Impact of the Underlying Mutation and the Route of Vector Administration on Immune Responses to Factor IX in Gene Therapy for Hemophilia B

Immune responses to factor IX (F.IX), a major concern in gene therapy for hemophilia, were analyzed for adeno-associated viral (AAV-2) gene transfer to skeletal muscle and liver as a function of the F9 underlying mutation. Vectors identical to those recently used in clinical trials were administered...

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Bibliografische gegevens
Hoofdauteurs: Cao, Ou, Hoffman, Brad E, Moghimi, Babak, Nayak, Sushrusha, Cooper, Mario, Zhou, Shangzhen, Ertl, Hildegund CJ, High, Katherine A, Herzog, Roland W
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Nature Publishing Group 2009
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Original Articles
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835008/
https://ncbi.nlm.nih.gov/pubmed/19603001
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.159
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