Gene Therapy for Inherited Retinal Disease: Focus on LCA1
This course provides a clinically relevant introduction to in vivo gene therapy with an emphasis on ocular applications. It begins with a review of the pathophysiology, clinical presentation, and optometric management of Leber Congenital Amaurosis. Then the course explores the foundational concepts of gene therapy viral vectors, with a deep dive into the therapeutic use of Adeno-Associated Viruses (AAV) as delivery vectors. Participants will review ocular diseases currently under investigation in gene therapy trials before examining a phase 1/2 study of AAV-mediated treatment for Leber Congenital Amaurosis Type 1 (LCA1), including the proposed mechanism of disease correction and current clinical trial outcomes. Designed for clinicians who want to understand emerging therapies from a clinical perspective, this course bridges complex science with practical relevance for patient care.