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Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells

2 Jan 2024

Abstract excerpt

Genome editing by homology directed repair (HDR) is leveraged to precisely modify the genome of therapeutically relevant hematopoietic stem and progenitor cells (HSPCs). Here, we present a new approach to increasing the frequency of HDR in human HSPCs by the delivery of an inhibitor of 53BP1 (named "i53") as a recombinant peptide. We show that the use of i53 peptide effectively increases the frequency of...

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PMID: 38169468
DOI: 10.1038/s41467-023-43413-w

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