SK-HEP cells and lentiviral vector for production of human recombinant factor VIII

Biotechnol Lett. 2012 Aug;34(8):1435-43. doi: 10.1007/s10529-012-0925-4. Epub 2012 Apr 10.

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Blotting, Western
  • Cell Culture Techniques
  • Cell Line
  • Disease Models, Animal
  • Factor VIII / biosynthesis*
  • Factor VIII / chemistry
  • Factor VIII / genetics
  • Factor VIII / pharmacology
  • Flow Cytometry
  • Genetic Vectors / genetics
  • Hemophilia A / drug therapy
  • Humans
  • Lentivirus / genetics*
  • Mice
  • Peptide Fragments / biosynthesis*
  • Peptide Fragments / chemistry
  • Peptide Fragments / genetics
  • Peptide Fragments / pharmacology
  • Recombinant Proteins / biosynthesis*
  • Recombinant Proteins / chemistry
  • Recombinant Proteins / genetics
  • Recombinant Proteins / pharmacology
  • Survival Analysis

Substances

  • B-domain-deleted factor VIII
  • Peptide Fragments
  • Recombinant Proteins
  • Factor VIII