In utero lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.
Journal: BioRxiv : The Preprint Server For Biology
Published:
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.
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, M Cromer, James Dahlman, Tippi Mackenzie