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Engineered lipid nanoparticles for in vivo and durable editing of haematopoietic stem cells within humanized mice

Academic source · Nature Biomedical Engineering

Source published: 2026-08-05

Site published: 2026-11-03 · Updated: 2026-11-03

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What the paper reports

Anti-CD34-conjugated LNPs delivered reporter mRNA and gene-editing cargos to human hematopoietic stem and progenitor cells. Intrafemoral administration in humanized mice supported durable BCL11A-enhancer editing and fetal hemoglobin reactivation; ELANE editing partially restored neutrophil development in another model. The study addresses difficult stem-cell targeting but uses a specialized administration route and preclinical systems.

Why this paper is included

Advances extrahepatic delivery to long-lived human stem cells; intrafemoral mouse experiments do not establish routine systemic treatment feasibility.

The interpretation above is analysis, not a conclusion attributed to the original source.

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mRNA / LNP

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