Applications of CRISPR/Cas9 in retinal degenerative diseases

被引：14

作者：

Peng, Ying-Qian ^{[1
]}

Tang, Luo-Sheng ^{[1
]}

Yoshida, Shigeo ^{[2
]}

Zhou, Ye-Di ^{[1
]}

机构：

[1] Cent S Univ, Dept Ophthalmol, Xiangya Hosp 2, Changsha 410011, Hunan, Peoples R China

[2] Kyushu Univ, Dept Ophthalmol, Grad Sch Med Sci, Fukuoka 8128582, Japan

来源：

INTERNATIONAL JOURNAL OF OPHTHALMOLOGY | 2017年 / 10卷 / 04期

关键词：

CRISPR/Cas9; gene therapy; genome editing; retinal degeneration; retinitis pigmentosa; leber congenital amaurosis; GENE-THERAPY; RETINITIS-PIGMENTOSA; ANIMAL-MODELS; KNOCKOUT PIGS; BREAK REPAIR; CAS9; MECHANISMS; GENERATION; MUTATIONS; REVEALS;

D O I：

10.18240/ijo.2017.04.23

中图分类号：

R77 [眼科学];

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 (iPSCs). 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

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