Applications of CRISPR/Cas9 in retinal degenerative diseases

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.