Gene therapy for retinal diseases: global progress and challenges
10.3760/cma.j.cn115989-20200716-00500
- VernacularTitle:视网膜疾病基因疗法：全球研究进展和挑战
- Author:
Zhijian WU
1
Author Information
1. PTC Therapeutics, New Jersey, 07080, USA
- From:
Chinese Journal of Experimental Ophthalmology
2020;38(8):633-638
- CountryChina
- Language:Chinese
-
Abstract:
In the past three decades, we have witnessed the great promise of gene therapy in treating a variety of inherited and acquired retinal diseases. The large number of preclinical studies in retinal gene therapy has led to over sixty clinical trials at different phases and the approval of Luxturna, a gene therapy product that treats type 2 Leber congenital amaurosis (LCA2) caused by mutations in the RPE65 gene. While current gene delivery systems have led to some success in clinical studies, safer and more efficient gene delivery calls for improved vectors and alternative intraocular administration routes. Progress has been made in large gene delivery and less invasive vector administration with the advancement of novel adeno-associated viral (AAV) vector technologies. Clustered regularly interspaced short palindromic repeats (CRISPR)-based technology has enlarged the toolbox of gene therapy and will inevitably find wide applications in the treatment of retinal diseases. Although gene therapy faces a number of challenges, in the coming years, it will become a viable treatment option for a number of currently incurable retinal disorders.
