Nature Communications (Oct 2019)

Functional profiling of single CRISPR/Cas9-edited human long-term hematopoietic stem cells

  • Elvin Wagenblast,
  • Maria Azkanaz,
  • Sabrina A. Smith,
  • Lorien Shakib,
  • Jessica L. McLeod,
  • Gabriela Krivdova,
  • Joana Araújo,
  • Leonard D. Shultz,
  • Olga I. Gan,
  • John E. Dick,
  • Eric R. Lechman

DOI
https://doi.org/10.1038/s41467-019-12726-0
Journal volume & issue
Vol. 10, no. 1
pp. 1 – 11

Abstract

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Previous gene editing in haematopoietic stem cells (HSCs) has focussed on a heterogeneous CD34+ population. Here, the authors demonstrate high efficiency CRISPR/Cas9-based editing of purified long-term HSCs using non-homologous end joining and homology-directed repair, by directing isoform-specific expression of GATA1.