hughes Medical Institute investigator at Rockefeller University, to speculate that some of the most common behavioral symptoms of autism may be caused by disruptions in a common biological pathway, like the one involved in serotonin signaling.

Some 30 percent of patients with autism have abnormal blood levels of serotonin. The chemical messenger plays an important role throughout the body, helping to regulate breathing, temperature and sleep as well as mood and learning. But it is produced by only a limited number of neurons in the brain.

Using a new molecular technique, the researchers exclusively targeted serotonin-producing neurons in healthy mice to look for genes that are "turned on" in these cells. They found nearly 200 genes involved in this process.

Then, they searched for variations in these genes in some 400 autistic children, whose DNA was made available to the scientists through the Autism Genetic Resource Exchange.

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Some autism behaviors linked to altered gene

Making Sense of Genetics
Cardiologists from Heidelberg University collaborate with Siemens to develop software for data analytics. Their goal: to establish next-generation genetic sequencing in patients with myocardial disease for personalized therapies and improved outcomes. Learn more http://www.siemens.com

By: Siemens

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Making Sense of Genetics - Video

Paris Jackson Looks Like Her Mother (Debbie Rowe)
Michael Jackson was a man interested in genetics, doctors found him genes for white skin! Thats why him children looks white! Him family is multiracial! So its possible!

By: kouldeliee

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Paris Jackson Looks Like Her Mother (Debbie Rowe) - Video

TAUN POOL PARTY 3
Thanks to everyone who came out and helped out! Much love x Bboys: Bboy The Curse, Silent Sam, Concrete Apostles, Genetics Breaking, Handbreakturn Bboy-crew, Luke Hall Trickers: Nathan Barris, Malcolm Wope, Mizter Banks DJ: Barry Bear Baumgaurt Thank you Smooch Beverages, Mainstay, Kiss This Purple Haze and Red Bull for making the party come alive! Quality dropped so watch in HD. Filmed with Gopro3 Black Music: Kill The Noise - My World (Brillz Remix)

By: TaunSA01

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TAUN POOL PARTY 3 - Video

Dragon Genetics Lab instructions

By: Aurelian Sennett

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Dragon Genetics Lab instructions - Video

Clay model of axolotl ectoderm contraction wave
Flip movie from Jack Butler photos of his models in: Gordon, R. (1999). The Hierarchical Genome and Differentiation Waves: Novel Unification of Development, Genetics and Evolution. Singapore London, World Scientific Imperial College Press. http://www.worldscientific.com

By: Richard Gordon

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Clay model of axolotl ectoderm contraction wave - Video

Seattle Genetics, Inc. (SGEN) reported fourth quarter 2012 net loss per share of 9 cents, narrower than the Zacks Consensus Estimate of a loss of 13 cents and the year-ago loss of 24 cents per share.

Fourth quarter revenues were $63.9 million, compared with $48.9 million in the year-ago quarter. Revenues surpassed the Zacks Consensus Estimate of $56 million. Revenues were mainly driven by collaboration and license agreement revenues and Adcetris (brentuximab vedotin) sales.

Full year 2012 net loss per share was 46 cents, narrower than the Zacks Consensus Estimate of a loss of 55 cents and the year-ago loss of $1.34 per share. Revenues in 2012 were $210.8 million, compared with $94.8 million in 2011. Revenues for 2012 were below the Zacks Consensus Estimate of $212 million.

The Quarter in Detail

Net revenues for the fourth quarter included Adcetris revenues (up 6.5% to $35.4 million), collaboration and license agreement revenues (up 68.4% to $26.4 million) and royalty revenues.

Seattle Genetics is making efforts to expand Adcetris label. In Jan 2013, a global phase III study (ECHELON-2) was initiated on Adcetris. In this study, Adcetris plus chemotherapy will be evaluated for the front-line treatment of CD30-positive MTCL including patients with sALCL and other types of peripheral T-cell lymphomas.

Earlier this month Health Canada approved Adcetris for the treatment of relapsed or refractory Hodgkin lymphoma (HL) and systemic anaplastic large cell lymphoma (sALCL). For similar indications, Adcetris was approved by the US Food and Drug Administration (:FDA) in Aug 2011 and in the EU in Oct 2012.

Seattle Genetics intends to submit a supplemental biologics license application (sBLA) in the first half of 2013 for the use of Adcetris in the retreatment of patients and for extended duration of use beyond 16 cycles of therapy.

Research and development expenses increased 18.7% year over year to $47.7 million. Selling, general and administrative expenses fell 6.4% year over year to $23.4 million.

2013 Guidance

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SGEN Narrows Loss, Sales Rise

BOTHELL, Wash.--(BUSINESS WIRE)--

Seattle Genetics, Inc. (SGEN) today reported financial results for the fourth quarter and twelve months ended December 31, 2012. The company also highlighted ADCETRIS (brentuximab vedotin) commercialization and clinical development activities, its antibody-drug conjugate (ADC) pipeline and collaborator progress as well as upcoming milestones.

We are strongly positioned to continue delivering on our goal of bringing ADCETRIS to patients through commercial execution. Additionally, we are building upon encouraging data in earlier lines of therapy and a range of CD30-positive malignancies through our clinical development and regulatory activities, said Clay B. Siegall, Ph.D., President and Chief Executive Officer at Seattle Genetics. There are more than 20 ongoing ADCETRIS clinical trials, including four phase III trials, designed to broadly explore its therapeutic potential. In addition, we have made important progress in advancing six other ADCs in clinical and preclinical development, and our ADC technology is being broadly applied in more than a dozen clinical development programs by our collaborators. Our strong financial position and cash flow from product sales and collaborations enable us to continue building value in the company.

Recent ADCETRIS Highlights

ADCETRIS is not approved in front-line HL, front-line MTCL or in DLBCL.

Other Recent Highlights

Upcoming Milestones

Fourth Quarter and Year 2012 Financial Results

Total revenues in the fourth quarter of 2012 were $63.9 million, compared to $48.9 million in the fourth quarter of 2011. Total revenues for the year ended December 31, 2012 were $210.8 million, compared to $94.8 million in 2011. Revenues in 2012 include ADCETRIS net product sales of $35.4 million in the fourth quarter and $138.2 million for the year. Revenues in 2012 reflect $2.6 million in ADCETRIS net sales to patients in Canada under a Special Access Program that were recognized in the fourth quarter. Revenues also reflect amounts earned under the companys ADCETRIS and ADC collaborations totaling $26.4 million in the fourth quarter of 2012 and $67.5 million for the year.

Research and development expenses for the fourth quarter of 2012 were $47.7 million, compared to $40.2 million for the fourth quarter of 2011. For 2012, total research and development expenses were $170.3 million, compared to $163.4 million in 2011. Selling, general and administrative expenses for the fourth quarter of 2012 were $23.4 million, compared to $25.0 million for the fourth quarter of 2011. For 2012, total selling, general and administrative expenses were $84.3 million, compared to $72.7 million in 2011. The planned increases in 2012 expenses were primarily driven by ADCETRIS commercialization activities and research and development of the companys ADC pipeline programs.

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Seattle Genetics Reports Fourth Quarter and Year 2012 Financial Results

London, Feb. 13 (ANI): Five beagles, who were suffering from diabetes, were no longer in need of insulin injections after they were given two extra genes.

Fatima Bosch, who treated the dogs at the Autonomous University of Barcelona, Spain, said that a number of attempts had earlier been made to treat the disease with gene therapy but this study is the first to show a long-term cure in a large animal, New Scientist reported.

To sense and regulate how much glucose is being circulated in the blood, the two genes have to work together in tandem.

People, who suffer from type 1 diabetes, lose this ability as their immune system kills the pancreatic cells that produce insulin.

The two genes, which were delivered into dogs' legs muscles by a harmless virus, appeared to compensate for the loss of these insulin producing pancreatic cells.

One gene created insulin while the other produced an enzyme that dictated how much glucose should be absorbed into muscles.

Dogs that received only one of the two genes remained diabetic, suggesting that both the genes are needed for the treatment to work. (ANI)

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Gene therapy cures diabetes in dogs

CARLSBAD, Calif.--(BUSINESS WIRE)--

Applied Spectral Imaging (ASI, http://www.spectral-imaging.com) announced today that its GenASIs Scan & Analysis automated microscopy platform has received FDA clearance as an aid in ALK gene analysis for lung cancer therapy selection. The introduction of automation in ALK therapy selection represents a new era in personalized medicine for patients with NSCLC (Non-Small-Cell-Lung-Cancer).

About 85% of lung cancer patients have NSCLC and are commonly diagnosed at an advanced disease stage with very low survival rate. Lung cancer is the leading cause of cancer related death in the US with over 1.6 million new cases diagnosed annually.

GenASIs Scan & Analysis offers clinicians a way to automatically perform genetic analysis on tissue samples, stained with the Abbott ALK probe kit, and identify the ALK gene rearrangement.

Automated genetic analysis with GenASIs Scan & Analysis ensures quick, repeatable and documented results proving unparalleled clinical accuracy and time savings. GenASIs further adds the ability to perform walk away automated genetic tissue scanning for ALK using an 81 slide continues scanning robot.

The Abbott Vysis ALK Break Apart FISH Probe test is designed to identify ALK-positive NSCLC patients for Pfizers approved NSCLC therapy, XALKORI (crizotinib), an oral first-in-class ALK inhibitor. The Vysis test uses in situ hybridization (FISH) technology to detect rearrangements of the ALK gene on the 2p23 chromosome. The diagnostic test offers clinicians a standardized, clinically validated method to identify patients more likely to benefit from XALKORI (crizotinib) therapy.

We are proud to be the first to offer an automated platform to aid in ALK therapy selection for Non-Small-Cell Lung Cancer patients. This significant achievement further affirms ASIs commitment to offer new and innovative tools to make personalized medicine using companion diagnostics more available and more effective. said Limor Shiposh, ASIs Chief Executive Officer.

Limor further added, ALK is an important addition to our suite of FDA cleared computerized clinical diagnostic aids. We plan to expand our portfolio of FDA cleared tools in our GenASIs platform to better serve our customers in achieving more accurate clinical results, quicker and more cost effectively. I am confident that this addition will help further establish ASI as the world leader in automated and manual FISH imaging and analysis.

About ASI

Applied Spectral Imaging (ASI) makes patient care better through advanced biomedical imaging.

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World's First FDA Cleared ALK Automated Gene Scanner for Lung Cancer Therapy-Selection Available from ASI

GAITHERSBURG, Md., Feb.13, 2013 /PRNewswire/ --Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery products, announced today that that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to P140K methylguanine methyltransferase (MGMT) transduced human CD34 cells (product name: LG631-CD34) for bone marrow protection in the treatment of glioblastoma multiforme. Orphan drug designation qualifies Lentigen for seven years of market exclusivity following marketing approval by the FDA and provides other development-related incentives.

"We are very pleased that the FDA has granted our brain cancer product orphan drug status," said Tim Ravenscroft CEO of Lentigen Corporation. "The market exclusivity which this designation provides, together with our intellectual property rights, gives us increased impetus to advance the development of this product, enabling us to play our part in improving the lives of patients with glioblastoma."

"LG631-CD34 consists of the patients' adult hematopoietic stem cells genetically modified with a Lentiviral vector expressing a human MGMT gene variant, which is designed to protect the cells from the toxic side-effects of Temodar*, a standard of care treatment for glioblastoma multiforme" said Dr. Boro Dropulic, the Chief Scientific Officer of Lentigen Corporation. "Protection of blood-forming hematopoietic stem cells from the side-effects effects of Temodar would provide immediate benefits to patients. It potentially enables higher doses and more intensive drug treatment with reduced toxicity, resulting in improved clinical outcomes.

LG631-CD34 is currently being evaluated in a NIH grant-funded Phase I clinical trial (http://www.clinicaltrials.gov/ct2/show/NCT01269424) at University Hospitals (UH) Case Medical Center under the leadership of Stanton Gerson, MD, Director of the Seidman Cancer Center at UH Case Medical Center, Director of the National Center for Regenerative Medicine and Case Comprehensive Cancer Center and Asa, Patricia Shiverick - Jane Shiverick (Tripp) Professor of Hematological Oncology at Case Western Reserve University School of Medicine and Andrew Sloan MD, Associate Professor & Vice-Chairman, Department of Neurological Surgery and the Peter D. Cristal Chair of Neurosurgical Oncology at Case Western Reserve University School of Medicine, Director, Brain Tumor and Neuro-Oncology Center, UH Case Medical Center and a member of the Case Comprehensive Cancer Center.

"Glioma is such an aggressive and challenging cancer," said Dr. Gerson. "When patients are diagnosed with this life threatening disease, they have an average life expectancy of less than 12 months. The medical community needs to find new treatment strategies that can improve clinical outcomes in this devastating disease. I am hopeful that LG631-CD34 can make an important contribution and help us improve the lives of our patients."

Dr. Stanton Gerson is an inventor on this mutant alkyltransferase technology. He, as well as Case Western Reserve University and University Hospitals, could gain from commercialization of the invention.

The clinical trial is supported by Grant Number R42CA128269 from the National Cancer Institute and a grant to the Center for Stem Cell & Regenerative Medicine (CSCRM) from Ohio's Third Frontier Commission under its Research Commercialization Program. The content of this news release is solely the responsibility of the authors and does not necessarily represent the official views of the National Cancer Institute or the National Institutes of Health.

The U.S. Orphan Drugs Act aims to encourage the development of drugs involved in the diagnosis, prevention or treatment of a medical condition affecting fewer than 200,000 people in the United States. Orphan drug designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA user fee, and the orphan status also allows the sponsor to apply for grants to support clinical trials.

About Lentigen CorporationLentigen Corporation is a privately owned biotechnology company focused on the development of lentiviral vector technology for a wide range of therapeutic, vaccine, and bioproduction applications. Lentiviral vectors are the most efficient vehicles for the delivery of genes or gene silencing sequences stably into cells. Lentigen is a highly collaborative company, co-developing Lentiviral vector-based products across a broad spectrum of bench to clinical applications. Collaborations include The National Institutes of Health, IAVI, Harvard Medical School, Expression Therapeutics, Scripps Institute, Case Western Reserve University, Loyola University Chicago, The University of Pittsburgh, and the University of Minnesota. Lentigen is primarily funded by Burrill Capital Fund IV, L.P. (San Francisco, CA). For further information, visit http://www.lentigen.com.

About Case Comprehensive Cancer Center Case Comprehensive Cancer Center is an NCI-designated Comprehensive Cancer Center located at Case Western Reserve University. The center, now in its 25th year of funding, integrates the cancer research activities of the largest biomedical research and health care institutions in Ohio Case Western Reserve, University Hospitals (UH) Case Medical Center and the Cleveland Clinic. NCI-designated cancer centers are characterized by scientific excellence and the capability to integrate a diversity of research approaches to focus on the problem of cancer. It is led by Stanton Gerson, MD, Asa and Patricia Shiverick- Jane Shiverick (Tripp) Professor of Hematological Oncology, director of the National Center for Regenerative Medicine, Case Western Reserve, and director of the Seidman Cancer Center at UH Case Medical Center.

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Lentigen Receives U.S. Orphan Drug Designation for Novel Gene Therapy for glioblastoma multiforme

Public release date: 11-Feb-2013 [ | E-mail | Share ]

Contact: Megan McCann memccann@nmh.org 312-926-5900 Northwestern Memorial Hospital

Each year, nearly 800,000 Americans suffer a stroke which can result in permanent brain damage, long term disability or death. As a leading cause of adult disability, stroke has an annual burden of more than $62 billion on the United States economy. With the exception of rehabilitation therapy, very few treatments are available to improve the chronic neurologic deficits caused by a stroke. In hopes of expanding therapeutic options, Northwestern Medicine researchers are investigating a novel stem cell therapy, known as SB623, that may hold the key to improving motor function following a disabling stroke.

Northwestern is currently one of only three sites in the nation enrolling participants in a landmark study to test the safety and efficacy of adult stem cell therapy for patients with stable ischemic stroke. Accounting for 87 percent of strokes, ischemic stroke occurs when a blocked artery interrupts the flow of oxygen and blood to the brain. This causes cell death and brain damage which can leave a person with impaired body functions, including paralysis, weakness on one side, difficulty with speech and language, vision issues, and cognitive challenges.

"Two million brain cells die each minute during a stroke making it critical to get treatment fast at the earliest sign of symptoms; once brain damage occurs, there's very little that can be done medically to reverse it," said principal investigator Joshua Rosenow, MD, director of Functional Neurosurgery at Northwestern Memorial Hospital and associate professor of neurosurgery, neurology, and physical medicine and rehabilitation at Northwestern University Feinberg School of Medicine. "While this study is only a preliminary step towards understanding the healing potential of these cells, we are excited about what a successful trial could do for a patient population that currently has very limited therapeutic options."

While the study's primary purpose is to examine the safety of SB623 stem cells, researchers will also seek to determine if the cells are effective in improving stroke symptoms. SB623 is derived from genetically engineered adult bone marrow cells from a healthy adult donor.

"Although not proven in humans, these stem cells have been shown to promote healing and improve function when administered in animal models of stable stroke," said co-investigator Richard Bernstein, MD, director of Northwestern Memorial's Stroke Center and associate professor of neurology at the Feinberg School. "The cells did not replace the neurons destroyed by stroke, but instead they appeared to encourage the brain to heal itself and promote the body's natural regenerative process. Eventually, the implanted stem cells disappeared."

"In this study, the cells are transplanted into the brain using brain mapping technology and scans, allowing us to precisely deposit the cells in the brain adjacent to the area damaged by stroke," explained Rosenow.

Early participants have received 2.5 million cells, but as the study progresses the dose will escalate to 5 million and eventually 10 million cells. Since SB623 cells are allogeneic, a single donor's cells can be used to treat many other individuals. Participants in the study will be followed for up to two years with periodic evaluations for safety and effectiveness in the improvement in motor function.

"Stroke can be a very disabling and life changing event," said Bernstein. "Even just a slight improvement in function could make a huge difference for a person impacted by stroke. To potentially give our patients the opportunity to permanently regain movement or speech is a very exciting prospect. In the animal models, the improvements appeared to remain even after the implanted stem cells disappeared."

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Northwestern Medicine researchers investigate stem cell therapy for stroke

Every gene in the nucleus of an animal or plant cell is packaged into a beads-on-a-string like structure called nucleosomes: the DNA of the gene forms the string and a complex of proteins called histones forms the beads around which the DNA is wrapped.

Scientists of the Max Planck Institute of Biochemistry in Martinsried near Munich, Germany, have now established that adding chemical tags on histones is critical for regulating gene activity during animal development. Studies over the past two decades revealed that many proteins that control the activity of genes are enzymes that add small chemical tags on histone proteins but also on a variety of other proteins. With their studies the researchers have now shown that it is the tags on the histones that control if genes are active or inactive. Their results were published in the journal Science.

Histone proteins can be modified by a number of different chemical tags at very specific sites. The researchers in the Research Group 'Chromatin Biology' of Jrg Mller focused on the histone tag that is added by an enzyme called Polycomb Repressive Complex 2 (PRC2). PRC2 is essential for a variety of different cell fate decisions in animals and plants. PRC2 functions to keep genes inactive in cells and at times where they should remain inactive.

Using the model organism Drosophila - the fruit fly - the scientists now generated animals with cells expressing an altered histone protein to which PRC2 can no longer add the tag. These cells cannot keep genes inactive anymore and many cell fate decisions go awry, exactly like in cells that lack the PRC2 enzyme. "This observation demonstrates that the business end is the tag on the histone and not on some other protein" says Ana Pengelly, the PhD student who conducted the experiments. Her colleague Omer Copur adds: "The approach we used permits us to now also investigate the function of other tags on histone proteins that have a different chemical nature." The insight gained from the work on PRC2 provides a strong impetus to figure how this tag alters the beads-on-a-string structure of genes and thereby controls gene activity.

More information: Pengelly, A.R., et al. A histone mutant reproduces the phenotype caused by loss of histone modifying factor Polycomb. Science, February 8, 2013. DOI: 10.1126/science.1231382

Journal reference: Science

Provided by Max Planck Society

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Histone modification controls development: Researchers demonstrate that chemical tags on histones regulate gene activity

Feb. 11, 2013 A study led by Dr Josep Tabernero, the Director of Clinical Research at the Vall d'Hebron Institute of Oncology (VHIO) and Head of the Medical Oncology Department at the Vall d'Hebron University Hospital, shows for the first time that ribonucleic acid interference (RNAi) is effective in the treatment of cancer patients. Harnessing these molecules to silence genes involved in the development and growth of cancer cells is an important step forward in developing a new and more targeted type of cancer therapy.

Dr Josep Tabernero, lead author of this study, said: "This is the first evidence to show that RNAi can be administered to cancer patients effectively, leading to significant tumour response."

RNAi is a gene-silencing mechanism that uses a subtype of RNA molecules to interfere with and silence genes. RNAi plays a vital role in normal cell development and differentiation, in cancer and viral defence, as it is powerful mechanism in the regulation of gene expression. Besides being a key natural cellular phenomenon, gene silencing shows great potential as a therapeutic device to shut down genes that have become hyperactive through cancer.

However, researchers have encountered difficulties in administering RNAi, as the molecules must penetrate cells in therapeutically effective concentrations, which in turn requires structural modifications. In the new study, led by the Vall d'Hebron Institute of Oncology (VHIO), along with several other cancer research centres and the U.S. biotech company Alnylam, scientists have developed a lipid nanoparticle approach that can deliver two of these molecules targeted against the genes encoding two key proteins involved in the development of cancer cells (VEGF and KSP). This system takes the form of a novel drug (ALN-VSP) made up of RNAi molecules and lipid nanoparticles (LNPs).

The new paper, published in the journal Cancer Discovery, presents the results of a Phase I clinical trial, involving 41 patients with advanced cancer that had metastasised to the liver. These patients were treated with the new drug twice a week with intravenous doses of between 0.01-1.5 mg/kg.

The trial found that not only was the drug safely administered, but also presented good evidence for clinical utility. In 11 of the 37 patients, the disease either did not progress or stabilized after six months of treatment. In some cases of patients with metastasis to the liver or abdominal lymph glands, there was a complete regression of metastasis. (It should be noted that the liver typically responds better to treatment due to its excellent capacity to absorb these molecules.)

The research team performed a pharmacodynamics analysis to determine the impact of the drug on the tumours by taking biopsy samples from the patients before and after the drug was administered. This revealed the presence of the RNAi constructs in the samples, thus showing that the structurally modified molecules reached the tumour and were effective.

Having previously tested the drug on animals, this first-in-human clinical test has demonstrated that an efficient formula has been developed to transport and deliver RNAi with clinically promising results. The results will have to be confirmed and extended in additional clinical studies.

The importance of clinical research

This study, which also involved other international research centres including the Dana-Farber Cancer Institute (Boston) and the Memorial Sloan-Kettering Cancer Center (New York), plus two other Spanish centres -- the Hospital Virgen del Roco in Seville and the INCLIVA Health Research Institute in Valencia -- is potentially transformative for new drug discovery that will allow targeted, population-based studies.

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Therapeutic effect of RNAi gene silencing effective in cancer treatment, study suggests

SUFFERN, N.Y.--(BUSINESS WIRE)--

Ryogen LLC, a genomic research and development company focusing on genes implicated in clinical diseases, had a very fruitful year in 2012, further developing its Intellectual Property portfolio. In 2012, Ryogen was awarded nine new patents, bringing the total number of issued U.S. Patents to 23 with 18 patent applications pending approval at the U.S. Patent Office.

Ryogens patents and patent applications are directed to genes that play important roles in various cancers, diabetes, obesity, hepatitis C, atherosclerosis, Alzheimer's and other diseases. They cover large segments of human chromosomes X, 1, 6, 7, 10, 11, 12, 17 and 19.

We are very pleased that the USPTO has awarded us these patents, said Dr. James W. Ryan, the named inventor on the patents and Chief Scientist at Ryogen. We can now license this technology to the research facilities that can turn them into tomorrows diagnostics, drugs and treatments.

Ryogen is becoming one of the major gene patent holders, said Valeria Poltorak, Ryogens Executive Vice President. Ryogens patents are available for licensing to the research and biotech community.

About Ryogen LLC

Ryogen LLC, headquartered in Suffern, N.Y., a genomics R&D company, owns an intellectual property (IP) portfolio of 23 gene patents and several patent applications directed to various genes with potentially important applications in biomedical research and, ultimately, diagnostics and drug development. Ryogen was formed with the purpose of making its intellectual property accessible to researchers via licensing of its patents. Ryogen is a business unit in the idea incubator operated by IP Holdings LLC, and Ryogen is managed by General Patent Corporation (GPC). For further information, visit http://www.ryogen.com.

The new additions to the Ryogen patent portfolio include:

The complete list of the issued patents with their descriptions is available at the Patents page of the Ryogen website.

About IP Holdings LLC

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Ryogen Receives Nine Human Gene Patents in 2012

Public release date: 11-Feb-2013 [ | E-mail | Share ]

Contact: University of Oxford press office press.office@admin.ox.ac.uk 44-018-652-80533 University of Oxford

Genes linked to autism and schizophrenia are only switched on during the early stages of brain development, according to a study in mice led by researchers at the University of Oxford.

This new study adds to the evidence that autism and schizophrenia are neurodevelopmental disorders, a term describing conditions that originate during early brain development.

The researchers studied gene expression in the brains of mice throughout their development, from 15-day old embryos to adults, and their results are published today in Proceedings of the National Academy of Sciences.

The study is a collaboration between researchers from the University of Oxford, King's College London and Imperial College London, and was funded by the Medical Research Council and the Wellcome Trust.

The research focused on cells in the 'subplate', a region of the brain where the first neurons (nerve cells) develop. Subplate neurons are essential to brain development, and provide the earliest connections within the brain.

'The subplate provides the scaffolding required for a brain to grow, so is important to consider when studying brain development,' says Professor Zoltn Molnr, senior author of the paper from the University of Oxford, 'Looking at the pyramids in Egypt today doesn't tell us how they were actually built. Studying adult brains is like looking at the pyramids today, but by studying the developing brains we are able to see the transient scaffolding that has been used to construct it.'

The study shows that certain genes linked to autism and schizophrenia are only active in the subplate during specific stages of development. 'The majority of the autism susceptibility genes are only expressed in the subplate of the developing mouse brain,' explains Dr Anna Hoerder-Suabedissen, who led the study at the University of Oxford, 'Many can only be found at certain stages of development, making them difficult to identify at later stages using previous techniques.'

The group were able to map gene activity in full detail thanks to powerful new methods which allowed them to dissect and profile gene expression from small numbers of cells. This also enabled them to identify the different populations of subplate neurons more accurately.

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Gene today, gone tomorrow: Genes for autism and schizophrenia only active in developing brains

London, February 11: Researchers have used molecular genetic engineering to optimize heart performance in models of diastolic heart failure by creating an optimized protein that can aid in high-speed relaxation similar to fast twitching muscles.

Researchers at the University of Minnesota's Department of Integrative Biology and Physiology and the Lillehei Heart Institute were behind the breakthrough.

Within heart cells, calcium plays a major role in orchestrating normal heart pump function. However, in diastolic failure the calcium signaling process is slowed; calcium levels rise to the peak needed for the squeezing action of the heart but don't then drop quickly enough for an efficient relaxation period - the condition known as diastolic heart failure.

University researchers were able to pinpoint a specific protein, parvalbumin - which aids in high-speed relaxation of fast twitching muscles in nature - and optimize it to become a calcium sponge for heart muscle. As a result, the optimized protein, ParvE101Q, soaks up excess calcium at a precise instant, allowing the heart to relax efficiently after contraction.

The advance offers a solid conceptual step forward in solving the puzzle of diastolic heart failure. The next step will be determining the best possible small molecule or gene delivery mechanism for the protein, which should allow the discovery to be used in clinics.

"In nature, there are unique organisms known to be able to contract and relax muscles quickly. We hoped research and discovery could help identify what was promoting this highly efficient activity so we could harness it for use in the heart. We've discovered that our optimized variation of parvalbumin can fulfill that role by treating diastolic heart failure," said Joseph M. Metzger, Ph.D., a University of Minnesota Medical School professor and chair of the Department of Integrative Biology and Physiology.

According to Metzger, who also serves as the Maurice B. Visscher Endowed Chair in Physiology, the sponge mechanism works as a temporary depot for calcium along its normal pathway. It increases productivity in the relaxation phase of the heart cycle without negatively impacting the contracting phase.

If they can develop an ideal delivery system for the optimized protein, the researchers believe they may have found a unique clinical application to treat diastolic heart failure.

The discovery is outlined in the latest issue of Nature Medicine.

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Protein turned into 'calcium sponge' to tackle diastolic heart failure

Feb. 11, 2013 Using cutting-edge time-lapse photography, Keck School of Medicine researchers have discovered clues to the development of the head at the cellular level, which could point scientists to a better understanding of how organs and birth defects form in humans.

A team of researchers at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC has for the first time determined the role of two important molecular signaling pathways that help control the number and position of repeated units of cells that pattern the head and face.

Two members of a "Wnt" signaling pathway are instrumental in forming pharyngeal pouches that organize the structure of the head and face. Problems with forming the pouches can result in birth defects, including the rare DiGeorge syndrome, which causes an array of symptoms including an abnormal facial appearance, cleft palate, congenital heart disease, and loss of the thyroid and thymus.

The research, "Wnt-Dependent Epithelial Transitions Drive Pharyngeal Pouch Formation," was published Feb. 11, 2013 in Developmental Cell. Principal author is Chong Pyo Choe, research associate at the Keck School of Medicine of USC.The research, conducted over a five-year period, was accomplished by photographing live zebrafish embryos every 10 minutes for 36-hour periods with a sophisticated microscope, enabling the researchers to see the pouches forming in real time, said Gage Crump, assistant professor in cell and neurobiology, and corresponding author on the research.

"Zebrafish and humans are similar at the genetic level and the organ level," Crump said. "They have almost all the same organs that we do, which makes the fish a very relevant system for understanding human health and disease."

The pharyngeal pouches develop the gills in fish, and in human fetuses they also form gill-like structures, which later organize the head skeleton and organs such as the thymus and thyroid. Birth defects like DiGeorge syndrome can be traced back to malformations in the development of the pharyngeal pouches, Crump said.

Choe developed more than 100 different transgenic lines, transferring key genes to live zebrafish embryos where they could be studied under the microscope at the single cell level. Surprisingly, the lengthy filming doesn't harm the embryos and they can grow up to be normal fish, Crump said.

The Wnt pathways are significant because they control two separate cell behaviors. Choe discovered this by finding a way to genetically block each pathway and then making time-lapse videos of how development went wrong in each case.

"In the future as we get better at harnessing stem cells to create organs, we hope to be able to bioengineer these cells to make a particularly shaped organ," Crump said. "What we're learning in zebrafish by studying these pouches will be generally applicable and we can pursue these basic principles to come up with new types of technology involving cellular therapy."

The team is now studying other signaling pathways and their possible contributions to organ development and defects.

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Possible genetic clues to organ development, birth defects

Newswise PISCATAWAY, NJ -- The social symbol for autism awareness, a ribbon of brightly-colored puzzle pieces, reflects the complexity of Autism Spectrum Disorder (ASD). A new five-year $2.125 million grant from the New Jersey Governors Council for Medical Research and Treatment of Autism will fund research of induced pluripotent stem cells that may be used to piece together the genetic pathways of autism and lead to new treatments for individuals affected by ASD. The research, led by James H. Millonig, PhD, associate professor of neuroscience and cell biology at the University of Medicine and Dentistry of New Jersey-Robert Wood Johnson Medical School, is being conducted as part of the Robert Wood Johnson Medical School New Jersey Autism Center of Excellence (NJ ACE).

Autism is defined by a spectrum of behavioral and neurological abnormalities, with distinct characteristics for each individual denoting that there are multiple underlying genetic causes, said Dr. Millonig, who also is assistant dean of medical science training at Robert Wood Johnson Medical School and a member of the Center for Advanced Biotechnology and Medicine, a joint institute of the medical school and Rutgers, The State University of New Jersey. Working with my colleagues in neuroscience and at the Child Health Institute of New Jersey and Rutgers, we hope to identify the neurobiological, molecular and genetic basis the biological signature of autism.

Autism is a disorder of the brain, which is mostly comprised of neurons. Induced pluripotent stem cells (iPSCs) are scientifically-derived stem cells from individuals that mimic the traits of embryonic stem cells and can be used to create other cell types. Therefore, the research team will use iPSCs to create human neurons in order to understand how they develop abnormally in individuals with autism. The scientists will then test FDA-approved drugs on the neurons to identify therapeutic treatments that may improve or reverse the disorder.

We know of a lot of genes associated with autism, but dont know when, where and how they act in development, explains Dr. Millonig. We need to look at neurons and determine how they mature differently in order to develop better drug therapies that are tailored to the needs of individuals with autism.

The multi-institutional NJ ACE team includes Emanuel DiCicco-Bloom, MD, professor of neuroscience and cell biology and pediatrics at the medical school; Linda M. Brzustowicz, MD, professor of genetics, Rutgers University; Chi-wei Lu, PhD, assistant professor of obstetrics, gynecology, and reproductive sciences and Zhiping Pang, PhD, assistant professor of neuroscience and cell biology both at the Child Health Institute of New Jersey; and Yong Lin, PhD, at The Cancer Institute of New Jersey. The Child Health Institute of New Jersey and The Cancer Institute of New Jersey are Centers of Excellence at Robert Wood Johnson Medical School. Consultants on the grant include the Rutgers University Cell and DNA Repository; Ronald Hart, PhD, professor of cell biology and neuroscience, Rutgers University; Bonnie Firestein, PhD, professor of cell biology and neuroscience, Rutgers University; Jennifer Moore, PhD, associate director, National Institute of Mental Health Stem Cell Center, and research assistant director, Stem Cell Research Center, Rutgers University.

About UMDNJ-ROBERT WOOD JOHNSON MEDICAL SCHOOL As one of the nations leading comprehensive medical schools, UMDNJ-Robert Wood Johnson Medical School is dedicated to the pursuit of excellence in education, research, health care delivery, and the promotion of community health. In cooperation with Robert Wood Johnson University Hospital, the medical schools principal affiliate, they comprise one of the nation's premier academic medical centers. In addition, Robert Wood Johnson Medical School has 34 other hospital affiliates and ambulatory care sites throughout the region.

As one of the eight schools of the University of Medicine and Dentistry of New Jersey, with 2,800 full-time and volunteer faculty, Robert Wood Johnson Medical School encompasses 22 basic science and clinical departments, and hosts centers and institutes including The Cancer Institute of New Jersey, the Child Health Institute of New Jersey, the Center for Advanced Biotechnology and Medicine, the Environmental and Occupational Health Sciences Institute, and the Stem Cell Institute of New Jersey. The medical school maintains educational programs at the undergraduate, graduate and postgraduate levels for more than 1,500 students on its campuses in New Brunswick, Piscataway, and Camden, and provides continuing education courses for health care professionals and community education programs. To learn more about UMDNJ-Robert Wood Johnson Medical School, log on to rwjms.umdnj.edu. Find us online at http://www.Facebook.com/RWJMS and http://www.twitter.com/UMDNJ_RWJMS. ###

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Stem Cell Research Focusing on Autism's Genetic Mysteries Earns $2.125 Million Grant at Robert Wood Johnson Medical ...

Public release date: 11-Feb-2013 [ | E-mail | Share ]

Contact: Vicki Bendure vicki@bendurepr.com 202-374-9259 Society for Maternal-Fetal Medicine

In a study to be presented on February 14 between 1:15 p.m., and 3:30 p.m. PST, at the Society for Maternal-Fetal Medicine's annual meeting, The Pregnancy Meeting , in San Francisco, researchers will report that variation in a gene involved in inflammation is associated with developmental problems after preterm birth in females, but not males.

This randomized study, Sex-specific genetic susceptibility to adverse neurodevelopmental outcome after early preterm birth, may improve understanding of how developmental problems occur after preterm birth and may help identify prevention strategies.

"Preterm birth is the leading cause of childhood brain injury," said the study's author, Erin Clark, MD. "Compared to preterm girls, preterm boys are more likely to die, and survivors are more likely to have long-term problems, including disability and cerebral palsy. We don't understand why preterm boys are at a disadvantage compared to girls."

Through her study, Clark, assistant professor of Maternal-Fetal Medicine at the University of Utah School of Medicine, determined whether genetic variants influence the risk of developmental problems after a preterm birth, and whether there is a difference in risk factors between males and females.

Clark evaluated patients previously enrolled in a randomized trial of magnesium sulfate before preterm birth for prevention of cerebral palsy. She evaluated children that died before their first birthday, or developed cerebral palsy or other developmental problems by age 2 years, and compared them to healthy children.

The research shows a variant in the inflammation gene, interleukin 6, was associated with developmental problems in females but not in males. Treatment with magnesium sulfate before birth didn't appear to change this risk.

"These results add to the evidence that inflammation genes play a role in risk of brain injury in preterm children. In addition, they suggest that genetic risk factors for brain injury after early delivery may be different in boys and girls," said Clark. "However, the effect of genes and gender on outcomes after preterm birth remains poorly understood. Additional research is necessary in order to better understand the differences in outcomes between males and females born preterm."

###

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Study suggests genetic predisposition to brain injury after preterm birth is sex-specific

Public release date: 12-Feb-2013 [ | E-mail | Share ]

Contact: Catherine Afarian cat@23andme.com 408-656-8872 PeerJ

A 23andMe study of consumers' reactions to genetic testing found that even when the tests revealed high-risk mutations in individuals, those individuals had few negative reactions to the news. Instead of inducing serious anxiety, the test results prompted people to take positive steps, including follow-up visits with a doctor and discussions with family members who could also be at risk.

The study, titled "Dealing with the unexpected: Consumer responses to direct-access BRCA mutation testing" published today as part of the launch of PeerJ, a new peer reviewed open access journal in which all articles are freely available to everyone.

"The paper addresses one of the most urgent questions in the field of genetics and genomics, namely the impact of receiving unexpected information about high genetic risk for a life-threatening disease," said the lead author of the paper and 23andMe's Senior Medical Director Dr. Uta Francke.

The study looked at how people reacted when they learned for the first time that they carried a mutation in either the BRCA1 or BRCA2 gene that put them at higher risk for breast and ovarian cancer. The study included interviews conducted with 32 individuals found to be mutation carriers and 31 individuals found to be non-carriers. Five to ten percent of breast cancers occur in women with a genetic predisposition for the disease, usually due to mutations in either the BRCA1 or BRCA2 gene. The mutations at the center of this study are responsible for a substantial number of hereditary breast and ovarian cancers among women with Ashkenazi Jewish ancestry.

Those who acquired the potentially life-saving information not only took appropriate actions on their own behalf, but also notified relatives who might share that risk. In what the study described as a "cascade effect," a number of relatives who were subsequently tested discovered they too had one of the mutations.

The findings are important given that a frequent concern regarding direct-to-consumer testing is based on the assumption that it causes either serious emotional distress or triggers deleterious actions on the part of consumers. Individuals who learned they had the mutation said they did not suffer serious emotional distress, and did not take inappropriate actions. All but one of the 32 mutation-positive participants appreciated learning their BRCA mutation status. None of the 31 mutation-negative individuals misinterpreted their result to think they are free from all risks and safe to abandon routine cancer screening.

The study provides important preliminary data that suggest some of the bioethical concerns may be overstated, at least for the self-selected pool of individuals who seek DTC personal genomics information. The authors suggested that broader screening of Ashkenazi Jewish women for these three BRCA mutations should be considered."

###

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Consumers have few negative reactions to the results of genetic testing for cancer mutations

Feb. 11, 2013 In a study to be presented on February 14 at the Society for Maternal-Fetal Medicine's annual meeting, The Pregnancy Meeting , in San Francisco, researchers will report that variation in a gene involved in inflammation is associated with developmental problems after preterm birth in females, but not males.

This randomized study, Sex-specific genetic susceptibility to adverse neurodevelopmental outcome after early preterm birth, may improve understanding of how developmental problems occur after preterm birth and may help identify prevention strategies.

"Preterm birth is the leading cause of childhood brain injury," said the study's author, Erin Clark, MD. "Compared to preterm girls, preterm boys are more likely to die, and survivors are more likely to have long-term problems, including disability and cerebral palsy. We don't understand why preterm boys are at a disadvantage compared to girls."

Through her study, Clark, assistant professor of Maternal-Fetal Medicine at the University of Utah School of Medicine, determined whether genetic variants influence the risk of developmental problems after a preterm birth, and whether there is a difference in risk factors between males and females.

Clark evaluated patients previously enrolled in a randomized trial of magnesium sulfate before preterm birth for prevention of cerebral palsy. She evaluated children that died before their first birthday, or developed cerebral palsy or other developmental problems by age 2 years, and compared them to healthy children.

The research shows a variant in the inflammation gene, interleukin 6, was associated with developmental problems in females but not in males. Treatment with magnesium sulfate before birth didn't appear to change this risk.

"These results add to the evidence that inflammation genes play a role in risk of brain injury in preterm children. In addition, they suggest that genetic risk factors for brain injury after early delivery may be different in boys and girls," said Clark. "However, the effect of genes and gender on outcomes after preterm birth remains poorly understood. Additional research is necessary in order to better understand the differences in outcomes between males and females born preterm."

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Genetic predisposition to brain injury after preterm birth is sex-specific

Agent Orange(tga genetics) Mystery strains
Cannabis grow

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Making Ripples in the World
Every winter our students bring their energy, insight, and ambition to the world for seven weeks during Field Work Term. Last year students volunteered more than 25000 hours of time collectively helping to find shelter for homeless women and children; teaching ordinary citizens how to decode their own genetics; making green building models; going door-to-door for the EPA; and lobbying for healthcare and livable wages. Some have even created their own non-profit organizations. Every gift to Bennington is a gift to the world. Make your gift to The Bennington Fund today.

By: BenningtonCollege

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Making Ripples in the World - Video