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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...
Autores principales: | Kyostio-Moore, Sirkka, Berthelette, Patricia, Piraino, Susan, Sookdeo, Cathleen, Nambiar, Bindu, Jackson, Robert, Burnham, Brenda, O’Riordan, Catherine R, Cheng, Seng H, Armentano, Donna |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4765533/ https://www.ncbi.nlm.nih.gov/pubmed/26958574 http://dx.doi.org/10.1038/mtm.2016.6 |
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