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Enhanced Factor VIII Heavy Chain for Gene Therapy of Hemophilia A
Hemophilia A gene therapy using recombinant adenovirus-associated virus (AAV) vectors has been hampered by the size of the factor VIII (FVIII) cDNA. Previously, splitting the FVIII coding sequence into a heavy-chain (HC) fragment and a light-chain (LC) fragment for dual recombinant AAV vector delive...
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| Hoofdauteurs: | , , , , , , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
Nature Publishing Group
2009
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2790722/ https://ncbi.nlm.nih.gov/pubmed/19127250 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.292 |
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