Production of recombinant coagulation factors: Are humans the best host cells?

Bioengineered. 2017 Sep 3;8(5):462-470. doi: 10.1080/21655979.2017.1279767. Epub 2017 Feb 23.

Abstract

The main treatment option for Hemophilia A/B patients involves the administration of recombinant coagulation factors on-demand or in a prophylactic approach. Despite the safety and efficacy of this replacement therapy, the development of antibodies against the coagulation factor infused, which neutralize the procoagulant activity, is a severe complication. The production of recombinant coagulation factors in human cell lines is an efficient approach to avoid such complication. Human cell lines can produce recombinant proteins with post translation modifications more similar to their natural counterpart, reducing potential immunogenic reactions. This review provides a brief overview of the most important characteristics of recombinant FVIII and FIX products available on the market and the improvements that have recently been achieved by the production using human cell lines.

Keywords: factor IX; factor VIII; hemophilia; human cell lines; immunogenicity; recombinant coagulation factors; recombinant protein production.

Publication types

  • Review

MeSH terms

  • Animals
  • Blood Coagulation Factors / biosynthesis
  • Blood Coagulation Factors / genetics
  • COS Cells
  • Cloning, Molecular / methods
  • Factor IX / biosynthesis*
  • Factor IX / genetics*
  • Factor VIII / biosynthesis*
  • Factor VIII / genetics*
  • Genetic Enhancement / methods*
  • HEK293 Cells
  • Hep G2 Cells
  • Humans
  • Protein Conformation
  • Protein Engineering / methods*
  • Recombinant Proteins / biosynthesis
  • Recombinant Proteins / genetics
  • Species Specificity

Substances

  • Blood Coagulation Factors
  • Recombinant Proteins
  • Factor VIII
  • Factor IX