Category Archives: Gene Therapy

ELK GROVE VILLAGE, Ill., Nov. 5, 2014 /PRNewswire-USNewswire/ -- A manuscript from the laboratory of Dr. Brian Kaspar of Nationwide Childrens Hospital was recently published in the journal Molecular Therapy. The paper, Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA-a dose response study in mice and nonhuman primates is the first publication resulting from a groundbreaking collaboration between Cure SMA, the National Institute of Neurological Disorders and Stroke (NINDS), and Dr. Kaspar. Beginning in 2010, Cure SMA made a series of grants to Dr. Kaspar to study gene therapy, also called gene transfer. Spinal muscular atrophy (SMA) is caused by a mutation in the survival motor neuron 1 gene (SMN1). Because of this mutation, the individual does not produce enough survival motor neuron (SMN) protein. Gene transfer may increase SMN levels by using a virus, called a vector, to deliver the SMN1 gene to affected cells. Kaspars laboratory discovered that Adeno-associated virus serotype 9 (AAV9) had the unique ability to cross the blood brain barrier and the Blood-Cerebrospinal Fluid Barrier (CSF). Dr. Kaspar and his team have studied two approaches for SMA: an injection into a vein, a process known as systemic delivery which is currently in … Continue reading →

By Cyndi Root RetroSense Therapeutics announced in a press release that the Food and Drug Administration (FDA) has granted Orphan Drug status to RST-001. The treatment for retinitis pigmentosa (RP) combines gene therapy and optogenetics. RetroSense developed the proprietary technology from research conducted at Wayne State University and Massachusetts General Hospital. Sean Ainsworth, RetroSense CEO, said, We are hopeful that the benefits associated with Orphan Drug status will better enable us to advance RST-001 through development and ultimately into the marketplace where it may benefit many who are suffering from blindness due to retinitis pigmentosa. Optogenetics Retinitis pigmentosa causes the degeneration and loss of rod and cone photoreceptors in the retina, causing severe vision loss and blindness. Currently there are no FDA-approved drugs to treat RP. RetroSenses work in optogenetics involves making the retina more light sensitive, thereby improving vision. The company expects RST-100 to have broad applications and to be useful in heredity or acquired RP. RST-001 uses a photosensitivity gene, channelrhodopsin-2, and creates new photosensors in the retinal cells. Channelrhodopsin-2 has been shown in numerous animal studies to restore light perception and vision, and in primate studies, the agent was well tolerated. RetroSense is using optogenetics and channelrhodopsin-2 … Continue reading →

MITTEILUNG UEBERMITTELT VON BUSINESS WIRE. FUER DEN INHALT IST ALLEIN DAS BERICHTENDE UNTERNEHMEN VERANTWORTLICH. Distinguished Scientist To Lead Companys Gene Therapy Research Efforts CAMBRIDGE, Mass. --(BUSINESS WIRE)-- 23.09.2014 -- Biogen Idec (NASDAQ:BIIB) today announced that Olivier Danos, Ph.D., has joined the company as senior vice president, Gene Therapy. Dr. Danos reports directly to Douglas Williams, Ph.D., executive vice president, Research and Development, and is a member of Biogen Idecs Senior Research and Development Leadership Team. Dr. Danos will lead the companys gene therapy research group, a team dedicated to identifying and developing new technologies for gene transfer and genome engineering. As part of his role, Dr. Danos will also identify potential collaborations that complement the companys internal capabilities and support the development of treatments across Biogen Idecs therapeutic focus areas. Olivier is a true pioneer in the field. We believe that his expertise will help accelerate our development of a world-class gene therapy platform that combines our growing internal capabilities with collaborations with leading research organizations across the globe, said Dr. Williams. Our commitment to this important area will complement our existing capabilities in antisense, small molecule and biologics research, and enhance our ability to effectively and efficiently develop new … Continue reading →

Stem cells and gene hold promising treatment options for Parkinson's, mandate doctors across the globe, including from Mumbai. Eleven trials to test stem cell and gene therapy for treating Parkinson's are underway currently of which the one in Mumbai had to be put on hold due to regulatory hurdles. Currently, neuro-augmentative therapies such as usage of drugs or deep brain stimulation (DBS) are being used to treat Parkinson's disorder. "The future holds hope for neuro-restorative therapies like that of stem cells or gene infusion in the Parkinson's disorder treatment. It involves restoration of brain function to normal. In the next five to seven years, this may pave the way for future," said Dr Paresh Doshi, neurologist at Jaslok Hospital, Peddar Road in Mumbai. Regulatory hurdles and resource constraints though have led to these trials being held up in Mumbai. Dr Doshi said that trials of Duodopa therapy which involves infusion of an active ingredient gel called Levodopa in the intestines has been kept on hold at the moment at privately-run Jaslok Hospital due to regulatory hurdles. The hospital was the only centre in entire South East Asia to have been running the trial. "Levodopa gets converted into dopamine in the … Continue reading →

In Vivo Gene Therapy for Cystic Fibrosis Part 4 In this video we discuss the use of In Vivo gene therapy to treat Cystic Fibrosis. By: Ben Garside … Continue reading →

In Vivo Gene Therapy for Cystic Fibrosis Part 2 In this video we discuss the use of In Vivo gene therapy to treat Cystic Fibrosis. By: Ben Garside … Continue reading →

Course Preview - Principles and Practices of Gene Therapy By: stanfordonline … Continue reading →

Cell Gene Therapy Opening ceremony 3rd International Conference and Exhibition on Cell Gene Therapy, October 27-29, 2014 at Las Vegas, USA. Cell Therapy conference is an outstanding event co... By: omicsgroup incorporation … Continue reading →

CAN GENE THERAPY CURE HIV? with PAULA CANNON Paula Cannon from the University of Southern California and the defeatHIV Collaboratory tells us why everyone should more seriously consider the gene therapy approach to fuctionally curing... By: defeatHIV … Continue reading →

Stem Cells FDA Oversight Celia Witten, M.D., Ph.D., Director, Office of Cellular, Tissue, Gene Therapy Center for Biologics Evaluation Research, Food Drug Administration (FDA) For more information on the 2014... By: Alliance for Regenerative Medicine … Continue reading →