Applications of CRISPR/Cas9 genome editing technology in gene therapy for hereditary eye diseases

Authors:  Wu Shijing,  Sui Ruifang

DOI: 10.3760/cma.j.issn.2095-0160.2018.11.016
Published 2018-11-10
Cite as Chin J Exp Ophthalmol, 2018,36(11): 892-896.

Abstract

Clustered regulatory interspaced short palindromic repeat (CRISPR)/CRISPR associated nuclease (Cas) system is an adaptive immune system that confers resistance to exogenous virus or plasmid in bacteria and archaea, over the 30 years since its discovery, researchers have a better understanding of its immune processes in vivo and the mechanisms of gene editing by using its function.Researches found that CRISPR/Cas9 system modified from typeⅡ CRISPR/Cas may edit genome accurately and effectively.In recent years, with the progress and development of gene sequencing technology, it is more explicit to make genetic diagnosis of a variety of hereditary eye diseases, and with the improvement of specificity for Cas9 in eukaryotic cells, gene editing is showing a great potential in the field of treating hereditary eye diseases.At present, the application of CRISPR/Cas9 gene editing technology has extended to the gene therapy of some hereditary eye diseases, such as congenital cataract, congenital glaucoma, retinitis pigmentosa (RP), congenital corneal dystrophy, Leber congenital amaurosis (LCA) and Usher syndrome.Besides, the combination of CRISPR/Cas9 gene editing technology with adeno-associated virus vectors (AAV) and induced pluripotent stem cells (iPSCs) research offers more possibilities and new approaches for the treatment of hereditary diseases.This article reviewed the mechanism of CRISPR/Cas9 and its applications in gene therapy for hereditary eye diseases.

Key words:

Clustered regularly interspaced short palindromic repeats; CRISPR-Cas systems; Gene therapy; Hereditary eye diseases

Contributor Information

Wu Shijing
Department of Ophthalmology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing 100730, China
Sui Ruifang
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Updated: September 4, 2019 — 10:50 am