The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo

The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo

Recombinant adeno-associated viral (rAAV) vectors containing oversized genomes provide transgene expression despite low efficiency packaging of complete genomes. Here, we characterized the properties of oversized rAAV2/8 vectors (up to 5.4 kb) encoding human factor VIII (FVIII) under the transcripti...

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Bibliographic Details
Main Authors: Sirkka Kyostio-Moore, Patricia Berthelette, Susan Piraino, Cathleen Sookdeo, Bindu Nambiar, Robert Jackson, Brenda Burnham, Catherine R O'Riordan, Seng H Cheng, Donna Armentano
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S232905011630184X

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