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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Hlavní autoři: Arruda, Valder R., Stedman, Hansell H., Nichols, Timothy C., Haskins, Mark E., Nicholson, Matthew, Herzog, Roland W., Couto, Linda B., High, Katherine A.
Médium: Artigo
Jazyk:Inglês
Vydáno: © 2005 by The American Society of Hematology 2005
Témata:
Gene Therapy
On-line přístup: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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