In Vivo RNA Delivery to Hematopoietic Stem and Progenitor Cells via Targeted Lipid Nanoparticles

Ex vivo autologous hematopoietic stem cell (HSC) gene therapy has provided new therapies for the treatment of hematological disorders. However, these therapies have several limitations owing to the manufacturing complexities and toxicity resulting from required conditioning regimens. Here, we develo...

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Bibliographic Details
Main Authors: Shi, Dennis, Toyonaga, Sho, Anderson, Daniel G
Other Authors: Massachusetts Institute of Technology. Department of Chemical Engineering
Format: Article
Language:English
Published: American Chemical Society 2024
Online Access:https://hdl.handle.net/1721.1/156899