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...

Descripción completa

Detalles Bibliográficos

Autores principales:	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
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	BMC 2026-02-01
Colección:	Genome Biology
Materias:	Base editing HUDEP-2 HSPCs Thalassemia Multiplex DNA editing Chromosomal aberration
Acceso en línea:	https://doi.org/10.1186/s13059-026-03974-7
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!