Advancements in Gene Therapy for Blindness

This chapter explores the pivotal transition from laboratory research to clinical trials in gene therapy targeting inherited retinal diseases. Focused on a groundbreaking trial for Leber Congenital Amaurosis type 1, it discusses engineering viruses for gene delivery, patient impact, and the challenges of gaining FDA approval. Personal patient stories illustrate the life-changing potential of these therapies, while the chapter also highlights evolving strategies and innovative technologies in the realm of gene therapy.