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Published on: October 24, 2016
SK-HEP cells and lentiviral vector for production of human recombinant factor VIII
Nathalia Gonsales da Rosa1, Kamilla Swiech, Virgínia Picanço-Castro
1Center for Cell Therapy and Regional Blood Center of Ribeirão Preto, University of São Paulo (USP), Ribeirão Preto, 14051-140, Brazil. nathaliagrosa@gmail.com
Abstract:
Hemophilia A is caused by a deficiency in coagulation factor VIII. Recombinant factor VIII can be used as an alternative although it is unavailable for most patients. Here, we describe the production of a human recombinant B-domain-deleted FVIII (rBDDFVIII) by the human cell line SK-HEP-1, modified by a lentiviral vector rBDDFVIII was produced by recombinant SK-HEP cells (rSK-HEP) at 1.5-2.1 IU/10(6) in 24 h. The recombinant factor had increased in vitro stability when compared to commercial pdFVIII. The functionality of rBDDFVIII was shown by its biological activity and by tail-clip challenge in hemophilia A mice. The rSK-HEP cells grew in a scalable system and produced active rBDDFVIII, indicating that this platform production can be optimized to meet the commercial production scale needs.
