The Application and Progression of CRISPR/Cas9 Technology in Ophthalmological Diseases

Overview

Journal Eye (Lond)

Specialty Ophthalmology

Date 2022 Aug 1

PMID 35915232

Authors

Xumeng Hu

Beibei Zhang

Xiaoli Li

Miao Li

Yange Wang

Handong Dan

Jiamu Zhou

Yuanmeng Wei

Keke Ge

Pan Li

Zongming Song

Affiliations

Soon will be listed here.

Abstract

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.

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