Gene therapy for retinal diseases: From genetics to treatment.

ABSTRACT: The gene therapy approach for retinal disorders has been considered largely over the last decade owing to the favorable outcomes of the US Food and Drug Administration-approved commercial gene therapy, Luxturna. Technological advances in recent years, such as next-generation sequencing, research in molecular pathogenesis of retinal disorders, and precise correlations with their clinical phenotypes, have contributed to the progress of gene therapies for various diseases worldwide, and more recently in India as well. Thus, considerable research is being conducted for the right choice of vectors, transgene engineering, and accessible and cost-effective large-scale vector production. Many retinal disease-specific clinical trials are presently being conducted, thereby necessitating the collatio

SUBMITTER: Khaparde A 

PROVIDER: S-EPMC11451791 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature