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- W4313387536 abstract "Abstract 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) can deliver mRNA encoding a reporter or a gene editing protein to HSC, with one injection transfecting ∼25% of mouse HSC, and repeated doses resulting in higher editing efficiencies. We also demonstrate LNP-driven in vivo mRNA delivery to HSC in non-human primates and humanized mice. These results demonstrate a translatable approach to deliver mRNA encoding therapeutic proteins, or gene correcting tools, to HSC that do not require cell culture or toxic conditioning. One-Sentence Summary LNP can deliver functional mRNA to mouse, non-human primate, and human HSC." @default.
- W4313387536 created "2023-01-06" @default.
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- W4313387536 date "2022-12-18" @default.
- W4313387536 modified "2023-10-10" @default.
- W4313387536 title "Functional mRNA delivery to hematopoietic stem and progenitor cells<i>in vivo</i>" @default.
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- W4313387536 doi "https://doi.org/10.1101/2022.12.15.520650" @default.
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