Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A

Human coagulation factor VIII (fVIII) is inefficiently biosynthesized in vitro and has proven difficult to express at therapeutic levels using available clinical gene-transfer technologies. Recently, we showed that a porcine and certain hybrid human/porcine fVIII transgenes demonstrate up to 100-fol...

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Autors principals: Doering, Christopher B, Denning, Gabriela, Dooriss, Kerry, Gangadharan, Bagirath, Johnston, Jennifer M, Kerstann, Keith W, McCarty, David A, Spencer, H Trent
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2009
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Original Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835206/
https://ncbi.nlm.nih.gov/pubmed/19259064
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.35
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