title: Production of recombinant coagulation factors: Are humans the best host cells?
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.
authors.affiliation: a Department of Pharmaceutical Sciences , School of Pharmaceutical Sciences of Ribeirão Preto, University of São Paulo , São Paulo , Brazil.|b Center for Cell-based Therapy , Regional Blood Center of Ribeirão Preto, University of São Paulo, Ribeirão Preto-SP , Brazil.
authors.forename: Kamilla
authors.identifier: http://orcid.org/0000-0002-4811-9751
authors.initials: K
authors.lastname: Swiech
authors.affiliation: b Center for Cell-based Therapy , Regional Blood Center of Ribeirão Preto, University of São Paulo, Ribeirão Preto-SP , Brazil.
authors.forename: Virgínia
authors.initials: V
authors.lastname: Picanço-Castro
authors.affiliation: b Center for Cell-based Therapy , Regional Blood Center of Ribeirão Preto, University of São Paulo, Ribeirão Preto-SP , Brazil.|c Department of Internal Medicine , Ribeirão Preto Medical School, University of São Paulo , São Paulo , Brazil.
authors.forename: Dimas Tadeu
authors.identifier: http://orcid.org/0000-0002-7364-2595
authors.initials: DT
authors.lastname: Covas
mesh_terms: D000818:Animals; D001779:Blood Coagulation Factors / Q000096:biosynthesis / Q000235:genetics; D019556:COS Cells; D003001:Cloning, Molecular / Q000379:methods; D005164:Factor IX / Q000096:biosynthesis* / Q000235:genetics*; D005169:Factor VIII / Q000096:biosynthesis* / Q000235:genetics*; D024861:Genetic Enhancement / Q000379:methods*; D057809:HEK293 Cells; D056945:Hep G2 Cells; D006801:Humans; D011487:Protein Conformation; D015202:Protein Engineering / Q000379:methods*; D011994:Recombinant Proteins / Q000096:biosynthesis / Q000235:genetics; D013045:Species Specificity
chemical_list: D001779:Blood Coagulation Factors; D011994:Recombinant Proteins; D005169:Factor VIII; D005164:Factor IX
keywords: factor IX; factor VIII; hemophilia; human cell lines; immunogenicity; recombinant coagulation factors; recombinant protein production