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DNA base editing corrects common Hemophilia A mutations and restores factor VIII expression in vitro and ex-vivo models.
J Thromb Haemost. 2024 May 6:S1538-7836(24)00246-0. doi: 10.1016/j.jtha.2024.04.020. Online ahead of print.
J Thromb Haemost. 2024.
PMID: 38718928
Counteracting the Common Shwachman-Diamond Syndrome-Causing SBDS c.258+2T>C Mutation by RNA Therapeutics and Base/Prime Editing.
Peretto L, Tonetto E, Maestri I, Bezzerri V, Valli R, Cipolli M, Pinotti M, Balestra D.
Peretto L, et al. Among authors: tonetto e.
Int J Mol Sci. 2023 Feb 16;24(4):4024. doi: 10.3390/ijms24044024.
Int J Mol Sci. 2023.
PMID: 36835434
Free PMC article.
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