Ophthalmology and Vision Science

Ocular Genetics – Gene & Cell Therapies

Ocular genetics is transforming the future of vision care through targeted gene therapies, precision diagnostics, and regenerative strategies. Next-generation sequencing has significantly advanced the identification of mutations responsible for inherited retinal diseases, congenital anomalies, and early-onset ocular disorders. Gene replacement therapies, antisense oligonucleotide treatments, and CRISPR-based genome editing are offering unprecedented opportunities to halt or reverse vision loss caused by congenital defects. Stem-cell–derived photoreceptors, retinal pigment epithelium transplants, and induced pluripotent cell technologies are expanding the potential for regenerative repair. Molecular insights continue to reveal disease pathways, guiding personalized therapeutic development and patient-specific prognostic assessments. Ethical considerations, regulatory frameworks, and long-term safety evaluations remain integral to advancing these therapies responsibly. Collectively, ocular genetics continues to reshape clinical possibilities by bridging molecular science with practical interventions that provide hope for individuals affected by challenging hereditary conditions.