The application and progression of CRISPR/Cas9 technology in ophthalmological diseases

被引：20

作者：

Hu, Xumeng ^{[1
]}

Zhang, Beibei ^{[1
]}

Li, Xiaoli ^{[1
]}

Li, Miao ^{[1
]}

Wang, Yange ^{[1
]}

Dan, Handong ^{[1
]}

Zhou, Jiamu ^{[1
]}

Wei, Yuanmeng ^{[1
]}

Ge, Keke ^{[1
]}

Li, Pan ^{[1
]}

Song, Zongming ^{[1
]}

机构：

[1] Zhengzhou Univ, Henan Prov Peoples Hosp, Peoples Hosp, Henan Eye Hosp,Henan Eye Inst, Zhengzhou 450003, Henan, Peoples R China

来源：

EYE | 2023年 / 37卷 / 04期

关键词：

LEBER CONGENITAL AMAUROSIS; RETINAL GENE-THERAPY; MACULAR DEGENERATION; RETINITIS-PIGMENTOSA; MUTATIONS; REPEATS; RNA; DNA; IDENTIFICATION; DYSTROPHY;

D O I：

10.1038/s41433-022-02169-1

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated nuclease (Cas) system is an adaptive immune defence system that has gradually evolved in bacteria and archaea to combat invading viruses and exogenous DNA. Advances in technology have enabled researchers to enhance their understanding of the immune process in vivo and its potential for use in genome editing. Thus far, applications of CRISPR/Cas9 genome editing technology in ophthalmology have included gene therapy for corneal dystrophy, glaucoma, congenital cataract, Leber's congenital amaurosis, retinitis pigmentosa, Usher syndrome, fundus neovascular disease, proliferative vitreoretinopathy, retinoblastoma and other eye diseases. Additionally, the combination of CRISPR/Cas9 genome editing technology with adeno-associated virus vector and inducible pluripotent stem cells provides further therapeutic avenues for the treatment of eye diseases. Nonetheless, many challenges remain in the development of clinically feasible retinal genome editing therapy. This review discusses the development, as well as mechanism of CRISPR/Cas9 and its applications and challenges in gene therapy for eye diseases.

引用

页码：607 / 617

页数：11

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