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.
In utero lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.
子宫内脂质纳米颗粒递送可实现对造血干细胞的强效编辑
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作者：Worthington Atesh K, Borges Beltran, Lum Tony, Echeverri Elisa Schrader, Zada Fareha Moulana, Cordero Marco A, Kim Hyejin, Zenhausern Ryan, Celik Ozgenur, Shaw Cindy, Gutierrez-Martinez Paula, Omarova Marzhana, Blanchard Chris, Burns Sean, Cromer M Kyle, Dahlman James E, MacKenzie Tippi C
| 期刊： | bioRxiv | 影响因子： | 0.000 |
| 时间： | 2025 | 起止号： | 2025 May 12 |
| doi： | 10.1101/2025.05.12.652147 | 研究方向： | 发育与干细胞、细胞生物学 |
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