Research progress of CRISPR/Cas9 in genetically inherited eye diseases
10.3980/j.issn.1672-5123.2025.6.07
- VernacularTitle:CRISPR/Cas9基因编辑技术在基因遗传性眼病中的应用进展
- Author:
Zhangyu LIU
1
;
Xun QIN
1
;
Jiayu HUANG
1
;
Qin JIANG
1
Author Information
1. The Affiliated Eye Hospital of Nanjing Medical University, Nanjing 210029, Jiangsu Province, China
- Publication Type:Journal Article
- Keywords:
clustered regularly interspaced short palindromic repeats/CRISPR associated proteins(CRISPR/Cas9);
gene editing;
gene therapy;
inherited eye diseases
- From:
International Eye Science
2025;25(6):912-917
- CountryChina
- Language:Chinese
-
Abstract:
Currently, researchers have identified several mutated genes associated with hereditary eye diseases; however, effective therapeutic options remain scarce. The emergence of clustered regularly interspaced short palindromic repeats(CRISPR)and its associated proteins(CRISPR-associated proteins, Cas)offers a promising approach for treating these diseases. CRISPR/Cas9 enables precise targeting and modification of specific genetic sequences, allowing for the correction of mutated genes, as well as knockout or replacement of pathogenic genes to achieve therapeutic effects. In ophthalmology, CRISPR/Cas9 has been applied to various hereditary eye disorders, including corneal dystrophy, congenital cataracts, glaucoma, and retinitis pigmentosa. Additionally, significant progress has been made to utilize CRISPR/Cas9 to develop disease models. Therefore, it has great potential for clinical applications. However, challenges such as delivery efficiency and off-target effects remain. This review summarizes the mechanism of CRISPR/Cas9, its applications in genetic eye diseases and disease models, as well as the existing challenges, aiming to provide new insights for treatment.
