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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Detalles Bibliográficos
Autores principales: Gao, Jian, Bergmann, Thorsten, Zhang, Wenli, Schiwon, Maren, Ehrke-Schulz, Eric, Ehrhardt, Anja
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6356096/
https://www.ncbi.nlm.nih.gov/pubmed/30690229
http://dx.doi.org/10.1016/j.omtn.2018.12.008

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6356096/
https://www.ncbi.nlm.nih.gov/pubmed/30690229
http://dx.doi.org/10.1016/j.omtn.2018.12.008

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