Tay-Sachs AAV gene therapy
MPC staff are busy with the start of a toxicology study for Tay-Sachs disease. Tay-Sachs is a genetic disease that severely affects the entire nervous system and often causes death with only a few years in children. The Tay-Sachs Gene Therapy Consortium is a collaboration between 4 international institutions (Auburn University, Boston College, Cambridge University (U.K.), Massachusetts General Hospital/Harvard University). The investigators aim to start human clinical trials within 3 years following the outstanding successes of AAV treatment in several small and large animals models of Tay-Sachs disease. We are pleased to be partnering with the UF Powell Gene Therapy Center Toxicology Core (Dr. Thomas Conlon) for the preclinical testing phase of the AAV gene therapy for the clinical trials. Dr. William Castleman, UF College of Veterinary Medicine, will serve as the pathologist on this study along with Lynda Schneider, lead histotechnologist, and Caroline King, cGLP study coordinator, from the Molecular Pathology Core. Please see Cure Tay-Sachs Foundation for more information.