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Category Archives: Gene Therapy

Employment of gene therapy in depression (psychiatric disorder) – Video

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Employment of gene therapy in depression (psychiatric disorder)
A study in 2010 discovered the gene responsible for causing symptoms of major depression. The discovery of this gene makes it possible to employ gene therapy...

By: EMPLOYMENT OF GENE THERAPY IN PSYCHIATRIC DISORDER

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Employment of gene therapy in depression (psychiatric disorder) - Video

Gene and Cell Therapy for Thrombosis, AIDS and Inherited Disorders

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Washington, DC (PRWEB) May 05, 2014

There will be a Media Event in Washington, DC, on Wednesday, May 21, 2014, from 10:45 am 12:15 pm in Wilson A of the Marriott Wardman Park Hotel, which is sponsored by the American Society of Gene & Cell Therapy (ASGCT).

The event will profile exciting clinical trial results in patients suffering from serious and often fatal diseases such as human immunodeficiency virus (HIV), Inherited Immunodeficiencies, Hemophilia B, and complications from Thrombosis. All registered media will have the opportunity for personal, one-on-one questions with academic and industry leaders in the field, including:

Dr. Bruce L. Levine of the University of Pennsylvania is developing a gene and cell therapy strategy to achieve a functional cure for HIV infection by genome editing of cells, and results from the first-in-human trial of this novel treatment strategy will be presented.

Professor Adrian Thrasher of the University College of Londons Institute of Child Health will discuss the impressive results of recent advances in gene therapy technology to treat inherited immunodeficiencies in patients, many of whom are children.

Researchers at St. Jude Children's Research Hospital have successfully treated Hemophilia patients with gene therapy that led to their disease-free living for several years. Dr. Andrew Davidoff will provide the latest update on these exciting clinical results.

Thrombosis, or the formation of blood clots inside a blood vessel, remains the major cause of death and disability in the western world. Dr. Bruce Sullenger of Duke University Medical Center will describe the recent development of gene and cell therapy strategies to control thrombosis that is currently in a Phase 3 clinical trial.

The media event will take place in Wilson A in the Marriott Wardman Park Hotel.

Members of the media are welcome to conduct individual interviews with each speaker following the presentation, and will receive complimentary full-access registration to the ASGCT 17th Annual Meeting. Representatives who wish to attend may contact ASGCT directly at 414.278.1341 or mdean(at)asgct(dot)org.

The American Society of Gene & Cell Therapy (ASGCT) is a professional nonprofit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website, http://www.asgct.org.

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Gene and Cell Therapy for Thrombosis, AIDS and Inherited Disorders

Bioinformatics approach helps researchers find new use for old drug

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Developing and testing a new anti-cancer drug can cost billions of dollars and take many years of research. Finding an effective anti-cancer medication from the pool of drugs already approved for the treatment of other medical conditions could cut a considerable amount of time and money from the process.

Now, using a novel bioinformatics approach, a team led by investigators at Beth Israel Deaconess Medical Center (BIDMC) has found that the approved antimicrobial drug pentamidine may help in the treatment of patients with advanced kidney cancer. Described online in the journal Molecular Cancer Therapeutics, the discovery reveals how linking cancer gene expression patterns with drug activity might help advance cancer care.

"The strategy of repurposing drugs that are currently being used for other indications is of significant interest to the medical community as well as the pharmaceutical and biotech industries," says senior author Towia Libermann, PhD, Director of the Genomics, Proteomics, Bioinformatics and Systems Biology Center at BIDMC and Associate Professor of Medicine at Harvard Medical School. "Our results demonstrate that bioinformatics approaches involving the analysis and matching of cancer and drug gene signatures can indeed help us identify new candidate cancer therapeutics."

Renal cell cancer consists of multiple subtypes that are likely caused by different genetic mutations. Over the years, Libermann has been working to identify new disease markers and therapeutic targets through gene expression signatures of renal cell cancer that distinguish these different cancer subtypes from each other, as well as from healthy individuals. In this new paper, he and his colleagues were looking for drugs that might be effective against clear cell renal cancer, the most common and highly malignant subtype of kidney cancer. Although patients with early stage disease can often be successfully treated through surgery, up to 30 percent of patients with renal cell cancer present with advanced stages of disease at the time of their diagnosis.

To pursue this search, they made use of the Connectivity Map (C-MAP) database (http://www.broadinstitute.org/cmap), a collection of gene expression data from human cancer cells treated with hundreds of small molecule drugs.

"C-MAP uses pattern-matching algorithms to enable investigators to make connections between drugs, genes and diseases through common, but inverse, changes in gene expression," says Libermann. "It provided us with an exciting opportunity to use our renal cell cancer gene signatures and a new bioinformatics strategy to match kidney cancer gene expression profiles from individual patients with gene expression changes inducted by various commonly used drugs."

After identifying drugs that may reverse the gene expression changes associated with renal cell cancer, the investigators used assays to measure the effect of the selected drugs on cells. This led to the identification of a small number of FDA-approved drugs that induced cell death in multiple kidney cancer cell lines. The investigators then tested three of these drugs in an animal model of renal cell cancer and demonstrated that the antimicrobial agent pentamidine (primarily used for the treatment of pneumonia) reduced tumor growth and enhanced survival. Gene expression experiments using microarrays also identified the genes in renal cell cancer that were counteracted by pentamidine.

"One of the main challenges in treating cancer is the identification of the right drug for the right individual," explains first author Luiz Fernando Zerbini, PhD, of the International Center for Genetic Engineering and Biotechnology in Cape Town, South Africa, adding that this bioinformatics approach could be a particularly valuable lower-cost model in developing countries.

The authors say their next step will be to evaluate the potential of pentamidine in combination with the current standard-of-care therapies to treat kidney cancer. "Since the drugs we are evaluating are already FDA-approved, successful studies in preclinical animal models may enable us to rapidly move these drugs into clinical trials," adds Libermann.

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Bioinformatics approach helps researchers find new use for old drug

Gene Therapy Used to Preserve Sight in Patients

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Newswise Orlando, Fla. In two separate studies, vision scientists have developed healthy genes to prevent blinding diseases that stem from genetic defects. The research is being presented at the 2014 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) this week in Orlando, Fla.

In a clinical trial to treat choroideremia, a rare disease that causes progressive and irreversible blindness, scientists developed a virus that can replace the missing gene (that causes the disease) in the cells at the back of the eye. Six months after the virus was injected into patients, findings showed that some patients experienced improved vision.

Abstract Title: Improved visual function in patients with choroideremia undergoing subretinal gene therapy Presentation Start/End Time: Sunday, May 4, 3:15 3:30pm Location: S 320AB Session Number: 147

In a separate study, researchers developed a gene therapy to stop the progression of a form of retinitis pigmentosa, an inherited disease transmitted from mothers to sons. Two years after the therapy was used to treat dogs at an early stage of the disease, the treatment remained effective. Further use of the technique in dogs with mid and late stages of the disease also resulted in a positive response to the intervention.

Abstract Title: RPGR gene augmentation delivered at early, mid and late stage disease in a canine model of XLRP rescues photoreceptor structure and function Presentation Start/End Time: Tuesday, May 6, 11am 12:45pm Location: Exhibit/Poster Hall SA Session Number: 342 # # #

The Association for Research in Vision and Ophthalmology (ARVO) is the largest eye and vision research organization in the world. Members include some 11,500 eye and vision researchers from over 70 countries. ARVO encourages and assists research, training, publication and knowledge-sharing in vision and ophthalmology.

All abstracts accepted for presentation at the ARVO Annual Meeting represent previously unpublished data and conclusions. This research may be proprietary or may have been submitted for journal publication.

Embargo policy: Journalists must seek approval from the presenter(s) before reporting data from paper or poster presentations. Press releases or stories on information presented at the ARVO Annual Meeting may not be released or published until the conclusion of the presentation.

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Gene Therapy Used to Preserve Sight in Patients

Regeneron, Avalanche To Develop Novel Gene Therapy Products In Ophthalmology

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By RTT News, May 05, 2014, 08:34:00 AM EDT

(RTTNews.com) - Regeneron Pharmaceuticals, Inc. ( REGN ) and Avalanche Biotechnologies, Inc. formed a broad collaboration to discover, develop and commercialize novel gene therapy products to treat ophthalmologic diseases. The collaboration includes novel gene therapy vectors and proprietary molecules, discovered jointly by Avalanche and Regeneron, and developed using the Avalanche Ocular BioFactory, an adeno-associated virus (AAV)-based, proprietary, next-generation platform for the discovery and development of gene therapy vectors for ophthalmology.

As part of the agreement, Avalanche would receive an upfront cash payment, contingent payments of up to $640 million upon achievement of certain development and regulatory milestones, plus a royalty on worldwide net sales of collaboration products. The collaboration comprises up to eight distinct therapeutic targets, and Regeneron will have exclusive worldwide rights for each product it moves forward in clinical development. Further, Avalanche has the option to share in development costs and profits for products directed toward two collaboration therapeutic targets selected by Avalanche.

Pursuant to the agreement, Regeneron has a time-limited right of first negotiation for certain rights to AVA-101, Avalanche's gene therapy product targeting vascular endothelial growth factor currently under development for the treatment of wet age-related macular degeneration, upon completion of the ongoing Phase 2a study.

For comments and feedback: contact editorial@rttnews.com

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Regeneron, Avalanche To Develop Novel Gene Therapy Products In Ophthalmology

Gene discovery links cancer cell 'recycling' system to potential new therapy

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1-May-2014

Contact: Leslie Orr Leslie_Orr@urmc.rochester.edu University of Rochester Medical Center

University of Rochester scientists have discovered a gene with a critical link to pancreatic cancer, and further investigation in mice shows that by blocking the gene's most important function, researchers can slow the disease and extend survival.

Published online by Cell Reports, the finding offers a potential new route to intrude on a cancer that usually strikes quickly, has been stubbornly resistant to targeted therapies, and has a low survival rate. Most recent improvements in the treatment of pancreatic cancer, in fact, are the result of using different combinations of older chemotherapy drugs. The research led by Hartmut "Hucky" Land, Ph.D., and Aram F. Hezel, M.D., of UR Medicine's James P. Wilmot Cancer Center, identifies a new target in the process of garbage recycling that occurs within the cancer cell called autophagy, which is critical to pancreatic cancer progression and growth.

Autophagy is derived from the Greek roots "auto" (self) and "phagein" (to eat), and is an intracellular digestive process that allows cells to survive under stress. During a cell's transformation from normal to malignant, autophagy speeds up to keep pace with rapid cellular changes and a tumor's quest to grow. The newly discovered PLAC-8 gene sustains the highly active recycling process, as it removes faulty proteins and organelles and degrades them into reusable building blocks during cancer progression.

"What makes this an exciting opportunity is that the gene we're studying is critical to the cancer cell's machinery but it is not essential to the function of normal cells," said Land, chair of Biomedical Genetics at the University of Rochester School of Medicine and Dentistry and director of research at Wilmot. "By targeting these types of non-mutated genes that are highly specific to cancer, we are looking for more effective ways to intervene."

The Cell Reports study underlines Wilmot's overall unique approach to cancer research. Rather than investigate single faulty genes linked to single subtypes of cancer, Rochester scientists have identified a larger network of approximately 100 non-mutated genes that cooperate and control the shared activities of many cancers. While investigating this larger gene network, Land and Hezel focused on PLAC-8.

Moreover, the team found that by inactivating PLAC-8 in mice and shutting down autophagy, they could significantly slow cancer's progression. The relevance of PLAC-8 may also extend to other tumors lung, colon, and liver, for example -- that share key genetic changes such as KRAS and p53 mutations that are present in the majority of pancreatic cancers. The breadth of these findings is an area of ongoing study in the Land and Hezel labs.

"PLAC-8 and its job within the cancer cell of accelerating recycling suggests new points of attack and what we all hope will be opportunities to identify and develop new treatments," said Hezel, vice chief of Wilmot's Division of Hematology and Oncology and a UR associate professor. "Our data showing PLAC 8's role in autophagy has great potential because while there are other drugs being evaluated to inhibit autophagy, not all of them target proteins specifically important to this process in tumors."

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Gene discovery links cancer cell 'recycling' system to potential new therapy


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