Article

Functional mRNA delivery to hematopoietic stem and progenitor cells <i>in vivo</i>

2022-12-18

Abstract excerpt

Gene correction of hematopoietic stem cells (HSC) is a promising therapeutic approach for multiple disorders. Current methods, however, require HSC collection from patients, gene correction during ex vivo culture, and re-infusion of corrected HSC into patients conditioned with chemotherapeutic agents. These approaches are complex, and the conditioning creates toxicities. We show that a lipid nanoparticle (LNP) ca...

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Identifiers and source

Literature Corpus work: 4fa7d2ea-1190-5f95-b301-205ea4e0fc09
DOI: 10.1101/2022.12.15.520650

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