CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis

Leber congenital amaurosis (LCA), one of the leading causes of childhood-onset blindness, is caused by autosomal recessive mutations in several genes including RPE65. In this study, we performed CRISPR-Cas9–mediated therapeutic correction of a disease-associated nonsense mutation in Rpe65 in rd12 mi...

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Detalhes bibliográficos
Publicado no:Sci Adv
Main Authors: Jo, Dong Hyun, Song, Dong Woo, Cho, Chang Sik, Kim, Un Gi, Lee, Kyu Jun, Lee, Kihwang, Park, Sung Wook, Kim, Daesik, Kim, Jin Hyoung, Kim, Jin-Soo, Kim, Seokjoong, Kim, Jeong Hun, Lee, Jung Min
Formato: Artigo
Idioma:Inglês
Publicado em: American Association for the Advancement of Science 2019
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6821465/
https://ncbi.nlm.nih.gov/pubmed/31692906
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aax1210
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