Abstract
Monogenic and multifactorial retinal diseases are genetically and clinically diverse conditions that have historically shared an untreatable clinical course. In recent years, the advent of targeted treatments like gene therapy has enabled intraocular delivery of viral vectors, such as adeno-associated viruses and retroviruses, that establish long-term stable protein expression. Despite the innate distinctions between mainly monogenic disorders affecting the retina such as Leber congenital amaurosis (LCA), heterogeneous monogenic conditions like retinitis pigmentosa (RP), and multifactorial diseases such as age-related macular degeneration (AMD), gene therapy has shown great promise as a gene replacement or augmentation strategy to treat or halt these disorders. This review will present an overview of retinal gene therapy and provide a comprehensive analysis of past and current clinical trials for LCA, RP, and AMD. It will discuss the paradigm shift that has occurred in ophthalmic care thanks to the development of gene therapy, as well as its challenges for the future.
Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
