The progress of the gene editing therapy of inherited retinal diseases based on CRISPR/Cas9
10.3760/cma.j.cn511434-20220908-00493
- VernacularTitle:基于CRISPR/Cas9的基因编辑技术在遗传性视网膜疾病治疗中的研究进展
- Author:
Kaiqin SHE
1
;
Qin CHEN
;
Fang LU
Author Information
1. 四川大学华西医院眼科, 成都 610041
- Keywords:
Inherited retinal diseases;
CRISPR/Cas9;
Base editing;
Prime editing;
Review
- From:
Chinese Journal of Ocular Fundus Diseases
2023;39(7):605-610
- CountryChina
- Language:Chinese
-
Abstract:
Inherited retinal diseases (IRDs) are the major cause of refractory blinding eye diseases, and gene replacement therapy has already made preliminary progress in the treatment of IRDs. For IRDs that cannot be treated by gene replacement therapy, gene editing provides an alternative therapeutic method. Strategies like disruption of pathogenic variants with or without gene augmentation therapy and precise repair of pathogenic variants can be applied for IRDs with various inheritance patterns and pathogenic variants. In animal models of retinitis pigmentosa, Usher syndrome, Leber congenital amaurosis, cone rod cell dystrophy, and other disorders, CRISPR/Cas9, base editing, and prime editing showed the potential to edit pathogenic variations in vivo, indicating a promising future for gene editing therapy of IRDs.
