Analysis of factor VIII mediated suppression of lentiviral vector titres

• Published in: Gene therapy Vol. 15; no. 4; pp. 289 - 297
• Main Authors: Radcliffe, P A, Sion, C J M, Wilkes, F J, Custard, E J, Beard, G L, Kingsman, S M, Mitrophanous, K A
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.02.2008; Nature Publishing Group
• Subjects: Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Applied cell therapy and gene therapy; Biological and medical sciences; Biomedical and Life Sciences; Biomedicine; Biotechnology; Cell Biology; Cell Line; Coagulation factors; Codon; Factor VIII - genetics; Fundamental and applied biological sciences. Psychology; Gene Expression; Gene Therapy; Genetic Therapy; Genetic Vectors; Genomes; Health. Pharmaceutical industry; Hemophilia; Hemophilia A - therapy; Human Genetics; Humans; Immune response; Industrial applications and implications. Economical aspects; Infectious Anemia Virus, Equine - genetics; Lentivirus; Medical sciences; Nanotechnology; original-article; Polymerase Chain Reaction; Producer cells; Promoter Regions, Genetic; RNA-directed DNA polymerase; Stomatitis; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Vesicular stomatitis virus; Viruses; haemophilia; codon optimization; pseudotyping; lentiviral vectors; factor VIII; Suppression; Factor VIII; Retroviridae; Hemopathy; Lentivirus; Hemophilia A; Optimization; Genetic disease; Virus; Codon; Gene therapy; Vector; Coagulopathy

Effective gene therapy for haemophilia A necessitates a vector system that is not subject to a pre-existing immune response, has adequate coding capacity, gives long-term expression and preferably can target non-dividing cells. Vector systems based on lentiviruses such as equine infectious anaemia v...