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Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

To generate sufficient clinical-grade vector to support a phase I/II clinical trial of adeno-associated virus serotype 8 (AAV8)-mediated factor IX (FIX) gene transfer for hemophilia B, we have developed a large-scale, good manufacturing practice (GMP)-compatible method for vector production and puri...

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Autors principals: Allay, James A., Sleep, Susan, Long, Scott, Tillman, David M., Clark, Rob, Carney, Gael, Fagone, Paolo, McIntosh, Jenny H., Nienhuis, Arthur W., Davidoff, Andrew M., Nathwani, Amit C., Gray, John T.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2011
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3081439/
https://ncbi.nlm.nih.gov/pubmed/21410419
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2010.202
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