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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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Detalles Bibliográficos
Autores principales:	Johnston, Jennifer M., Denning, Gabriela, Doering, Christopher B., Spencer, H. Trent
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2012
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3552131/ https://www.ncbi.nlm.nih.gov/pubmed/22996197 http://dx.doi.org/10.1038/gt.2012.76

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