In utero and neonatal lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.
Atesh K. Worthington, Beltran Borges, Elisa Schrader Echeverri, Fareha Moulana Zada, Tony Lum, Andrew Davis, Kun Jia, Barbara Perez, Amanda Graveson, Daniel Oder, Marco A. Cordero, Hyejin Kim, Ryan Zenhausern, Ozgenur Celik, Travis McCreary, , Tippi C. MacKenzie
This study demonstrates efficient in utero and neonatal lipid nanoparticle delivery for genome editing of hematopoietic stem cells, achieving robust editing and multilineage reconstitution.
bioRxiv · May 12, 2025 · PreprintCell Biology