lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.

bioRxiv : the preprint server for biology
Authors
Atesh Worthington
Beltran Borges
Tony Lum
Elisa Echeverri
Fareha Zada
Marco Cordero
Hyejin Kim
Ryan Zenhausern
Ozgenur Celik
Cindy Shaw
Paula Gutierrez-Martinez
Marzhana Omarova
Chris Blanchard
Sean Burns
Kyle Cromer
James Dahlman
Tippi MacKenzie
Abstract

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 delivery of LNPs without active targeting ligands to the fetal liver results in potentially therapeutic levels of HSC editing.
Year of Publication
2025
Journal
bioRxiv : the preprint server for biology
Date Published
05/2025
ISSN
2692-8205
DOI
10.1101/2025.05.12.652147
PubMed ID
40463054
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