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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Foilsithe in:Sci Transl Med
Main Authors: 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
Formáid: Artigo
Teanga:Inglês
Foilsithe: 2015
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Article
Rochtain Ar Líne: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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