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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Detalhes bibliográficos
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2011
Assuntos:
Methods
Acesso em linha: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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