Advances in Precision Therapeutics and Gene Therapy Applications for Retinal Diseases: Impact and Future Directions.

Moustafa S Magliyah, Abeer Aljahdali, Khaled K Abu-Amero, Mariam M AlEissa, Abrar A Alhawsawi, Raghad Alonazi, Enas Magharbil, Hani B AlBalawi, Naif M Alali, Syed Hameed

Journal: Genes 2025;16(7):

PMID: 40725503

Free Full Text on MDPI View on PubMed

Abstract

Gene therapy has emerged as a promising treatment for several eye diseases since it may restore vision and stop blindness. Many eye diseases, including retinitis pigmentosa and macular degeneration, have historically been rather difficult to treat and usually cause permanent vision loss. However, thanks to advances in gene therapy, many disorders can now be effectively targeted and genetically changed, providing a safer, more direct, maybe even curative approach. By introducing, altering, or repairing specific genes inside the eye, gene therapy seeks to fix the defective genes causing these disorders, thereby improving general eye health and visual ability. Voretigene neparvovec is one FDA- and EMA-approved treatment for mutations. Retinitis pigmentosa, age-related macular degeneration, X-linked retinoschisis, choroideremia, and Stargardt disease are among the several eye disorders still under clinical trials, and experimental treatment is in progress. As research on gene therapy develops, it opens the path for groundbreaking treatments that could fundamentally change the ophthalmic care scene.

Address: Research Department, King Khaled Eye Specialist Hospital, Riyadh 11462, Saudi Arabia.; College of Medicine, Alfaisal University, Riyadh 11533, Saudi Arabia.; Public Health Laboratory, Public Health Authority, Riyadh 12382, Saudi Arabia.; The Computational Sciences Department at the Centre for Genomic Medicine (CGM), King Faisal Specialist Hospital & Research Centre, Riyadh 11211, Saudi Arabia.; Division of Ophthalmology, Department of Surgery, College of Medicine, University of Jeddah, Jeddah 23218, Saudi Arabia.; College of Medicine, Alfaisal University, Riyadh 11533, Saudi Arabia.; Retina and Uveitis Department, Jeddah Eye Hospital, Jeddah 23454, Saudi Arabia.; Ophthalmology Department, King Abdulaziz University, Jeddah 21589, Saudi Arabia.; Division of Ophthalmology, Department of Surgery, Faculty of Medicine, University of Tabuk, Tabuk 47311, Saudi Arabia.; Research Department, King Khaled Eye Specialist Hospital, Riyadh 11462, Saudi Arabia.; Research Department, King Khaled Eye Specialist Hospital, Riyadh 11462, Saudi Arabia.; Vitreoretinal Division, King Khaled Eye Specialist Hospital, Riyadh 11462, Saudi Arabia.

DOI: 10.3390/genes16070847

Link outs

Free resources

Full Text Sources:

Medical:

Research Materials:

https://antibodies.cancer.gov/detail/CPTC-MUC1-1

MeSH Terms: Humans, Genetic Therapy, Retinal Diseases, Precision Medicine, Macular Degeneration, cis-trans-Isomerases