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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: breton 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.
Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases.
Greig JA, Breton C, Ashley SN, Martins KM, Gorsuch C, Chorazeczewski JK, Furmanak T, Smith MK, Zhu Y, Bell P, Shoop W, Li H, Smith J, Tomberlin G, Clark P, Mitchell TW, Buza EL, Yan H, Jantz D, Wilson JM. Greig JA, et al. Among authors: breton c. Hum Gene Ther. 2022 Nov;33(21-22):1174-1186. doi: 10.1089/hum.2022.061. Hum Gene Ther. 2022. PMID: 36375122 Free PMC article.
541 results