Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells.

doi:10.1038/s41467-024-49353-3

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Article

Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells.

Nature communications - 11 Jun 2024

Moiani Arianna, Letort Gil, Lizot Sabrina, Chalumeau Anne, Foray Chloe, Felix Tristan, Le Clerre Diane, Temburni-Blake Sonal, Hong Patrick, Leduc Sophie, Pinard Noemie, Marechal Alan, Seclen Eduardo, Boyne Alex, Mayer Louisa, Hong Robert, Pulicani Sylvain, Galetto Roman, Gouble Agnès, Cavazzana Marina, Juillerat Alexandre, Miccio Annarita, Duclert Aymeric, Duchateau Philippe, Valton Julien

Abstract excerpt

Sickle cell disease is a devastating blood disorder that originates from a single point mutation in the HBB gene coding for hemoglobin. Here, we develop a GMP-compatible TALEN-mediated gene editing process enabling efficient HBB correction via a DNA repair template while minimizing risks associated with HBB inactivation. Comparing viral versus non-viral DNA repair template delivery in hematopoietic stem and...

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Identifiers

PMID: 38862518
DOI: 10.1038/s41467-024-49353-3
PMCID: PMC11166989

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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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