Send to

Choose Destination
Int J Ophthalmol. 2017 Apr 18;10(4):646-651. doi: 10.18240/ijo.2017.04.23. eCollection 2017.

Applications of CRISPR/Cas9 in retinal degenerative diseases.

Author information

Department of Ophthalmology, the Second Xiangya Hospital, Central South University, Changsha 410011, Hunan Province, China.
Department of Ophthalmology, Kyushu University Graduate School of Medical Sciences, Fukuoka 812-8582, Japan.


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.


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

Supplemental Content

Full text links

Icon for PubMed Central
Loading ...
Support Center