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

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

Gene editing following designer nuclease cleavage in the presence of a DNA donor template can revert mutations in disease-causing genes. For optimal benefit, reversion of the point mutation in HBB leading to sickle cell disease (SCD) would permit precise homology-directed repair (HDR) while concurre...

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Bibliographic Details
Main Authors: Sowmya Pattabhi, Samantha N. Lotti, Mason P. Berger, Swati Singh, Christopher T. Lux, Kyle Jacoby, Calvin Lee, Olivier Negre, Andrew M. Scharenberg, David J. Rawlings
Format: Article
Language:English
Published: Elsevier 2019-09-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253119301568

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http://www.sciencedirect.com/science/article/pii/S2162253119301568

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