CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.
Bortolussi G, Iaconcig A, Canarutto G, Porro F, Ferrucci F, Galletta C, Díaz-Muñoz C, Rawat V, De Caneva A, Olajide OJ, Zentilin L, Piazza S, Bočkor L, Muro AF.
Bortolussi G, et al. Among authors: ferrucci f.
Mol Ther Methods Clin Dev. 2023 Nov 19;31:101161. doi: 10.1016/j.omtm.2023.101161. eCollection 2023 Dec 14.
Mol Ther Methods Clin Dev. 2023.
PMID: 38094199
Free PMC article.