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Updated: Aug 10, 2026

Identifying DNA Mutations in Purified Hematopoietic Stem/Progenitor Cells
Published on: February 24, 2014
Base editors dissect genetic variants in human hematopoietic cells on a large scale
1Department of Oncological Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Center for Advancement of Blood Cancer Therapies, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Abstract:
In a recent study, Martin-Rufino and colleagues combined massively parallel base editing in primary human hematopoietic stem and progenitor cells (HSPCs) with functional and single-cell transcriptomic readouts. A series of proof-of-principle experiments highlight the breadth of applications made possible with this approach, which range from gene therapy and immunotherapy, to characterizing single nucleotide variants.
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