In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

Published 2019 View Full Article

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Title
In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods
Authors
Keywords
sickle cell disease, gene editing, rAAV6, ssODN, homology-directed repair, Crispr/Cas9, hemoglobin disorders, NHEJ versus HDR, in vivo, engraftment, NBSGW41 mice, CD34, hematopoietic stem cells, stem cell cures
Journal
Molecular Therapy-Nucleic Acids
Volume 17, Issue -, Pages 277-288
Publisher
Elsevier BV
Online
2019-06-08
DOI
10.1016/j.omtn.2019.05.025

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