Original paper
Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking
Abstract
Targeted gene editing in hematopoietic stem cells (HSCs) is a promising treatment for several diseases. However, the limited efficiency of homology-directed repair (HDR) in HSCs and the unknown impact of the procedure on clonal composition and dynamics of transplantation have hampered clinical translation. Here, we apply a barcoding strategy to clonal tracking of edited cells (BAR-Seq) and show that editing activates p53, which substantially...
Paper Details
Title
Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking
Published Date
Jun 29, 2020
Journal
Volume
38
Issue
11
Pages
1298 - 1308
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Notes
History