Application of CRISPR/Cas9 gene editing technology in the construction of animal models of ophthalmic diseases / 中华实验眼科杂志

ABSTRACT

The clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated endonuclease 9 (Cas9) technology is a gene editing technology that uses RNA to guide endonucleases.This technology is rapidly used in gene editing and disease gene therapy in multiple species because of its easy operation, precise targeting, short cycle, and high gene knockout efficiency.At present, the corneal dystrophy model ( UBIAD1, TGF- β R124C gene mutations), glaucoma model ( MYOC Y435H, OPTN E50K and PMEL gene mutations), cataract model ( GJA8, KPNA4, C- MAF, AQP5 and PIKFYVE gene mutations), Leber congenital amaurosis animal model ( KCNJ13 and LCA5 gene mutations), retinblastoma animal model ( RB1/ RBL gene mutations) and retinitis pigmentosa models ( HKDC1, C8ORF37, CERKL, PRCD, ASRGL1, LRAT and PDE6B gene mutations) have been constructed by using this technology.The role of MFRP, CPAMD8, Pax6, and FREM genes in animal eye development has been further confirmed via this technology.The application of CRISPR/Cas9 gene editing technology in the construction of animal models of ophthalmic diseases was reviewed in this article.