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

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...

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Bibliographic Details
Published in:Biotechnology letters 2012-08, Vol.34 (8), p.1435-1443
Main Authors: da Rosa, Nathalia Gonsales, Swiech, Kamilla, Picanço-Castro, Virgínia, de Sousa Russo-Carbolante, Elisa Maria, Neto, Mario Abreu Soares, de Castilho-Fernandes, Andrielle, Faça, Vitor Marcel, Fontes, Aparecida Maria, Covas, Dimas Tadeu
Format: Article
Language:English
Subjects:
Animals
Applied Microbiology
bioactive properties
Biochemistry
Biological and medical sciences
Biomedical and Life Sciences
Biotechnology
Blotting, Western
Cell Culture Techniques
Cell Line
Cells
Coagulation
Disease Models, Animal
factor VIII
Factor VIII - biosynthesis
Factor VIII - chemistry
Factor VIII - genetics
Factor VIII - pharmacology
Flow Cytometry
Fundamental and applied biological sciences. Psychology
Genetic Vectors - genetics
Hemophilia
Hemophilia A - drug therapy
Human
Humans
Lentivirus - genetics
Life Sciences
Mathematical analysis
Mice
Microbiology
Original Research Paper
patients
Peptide Fragments - biosynthesis
Peptide Fragments - chemistry
Peptide Fragments - genetics
Peptide Fragments - pharmacology
Platforms
Proteins
Recombinant
Recombinant Proteins - biosynthesis
Recombinant Proteins - chemistry
Recombinant Proteins - genetics
Recombinant Proteins - pharmacology
Stability
Survival Analysis
Vectors (mathematics)
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Summary: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/106 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.
ISSN:0141-5492
1573-6776
DOI:10.1007/s10529-012-0925-4