Applications of CRISPR/Cas9 in retinal degenerative diseases

作者：Peng, Ying-Qian; Tang, Luo-Sheng; Yoshida, Shigeo; Zhou, Ye-Di^*

来源：International Journal of Ophthalmology, 2017, 10(4): 646-651.

DOI：10.18240/ijo.2017.04.23

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

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.

• 出版日期2017-4-18
• 单位中南大学