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

• প্রধান লেখক: Jian Gao (লেখক), Thorsten Bergmann (লেখক), Wenli Zhang (লেখক), Maren Schiwon (লেখক), Eric Ehrke-Schulz (লেখক), Anja Ehrhardt (লেখক)
• বিন্যাস: গ্রন্থ
• প্রকাশিত: Elsevier, 2019-03-01T00:00:00Z.
• বিষয়গুলি: article
• অনলাইন ব্যবহার করুন: 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...