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Generation of an Optimized Lentiviral Vector Encoding a High-Expression Factor VIII Transgene for Gene Therapy of Hemophilia A
We previously compared the expression of several human factor VIII (fVIII) transgene variants and demonstrated the superior expression properties of B domain deleted porcine fVIII. Subsequently, a hybrid human/porcine fVIII molecule (HP-fVIII) comprising 91% human amino acid sequence was engineered...
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| Auteurs principaux: | , , , |
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| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
2012
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3552131/ https://ncbi.nlm.nih.gov/pubmed/22996197 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2012.76 |
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