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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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| Published in: | Mol Ther Nucleic Acids |
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| Main Authors: | , , , , , |
| Format: | Artigo |
| Language: | Inglês |
| Published: |
American Society of Gene & Cell Therapy
2018
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6356096/ https://ncbi.nlm.nih.gov/pubmed/30690229 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.12.008 |
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