Applications of CRISPR/Cas9 in retinal degenerative diseases

Abstract

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.

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

Figure 1. Structure and applications of CRISPR/Cas9 in retinal degenerative diseases

BH: Bridge helix; PI: PAM interacting. The structure of Cas9 protein of this figure is adapted from Cavanagh&Garrity, “CRISPR Mechanism”, CRISPR/Cas9, Tufts University, 2014. Available at https://sites.tufts.edu/crispr/; accessed on Dec. 23, 2016.