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

• Main Authors: Jian Gao (Author), Thorsten Bergmann (Author), Wenli Zhang (Author), Maren Schiwon (Author), Eric Ehrke-Schulz (Author), Anja Ehrhardt (Author)
• Format: Book
• Published: Elsevier, 2019-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...