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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Bibliografiska uppgifter
I publikationen:Sci Transl Med
Huvudupphovsmän: 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
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2015
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Article
Länkar: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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