Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs

Muscle represents an attractive target tissue for adeno-associated virus (AAV) vector–mediated gene transfer for hemophilia B (HB). Experience with direct intramuscular (i.m.) administration of AAV vectors in humans showed that the approach is safe but fails to achieve therapeutic efficacy. Here, we...

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Bibliografische gegevens
Hoofdauteurs: Haurigot, Virginia, Mingozzi, Federico, Buchlis, George, Hui, Daniel J, Chen, Yifeng, Basner-Tschakarjan, Etiena, Arruda, Valder R, Radu, Antoneta, Franck, Helen G, Wright, J Fraser, Zhou, Shangzhen, Stedman, Hansell H, Bellinger, Dwight A, Nichols, Timothy C, High, Katherine A
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Nature Publishing Group 2010
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Original Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2911254/
https://ncbi.nlm.nih.gov/pubmed/20424599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.73
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