Abstract
The treatment of hereditary retinal disease is still one of the contemporary scientific problems. Leber congenital amaurosis （LCA） is one type of congenital retinal diseases. Desirable results have been achieved in ongoing clinical trials of gene therapy for LCA, and the efficacy and safety in the intraocular injection of a gene inserted in an adeno-associated virus （AAV） have been verified abroad. These results bring hope and opportunity to LCA patients. China has more hereditary retinal disease patients, but gene therapy for hereditary retinal disease and LCA is lacking. Rightly interpreting and objectively evaluating the clinical trials of gene therapy of LCA will provide us with many important references and useful clues to further help us organize and implement clinical trials of gene therapy for hereditary retinal disease in the future. Key words: Leber congenital amaurosis; Hereditary retinal disease; Gene therapy; Clinical trial
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.
