Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection

Several ongoing clinical studies are evaluating recombinant adeno-associated virus (rAAV) vectors as gene delivery vehicles for a variety of diseases. However, the production of vectors with genomes >4.7 kb is challenging, with vector preparations frequently containing truncated genomes. To deter...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther Methods
Main Authors: Nambiar, Bindu, Cornell Sookdeo, Cathleen, Berthelette, Patricia, Jackson, Robert, Piraino, Susan, Burnham, Brenda, Nass, Shelley, Souza, David, O'Riordan, Catherine R., Vincent, Karen A., Cheng, Seng H., Armentano, Donna, Kyostio-Moore, Sirkka
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2017
Assuntos:
Special Issue on Adeno-Associated Viral (AAV) Vectors:Production, Purification, and Beyond—Part 1Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5314997/
https://ncbi.nlm.nih.gov/pubmed/28166648
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hgtb.2016.124
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