Ophthalmology and Vision Science

Retinal Gene Therapy and Personalized Medicine

Gene therapy represents a transformative approach in the management of inherited retinal disorders, including retinitis pigmentosa and Leber congenital amaurosis. Using viral vectors or novel delivery systems, targeted genetic correction aims to restore photoreceptor function and halt disease progression. Challenges include safe delivery, immune response management, and long-term efficacy. Personalized medicine strategies, guided by genomic profiling, allow tailored interventions for individual patients. Advanced imaging modalities and electrophysiological tests are crucial for monitoring therapeutic response. Artificial intelligence supports patient selection, treatment optimization, and outcome prediction. Regulatory frameworks ensure stringent evaluation of gene therapy products for safety, quality, and efficacy. Nanotechnology is being investigated to enhance vector delivery and precision targeting within retinal tissues. Emerging trends emphasize combining gene therapy with stem cell transplantation, optogenetics, and pharmacological adjuncts to maximize visual restoration. By integrating genetic science, clinical expertise, and global regulatory standards, retinal gene therapy is redefining personalized care, offering hope for patients with previously untreatable hereditary retinal diseases, and driving innovation in vision science.