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- W2895598593 abstract "Sickle Cell Disease (SCD), one of the world9s most common genetic disorders, causes anemia and progressive multiorgan damage that typically shortens lifespan by decades; currently there is no broadly applicable curative therapy. Here we show that Cas9 RNP-mediated gene editing with an ssDNA oligonucleotide donor yields more than 20% correction of the sickle mutation in long-term engrafting human HSCs. Using RNA-seq, we further find that in vivo erythroid differentiation markedly enriches for cells carrying corrected β-globin alleles. Adoption of a high-fidelity Cas9 variant demonstrates that this approach can yield efficient editing with almost no off-target events. These findings indicate that the sickle mutation can be corrected in human HSCs at levels that are likely to be curative if translated into a therapy." @default.
- W2895598593 created "2018-10-12" @default.
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- W2895598593 date "2018-10-03" @default.
- W2895598593 modified "2023-10-17" @default.
- W2895598593 title "In vivo selection for corrected β-globin alleles after CRISPR/Cas9 editing in human sickle hematopoietic stem cells enhances therapeutic potential" @default.
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