CRISPR(clustered regularly interspaced short palindromic repeats)基因编辑技术通过精准改变细胞DNA序列，控制细胞命运及表型，是有望从根本上改变疾病治疗的新技术。由于眼球独特的生理构造，基因编辑疗法在治疗眼科疾病方面的应用具有明显的优势。目前，CRISPR基因编辑疗法治疗10型Leber先天性黑矇(Leber congenital amaurosis 10，LCA10)的临床试验已经展开，治疗其他多种眼科疾病的临床试验也即将开始。随着新一代CRISPR基因编辑技术的发展，基因编辑疗法有望为眼科疾病的治疗提供新的手段。

Clustered regularly interspaced short palindromic repeats (CRISPR) genome editing is a newly developed technology to precisely modify cellular DNA sequence, which could control cell fate and phenotype and fundamentally reform disease treatment. The structure of the eye offers unique advantages as a genome editing target. Recently, a CRISPR genome editing therapy has begun to be tested in Leber congenital amaurosis 10 (LCA10) patients, and the clinical trials for more ocular diseases are about to start. The development of CRISPR/Cas genome editing tools will drive major advances in the application of gene therapies in the treatment of ophthalmic disease.