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Optimizing liver health before and after gene therapy for hemophilia A.
Ragni MV, Mead H, de Jong YP, Kaczmarek R, Leavitt AD, Long B, Nugent DJ, Sabatino DE, Fong S, von Drygalski A, Walsh CE, Luxon BA. Ragni MV, et al. Among authors: walsh ce. Blood Adv. 2024 Oct 8;8(19):5203-5212. doi: 10.1182/bloodadvances.2024013059. Blood Adv. 2024. PMID: 38843379 Free PMC article. Review.
BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression.
Konkle BA, Walsh CE, Escobar MA, Josephson NC, Young G, von Drygalski A, McPhee SWJ, Samulski RJ, Bilic I, de la Rosa M, Reipert BM, Rottensteiner H, Scheiflinger F, Chapin JC, Ewenstein B, Monahan PE. Konkle BA, et al. Among authors: walsh ce. Blood. 2021 Feb 11;137(6):763-774. doi: 10.1182/blood.2019004625. Blood. 2021. PMID: 33067633 Free PMC article. Clinical Trial.
99 results