Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Adeno-associated virus (AAV)–mediated gene transfer of factor IX (F.IX) to the liver results in long-term expression of transgene in experimental animals, but only short-term expression in humans. Loss of F.IX expression is likely due to a cytotoxic immune response to the AAV capsid, which results i...

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Hlavní autoři: Mingozzi, Federico, Hasbrouck, Nicole C., Basner-Tschakarjan, Etiena, Edmonson, Shyrie A., Hui, Daniel J., Sabatino, Denise E., Zhou, Shangzhen, Wright, J. Fraser, Jiang, Haiyan, Pierce, Glenn F., Arruda, Valder R., High, Katherine A.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Hematology 2007
Témata:
Gene Therapy
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC1988950/
https://ncbi.nlm.nih.gov/pubmed/17609423
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2007-03-080093
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