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Functional correction and genome integrity with duplex base editing of β-thalassemic hematopoietic stem cells

Abstract Background Beta-thalassemia is among the most common monogenic disorders, posing a major global health challenge. Editing of genetic modifiers, such as BCL11A erythroid enhancer and HBG promoters, enhances fetal hemoglobin expression and confers major therapeutic potential. Double-strand-br...

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Furkejuvvon:
Váldodahkkit: Nikoletta Y. Papaioannou, Petros Patsali, Julia Klermund, Panayiota L. Papasavva, Geoffroy Andrieux, Lola Koniali, Basma Naiisseh, Soteroula Christou, Maria Sitarou, Marina Kleanthous, Toni Cathomen, Carsten W. Lederer
Materiálatiipa: Artigo
Giella:Inglês
Almmustuhtton: BMC 2026-02-01
Ráidu:Genome Biology
Fáttát:
Base editing
HUDEP-2
HSPCs
Thalassemia
Multiplex DNA editing
Chromosomal aberration
Liŋkkat:https://doi.org/10.1186/s13059-026-03974-7
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