Youtor
Gene Therapy for Refsum Disease
>> YOUR LINK HERE: ___ http://youtube.com/watch?v=bQaTZI6UU6w
Ryan Butler, PhD from UT Southwestern will provide an overview of Gene Therapy as a potential future therapy for Refsum Diseases. • Adult Refsum disease is a metabolic disorder caused by a number of faulty genes (principally PHYH) which affect the pathway that degrades a plant-derived chemical called phytol which some bacteria found in ruminant animals turn into Phytanic Acid. In humans if phytanic acid is not degraded it accumulates in nerves, liver and fat. Phytanic acid is not produced in the body but found in many foods. The highest concentration of phytanic acid is found in dairy products, beef, lamb and some fish. • Presenter from UT Southwestern: • Dr. Ryan Butler received his B.Sc. from James Madison University, his Ph.D. from the National University of Ireland, Galway and most recently was a postdoctoral fellow at the University of North Carolina at Chapel Hill. Dr. Butler has been an assistant professor in the departments of Psychiatry and Pediatrics at UT Southwestern Medical Center for two years. Prior to his faculty appointment, Dr. Butler investigated the underpinnings of psychiatric and physiological conditions such as alcohol abuse, anxiety and chronic pain with the goal to find therapeutic targets for human clinical treatment. Since opening his own independent lab at UTSW, he has pursued the development of gene therapies for the treatment of Angelman Syndrome, Peroxisome Biogenesis Disorders and other neurologic disorders. He is also a part of the broader gene therapy leadership community at UTSW who collaborate in every phase of gene therapy development from the proof-of-concept to Phase III clinical trials. • Learn more about Adult Refsum Disease at Global DARE Foundation's website: https://www.defeatadultrefsumeverywhe... If you have any questions please contact Global DARE Foundation at [email protected]
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