Developing rDNA products for treatment of hemophilia A.
Current therapy for hemophilia A requires frequent infusion of plasma-derived human factor VIII with the associated drawbacks of potential viral contamination, high cost and limited plasma availability. Factor VIII replacement therapy has been improved through increased knowledge of molecular mechanisms regulating blood coagulation, derived largely from the isolation of the factor VIII gene and its expression in mammalian cells. Homogeneous pure preparations of factor VIII--the largest, most complex protein pharmaceutical produced to date through recombinant DNA technology--can now be produced for successful treatment of hemophilia A.
Kaufman
R J
RJ
Genetics Institute, Cambridge, MA 02140-2387.
D000818:Animals; D003001:Cloning, Molecular; D004274:DNA, Recombinant; D005169:Factor VIII / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use*; D005544:Forecasting; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans; D011994:Recombinant Proteins / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use; D013917:Thrombin / Q000378:metabolism; D014841:von Willebrand Factor / Q000235:genetics / Q000378:metabolism
D004274:DNA, Recombinant; D011994:Recombinant Proteins; D014841:von Willebrand Factor; D005169:Factor VIII; D013917:Thrombin