CRISPR-Cas9-mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis.
Jo DH, Song DW, Cho CS, Kim UG, Lee KJ, Lee K, Park SW, Kim D, Kim JH, Kim JS, Kim S, Kim JH, Lee JM.
Jo DH, et al. Among authors: kim ug, kim d, kim s, kim js, kim jh.
Sci Adv. 2019 Oct 30;5(10):eaax1210. doi: 10.1126/sciadv.aax1210. eCollection 2019 Oct.
Sci Adv. 2019.
PMID: 31692906
Free PMC article.