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A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency.
Wang L, Yang Y, Breton C, Bell P, Li M, Zhang J, Che Y, Saveliev A, He Z, White J, Latshaw C, Xu C, McMenamin D, Yu H, Morizono H, Batshaw ML, Wilson JM. Wang L, et al. Among authors: latshaw c. Sci Adv. 2020 Feb 12;6(7):eaax5701. doi: 10.1126/sciadv.aax5701. eCollection 2020 Feb. Sci Adv. 2020. PMID: 32095520 Free PMC article.