bioRxiv / medRxiv
open access
In utero and neonatal lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.
Efficient delivery of genome editing reagents to hematopoietic stem cells (HSCs) has limited the development of in vivo gene editing therapies for hematologic disease. Here, we exploit developmental hematopoiesis to enable HSC targeting using clinically scalable lipid nanoparticles (LNPs). During fe
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