Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

In earlier work, we showed that adeno-associated virus–mediated delivery of a Factor IX gene to skeletal muscle by direct intramuscular injection resulted in therapeutic levels of circulating Factor IX in mice. However, achievement of target doses in humans proved impractical because of the large nu...

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Enregistré dans:
Détails bibliographiques
Auteurs principaux: Arruda, Valder R., Stedman, Hansell H., Nichols, Timothy C., Haskins, Mark E., Nicholson, Matthew, Herzog, Roland W., Couto, Linda B., High, Katherine A.
Format: Artigo
Langue:Inglês
Publié: © 2005 by The American Society of Hematology 2005
Sujets:
Gene Therapy
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC1895010/
https://ncbi.nlm.nih.gov/pubmed/15479726
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2004-07-2908
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