Advancements in Gene Therapy for Inherited Retinal Diseases: Current Approaches and Future Directions

Abstract

Inherited retinal diseases (IRDs) encompass a group of genetically determined disorders that

lead to progressive vision loss and blindness. Gene therapy has emerged as a promising

treatment for IRDs, offering the potential for long-term visual improvement by targeting the

underlying genetic causes of these conditions. This paper reviews the advancements in gene

therapy for IRDs, focusing on current therapeutic approaches, challenges, and future

directions. The use of viral vectors, CRISPR/Cas9-based gene editing, and retinal cell

transplantation has shown great promise in preclinical and clinical studies. While some gene

therapies have already been approved, the field faces several hurdles, including delivery

efficiency, immune responses, and long-term safety. As the understanding of retinal diseases

and gene therapy technologies continues to evolve, new strategies hold the potential to

revolutionize the treatment of these debilitating disorders.