Viral Vector-Based Delivery of CRISPR/Cas9 and Donor DNA for Homology-Directed Repair in an In Vitro Model for Canine Hemophilia B

Gene therapy represents an attractive alternative to treat hemophilia B. Here we established three hepatocyte-derived cell lines based on Huh7, PLC/PRF/5, and Hep3B cells stably carrying a mutated canine FIX (cFIXmut) transgene containing a single point mutation in the catalytic domain. Based on the...

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Bibliographic Details

Published in:	Molecular Therapy: Nucleic Acids
Main Authors:	Jian Gao, Thorsten Bergmann, Wenli Zhang, Maren Schiwon, Eric Ehrke-Schulz, Anja Ehrhardt
Format:	Article
Language:	English
Published:	Elsevier 2019-03-01
Online Access:	http://www.sciencedirect.com/science/article/pii/S2162253118303238