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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Kyostio-Moore, Sirkka, Berthelette, Patricia, Piraino, Susan, Sookdeo, Cathleen, Nambiar, Bindu, Jackson, Robert, Burnham, Brenda, O’Riordan, Catherine R, Cheng, Seng H, Armentano, Donna
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4765533/
https://ncbi.nlm.nih.gov/pubmed/26958574
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.6
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