Applications of CRISPR/Cas9 in retinal degenerative diseases

被引：0

作者：

Ying-Qian Peng ^{[1
]}

Luo-Sheng Tang ^{[1
]}

Shigeo Yoshida ^{[2
]}

Ye-Di Zhou ^{[1
]}

机构：

[1] Department of Ophthalmology,the Second Xiangya Hospital,Central South University

[2] Department of Ophthalmology,Kyushu University Graduate School of Medical Sciences

来源：

International Journal of Ophthalmology | 2017年 / 04期

关键词：

CRISPR/Cas9; gene therapy; genome editing; retinal degeneration; retinitis pigmentosa; leber congenital amaurosis;

D O I：

暂无

中图分类号：

R774.1 [视网膜疾病];

学科分类号：

100212 ;

摘要：

Gene therapy is a potentially effective treatment for retinal degenerative diseases.Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9(Cas9) system has been developed as a new genome-editing tool in ophthalmic studies.Recent advances in researches showed that CRISPR/Cas9 has been applied in generating animal models as well as gene therapy in vivo of retinitis pigmentosa(RP) and leber congenital amaurosis(LCA).It has also been shown as a potential attempt for clinic by combining with other technologies such as adeno-associated virus(AAV) and induced pluripotent stem cells(i PSCs).In this review,we highlight the main points of further prospect of using CRISPR/Cas9 in targeting retinal degeneration.We also emphasize the potential applications of this technique in treating retinal degenerative diseases.

引用

页码：646 / 651

页数：6

共 23 条

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