Category Archives: Gene Therapy

SCID gene therapy class By: sw tue … Continue reading →

Contact Information Available for logged-in reporters only 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 … Continue reading →

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 … Continue reading →

Retrovirus in Gene Therapy A video created for gene therapy (SQG 4143-01). Lecturer: Dr. Salehhuddin Hamdan Students: (1) Tang Jiaa Earn (AQ100082) (2) Phang Shi YI (AQ100073) By: Phang n Tang … Continue reading →

Gene therapy for haemophilia - Professor Amit Nathwani A Medicine for Members talk given on 22 April 2014 at the Royal Free Hospital by Professor Amit Nathwani. By: Royal Free London NHS Foundation Trust … Continue reading →

Cochlear Implant Gets an Upgrade with Gene Therapy Cochlear Implant Gets an Upgrade with Gene Therapy This is a video of 3-D confocal imaging showing the cells secreting the neurotrophin (cyan) and the regene... By: Stefano Di Criscio … Continue reading →

Biotechnology - GMO's Gene Therapy Video notes on genetically modified organisms gene therapy. By: vancebiology … Continue reading →

Gene Therapy To Enhance Hearing - ABC Austrlia News - 2014 - http://www.abc.net.au/am/content/2014/s3991064.htm Paper http://stm.sciencemag.org/content/6/233/233ra54. By: sixesfullofnines … Continue reading →

By Cathy Yarbrough, Contributing Editor Gene therapy pioneer Katherine High, M.D., was looking forward to her first meeting in 2011 with Jeffrey Marrazzo, then a consultant to the CEO of Childrens Hospital of Philadelphia (CHOP). A veteran of three life sciences companies, Marrazzo was meeting with Dr. High and other CHOP leaders to identify potential new revenue streams for the hospital. Dr. High, an international leader in gene therapy research and clinical application, had considered postponing the meeting because she was so busy with her work as director of the hospitals Center for Cellular and Molecular Therapeutics (CCMT). However, she did not reschedule because she wanted to ask Marrazzo for a favor: Could he speak with the VCs who were calling her and inquiring about investing in CCMTs work on RPE65? I hadnt spoken to them yet, because at the time I was busier than usual with my patient care, research, and teaching responsibilities. In addition, VCs are not a constituency that I normally deal with, said Dr. High, professor of pediatrics at the University of Pennsylvania as well as a Howard Hughes medical investigator. Scheduled to last just 60 minutes, Dr. Highs first meeting with Marrazzo stretched to seven … Continue reading →

Ethics of Gene therapy By: Jessi Santos … Continue reading →