In utero lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.

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.