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

Author

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, and Eric R. Lechman

Subjects
Science
Abstract

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.

Published
2019
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