2018 FAST Science Summit - PTC Therapeutics - Gene Therapy for Angelman Syndrome
Edwin Weeber, PhD. and Jodi A. Cook, PhD discuss utilization of the mouse and rat models for Angelman syndrome has allowed us to obtain preclinical data necessary to move AAV-mediated gene replacement therapy toward a human clinical application. Insights into the route of administration,, gene distribution, phenotypic recovery, and potential biomarkers will be discussed.
