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In vivo Treatment of a Severe Vascular Disease via a Bespoke CRISPR-Cas9 Base Editor.
Alves CRR, Das S, Krishnan V, Ha LL, Fox LR, Stutzman HE, Shamber CE, Kalailingam P, McCarthy S, Lino Cardenas CL, Fong CE, Imai T, Mitra S, Yun S, Wood RK, Benning FMC, Lawton J, Kim N, Silverstein RA, da Silva JF, de la Cruz D, Richa R, Malhotra R, Chung DY, Chao LH, Tsai SQ, Maguire CA, Lindsay ME, Kleinstiver BP, Musolino PL. Alves CRR, et al. Among authors: richa r. bioRxiv [Preprint]. 2024 Nov 11:2024.11.11.621817. doi: 10.1101/2024.11.11.621817. bioRxiv. 2024. PMID: 39605323 Free PMC article. Preprint.
Gain-of-function variants in SMAD4 compromise respiratory epithelial function.
Lindsay ME, Scimone ER, Lawton J, Richa R, Yonker LM, Di YP, Buch K, Ouyang W, Mo X, Lin AE, Mou H. Lindsay ME, et al. Among authors: richa r. J Allergy Clin Immunol. 2025 Jan;155(1):107-119.e2. doi: 10.1016/j.jaci.2024.08.024. Epub 2024 Sep 5. J Allergy Clin Immunol. 2025. PMID: 39243984
34 results