The Potential of Gene Therapy as A Ground Breaking Approach to Treating Inherited Retinal Diseases and Other Genetic Eye Conditions: Review

Abstract

Background: Inherited retinal degenerations (IRDs) represent a diverse group of genetic disorders that often lead to significant visual impairment and were historically considered untreatable. Recent advancements in molecular biology have spurred the development of gene therapies, notably the FDA-approved voretigene neparvovec-rzyl, which targets RPE65-associated Leber congenital amaurosis (LCA).

Methods: This review examines various therapeutic strategies currently in clinical trials for IRDs, including gene augmentation, gene editing, optogenetics, neuroprotection, and stem cell therapies. The efficacy of these methods is assessed through a comprehensive analysis of ongoing interventional clinical trials and preclinical investigations.

Results: Over 60 active clinical trials are exploring gene therapies for different IRDs, with promising results indicating potential improvements in visual function. Notably, gene augmentation therapies using adeno-associated viruses (AAVs) have shown efficacy in restoring vision in patients with specific genetic mutations. Emerging techniques such as RNA interference and CRISPR/Cas9 gene editing are also being evaluated for their ability to address a wider range of genetic mutations associated with retinal diseases.

Conclusion: The field of gene therapy in ophthalmology is rapidly evolving, offering new hope for patients with inherited retinal diseases. Ongoing research and clinical trials are critical for establishing the safety and long-term efficacy of these innovative therapies. The future of ophthalmologic treatments is likely to see a shift towards personalized medicine that targets the underlying genetic causes of visual impairment.