Sign in to view content
Gene Therapy - A New Hope
- Published 2018
Dr. Mark Pennesi discusses the latest developments in the field through a seminal paper on adenoviral-RPE65 gene therapy for Leber Congenital Amaurosis that led to the landmark approval by the FDA of gene therapy for this inherited retinal degeneration. Dr. Pennesi will also discusses how gene therapy may be applied to more complex retinal diseases that are not due to monogenetic mutations through a clinical trial for gene therapy targeting the VEGF pathway in age-related macular degeneration.
Category: Hereditary Retinal Disease & Genetics