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Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors.
Moscioni D, Morizono H, McCarter RJ, Stern A, Cabrera-Luque J, Hoang A, Sanmiguel J, Wu D, Bell P, Gao GP, Raper SE, Wilson JM, Batshaw ML. Moscioni D, et al. Among authors: batshaw ml. Mol Ther. 2006 Jul;14(1):25-33. doi: 10.1016/j.ymthe.2006.03.009. Epub 2006 May 3. Mol Ther. 2006. PMID: 16677864 Free article.
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: batshaw ml. Sci Adv. 2020 Feb 12;6(7):eaax5701. doi: 10.1126/sciadv.aax5701. eCollection 2020 Feb. Sci Adv. 2020. PMID: 32095520 Free PMC article.
125 results