Steven Grey at the ATP1A3 symposium in Chicago скачать в хорошем качестве

Steven Grey at the ATP1A3 symposium in Chicago

Dr Steven Gray talks about AAV gene therapy that can potentially help AHC patients. Dr. Steven Gray is one of the world leaders in developing gene therapies for rare and ultra-rare diseases. He works at the University of Texas Southwestern, where he is currently an Associate Professor in the Department of Pediatrics at the University of Texas Southwestern Medical Center. Dr. Gray is the director of the UTSW Viral Vector Facility and maintains affiliations with the Department of Molecular Biology, the Department of Neurology and Neurotherapeutics, the Eugene McDermott Center for Human Growth and Development, and the Hamon Center for Regenerative Science and Medicine at UT Southwestern. He also maintains an adjunct faculty appointment in the Department of Pediatrics and the Carolina Institute for Developmental Disorders at the University of North Carolina at Chapel Hill. Dr. Gray’s core expertise is in AAV gene therapy vector engineering and optimizing approaches to deliver a gene to the nervous system. His major focus is in AAV vector development to develop vectors tailored to serve specific clinical and research applications involving the nervous system. “What exactly is gene therapy?” you ask. In AHC and thousands of other rare diseases, a single gene mutation causes a protein to either malfunction or not be produced. This can cause death or destruction of certain cells that play key roles in vital functions. Gene therapy involves pairing up healthy copies of the malfunctioning gene with a harmless virus, referred to as a “viral vector”. Trillions of copies of the vector are made and then given to a person through a one-time injection. The healthy, normal-functioning genes are incorporated into cells, which begin to produce the missing or malfunctioning protein. Researchers believe that gene therapy for AHC has the potential to not only halt disease progression, but also reverse some of the symptoms and damage that has already occurred. Gene therapy is currently being tested in over 600 clinical trials.