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Key points: - IVTsgRNAs in K562 predict response to highly effective genome editing in HSPCs - Low cost and efficient nucleofection protocols for RNP based editing in K562 and HSPCs - Genome editing efficiencies in HSPCs up to 80% is independent of cell number, and CD34 subpopulations are equally sensitive for genome editing - CRISPR-Cas9 gene editing does not impact细胞增殖和分化或长期p21诱导细胞衰老
造血茎和祖细胞中高效的基因组编辑
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