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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Bibliographic Details
Main Authors: Johnston, Jennifer M., Denning, Gabriela, Doering, Christopher B., Spencer, H. Trent
Format: Artigo
Language:Inglês
Published: 2012
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Article
Online Access: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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