Melissa Spencer, PhD, Chair of the C3 Scientific Advisory Board and Professor of Neurology at UCLA, recently presented during a webinar hosted by the Muscular Dystrophy Association (MDA). The presentation, aimed at educating the public, gave an excellent background about genes, mutations, and adeno-associated vector (AAV)-based gene therapies. She highlights some of the latest advances in gene therapy technologies and provides an update on her program developing an AAV-based gene therapy for LGMD2A/R1. This project received funding from C3, the MDA, and the National Institutes of Health.
Read more about Dr. Spencer’s C3-funded gene therapy program here and here.
Dr. Melissa Spencer provides update on development of an LGMD2A/R1 gene therapy