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Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells

25 May 2022

Abstract excerpt

Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) is proving successful to treat several genetic diseases. HSPCs are mobilized, harvested, genetically corrected ex vivo, and infused, after the administration of toxic myeloablative conditioning to deplete the bone marrow (BM) for the modified cells. We show that mobilizers create an opportunity for seamless engraftment of exogenous cells, which effectively...

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PMID: 35617958
DOI: 10.1016/j.cell.2022.04.039

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