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Optimized guide RNA selection recommendations for using <i>sp</i> Cas9 gene editing in human hematopoietic stem and progenitor cells

2020-12-19

Abstract excerpt

Ribonucleoproteins (RNPs) are frequently applied for therapeutic gene editing as well as fundamental research, because the method is fast, viral free, and does not rely on clonal selection. We evaluated various parameters to genetically engineer human hematopoietic stem progenitor cells (HSPCs) using sp Cas9-RNPs and achieve gene editing efficiencies up to 80%. We find that single guide RNA (sgRNA) design is crit...

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Literature Corpus work
983e88bf-07df-5016-8d23-4a85d1cd7b32
DOI
10.1101/2020.12.18.423329
Open publication

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Optimized guide RNA selection recommendations for using <i>sp</i> Cas9 gene editing in human hematopoietic stem and progenitor cellsDOI 10.1101/2020.12.18.423329
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