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Article

DNA base editing corrects common hemophilia A mutations and restores factor VIII expression in in vitro and ex vivo models.

Journal of thrombosis and haemostasis : JTH - 1 Aug 2024

Tonetto Elena, Cucci Alessia, Follenzi Antonia, Bernardi Francesco, Pinotti Mirko, Balestra Dario

Abstract excerpt

BACKGROUND: Replacement and nonreplacement therapies effectively control bleeding in hemophilia A (HA) but imply lifelong interventions. Authorized gene addition therapy could provide a cure but still poses questions on durability. FVIIIgene correction would definitively restore factor (F)VIII production, as shown in animal models through nuclease-mediated homologous recombination (HR). However, low efficiency...

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Identifiers

PMID: 38718928
DOI: 10.1016/j.jtha.2024.04.020

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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