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Liver-Directed Lentiviral Gene Therapy in a Dog Model of Hemophilia B
We investigated the safety and efficacy of liver-directed gene therapy using lentiviral vectors in a large animal model of hemophilia B, and evaluated the risk of insertional mutagenesis in tumor-prone mouse models. We show that gene therapy using lentiviral vectors targeting expression of a canine...
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| Izdano u: | Sci Transl Med |
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| Glavni autori: | , , , , , , , , , , , , , , , , , , , , , |
| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
2015
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| Teme: | |
| Online pristup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5669486/ https://ncbi.nlm.nih.gov/pubmed/25739762 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.aaa1405 |
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