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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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Bibliografski detalji
Izdano u:Sci Transl Med
Glavni autori: Cantore, Alessio, Ranzani, Marco, Bartholomae, Cynthia C., Volpin, Monica, Della Valle, Patrizia, Sanvito, Francesca, Sergi Sergi, Lucia, Gallina, Pierangela, Benedicenti, Fabrizio, Bellinger, Dwight, Raymer, Robin, Merricks, Elizabeth, Bellintani, Francesca, Martin, Samia, Doglioni, Claudio, D’Angelo, Armando, VandenDriessche, Thierry, Chuah, Marinee K., Schmidt, Manfred, Nichols, Timothy, Montini, Eugenio, Naldini, Luigi
Format: Artigo
Jezik:Inglês
Izdano: 2015
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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