Application of CRISPR/Cas9 genome editing technology in hereditary retinal diseases
10.3760/cma.j.cn115989-20201120-00787
- VernacularTitle:CRISPR/Cas9基因编辑技术在遗传性视网膜疾病中的应用
- Author:
Xihao SUN
1
;
Shibo TANG
;
Jiansu CHEN
Author Information
1. 中南大学爱尔眼科学院 爱尔眼科研究所，长沙 410015
- Keywords:
Gene editing;
CRISPR/Cas9;
Inherited retinal diseases
- From:
Chinese Journal of Experimental Ophthalmology
2023;41(9):925-930
- CountryChina
- Language:Chinese
-
Abstract:
Several mutant genes for inherited retinal diseases have been identified, but effective treatments are still lacking.The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) system can edit human genomic DNA by nonhomologous end joining or homology-directed repair, offering more possibilities for the treatment of hereditary retinal diseases.CRISPR/Cas9 not only can genetically correct patient-derived induced pluripotent stem cells (iPSCs) to observe their differentiation into retinal cells thereby, thereby exploring the pathogenesis of the disease and implementing cell therapy, but can also be delivered to the body via vectors and directly act on target cells to achieve in vivo gene editing.CRISPR/Cas9 gene editing technology in hereditary retinal diseases has been mainly used in retinitis pigmentosa, hereditary X-linked juvenile retinoschisis, and Leber congenital amaurosis 10, of which the in vitro application of CRISPR/Cas9 for Leber congenital amaurosis 10 has entered the clinical trial stage.In this paper, we reviewed the mechanism and key advances of CRISPR/Cas9 and provided an overview of gene editing in IRDs.
