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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Bibliografske podrobnosti
izdano v: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
Format: Artigo
Jezik:Inglês
Izdano: 2015
Teme:
Article
Online dostop: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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