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Updated: Jun 22, 2026

Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model
Published on: October 9, 2018
In utero lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells
Atesh K Worthington1,2, Beltran Borges1,2, Tony Lum1,2
1Department of Surgery, University of California, San Francisco, CA, USA.
Abstract:
In vivo genome editing for hematologic malignancies is limited by inefficient delivery of genome editors to hematopoietic stem cells (HSC) in the bone marrow. To overcome this limitation, we capitalized on the inherent liver tropism of lipid nanoparticles (LNPs) and the liver niche of fetal HSCs. We demonstrate that in utero delivery of LNPs without active targeting ligands to the fetal liver results in potentially therapeutic levels of HSC editing.
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