Advances and Challenges in Gene Therapy for Inherited Retinal Dystrophies: A Comprehensive Review

Abstract

Inherited retinal dystrophies (IRDs) are a diverse group of genetic disorders leading to progressive vision loss due to the degeneration of retinal photoreceptors. Gene therapy has emerged as a promising approach to address the underlying genetic causes of IRDs, offering the potential for restoring vision and halting disease progression. This review provides a comprehensive overview of gene therapy innovations for IRDs, focusing on the mechanisms, recent advancements, and ongoing challenges. We discuss the fundamental principles of gene therapy, including the use of viral and non-viral vectors, and highlight key developments such as the approval of Luxturna for RPE65-mediated retinal dystrophy and the application of gene editing technologies like CRISPR/Cas9. Despite these advancements, significant challenges remain, including vector delivery, long-term safety, and variable patient responses. This review also explores the future directions of gene therapy, emphasizing the need for further research to address these challenges and enhance therapeutic efficacy. By examining the current state of gene therapy for IRDs, this review aims to provide valuable insights into the potential for these treatments to transform the management of retinal diseases and improve the quality of life for affected individuals.