国际眼科杂志2025，Vol.25Issue(6)：912-917,6.DOI:10.3980/j.issn.1672-5123.2025.6.07
CRISPR/Cas9基因编辑技术在基因遗传性眼病中的应用进展
Research progress of CRISPR/Cas9 in genetically inherited eye diseases
摘要
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.关键词
成簇规律间隔短回文重复序列/CRISPR相关蛋白9(CRISPR/Cas9)/基因编辑/基因治疗/遗传性眼病Key words
clustered regularly interspaced short palindromic repeats/CRISPR associated proteins(CRISPR/Cas9)/gene editing/gene therapy/inherited eye diseases引用本文复制引用
刘张愉,秦勋,黄嘉钰,蒋沁..CRISPR/Cas9基因编辑技术在基因遗传性眼病中的应用进展[J].国际眼科杂志,2025,25(6):912-917,6.基金项目
国家自然科学基金项目(No.82471111) National Natural Science Foundation of China(No.82471111) （No.82471111）
