Applications of CRISPR/Cas9 in retinal degenerative diseases

International Journal of Ophthalmology
Ying-Qian PengYe-Di Zhou

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.

Citations

Nov 30, 2018·Genes·David J JiangStephen H Tsang
Feb 19, 2019·The Tohoku Journal of Experimental Medicine·Yuanjun Li, Yedi Zhou
Aug 21, 2018·Stem Cells International·Nina XieBeisha Tang
Jun 6, 2018·Frontiers in Cell and Developmental Biology·Wenhan Yu, Zhijian Wu

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