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

20260 citationsJournal Articlegold Open Access

Authors

Nikoletta Y. Papaioannou · Cyprus Institute of Neurology and Genetics

Petros Patsali · Cyprus Institute of Neurology and Genetics

Julia Klermund · University of Freiburg

Panayiota L. Papasavva · Cyprus Institute of Neurology and Genetics

Geoffroy Andrieux · University of Freiburg

Lola Koniali · Cyprus Institute of Neurology and Genetics

Basma Naiisseh

Abstract

Duplex BE targeting both BCL11A erythroid enhancer and HBG promoter enables functional correction and genome integrity. Our study highlights the efficacy, safety, and therapeutic potential of the present duplex BE approach.

Topics & Keywords

Hemoglobinopathies and Related Disorders CRISPR and Genetic Engineering Acute Myeloid Leukemia Research

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Genome biology

Volume 27, Issue 1

DOI: 10.1186/s13059-026-03974-7

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