Balloon Catheter Delivery of Helper-dependent Adenoviral Vector Results in Sustained, Therapeutic hFIX Expression in Rhesus Macaques

Hemophilia B is an excellent candidate for gene therapy because low levels of factor IX (FIX) (≥1%) result in clinically significant improvement of the bleeding diathesis. Helper-dependent adenoviral (HDAd) vectors can mediate long-term transgene expression without chronic toxicity. To determine the...

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Autors principals: Brunetti-Pierri, Nicola, Liou, Aimee, Patel, Priti, Palmer, Donna, Grove, Nathan, Finegold, Milton, Piccolo, Pasquale, Donnachie, Elizabeth, Rice, Karen, Beaudet, Arthur, Mullins, Charles, Ng, Philip
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2012
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3464633/
https://ncbi.nlm.nih.gov/pubmed/22828499
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.143
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