Gene Replacement Therapy for Rare Diseases: From Bench to Bedside

Gene replacement therapy is coming to the pediatric neurology clinic. Experts will describe how this technology has gone from the bench to the bedside. Questions about viral vectors and their safety will be addressed, as well as practical considerations for bringing gene therapy to your clinic. Dr. Jerry Mendell, Dr. Samiah Al-Zaidy and Nationwide Hospital's Director of Good Manufacturing Practice operations, Jaysson Eicholtz, will dispel the myths and answer your questions about this exciting new therapy.