Article

Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells

2024-06-03

Abstract excerpt

Precision gene editing in primary hematopoietic stem and progenitor cells (HSPCs) would facilitate both curative treatments for monogenic disorders as well as disease modelling. Precise efficiencies even with the CRISPR/Cas system, however, remain limited. Through an optimization of guide RNA delivery, donor design, and additives, we have now obtained mean precise editing efficiencies >90% on primary cord blood HS...

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Literature Corpus work: 5a6b91f5-b005-5011-b1e0-beb13dbfdf32
DOI: 10.7554/elife.91288.3

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