Targeted genome editing in human repopulating haematopoietic stem cells
Abstract
Targeted genome editing by artificial nucleases has brought the goal of site-specific transgene integration and gene correction within the reach of gene therapy. However, its application to long-term repopulating haematopoietic stem cells (HSCs) has remained elusive. Here we show that poor permissiveness to gene transfer and limited proficiency of the homology-directed DNA repair pathway constrain gene targeting in human HSCs. By tailoring...
Paper Details
Title
Targeted genome editing in human repopulating haematopoietic stem cells
Published Date
May 28, 2014
Journal
Volume
510
Issue
7504
Pages
235 - 240
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