Availability of clotting factor concentrates in genetically engineered form.
Recombinant clotting factor concentrates provide new opportunities for the treatment of hemostatic disorders such as haemophilia A and B and von Willebrand disease. This article briefly reviews the clinical results of currently available products, the development of concentrates of clotting factor IX and von Willebrand factor and the outlook of second generation products (e.g., deletion mutants) and gene therapy of haemophilia. Further elucidation of the structure/function relationship of plasma proteins will probably lead to a new category of medicinal products for the treatment of bleeding and thrombotic diseases.
van Aken
W G
WG
Central Laboratory of The Netherlands Red Cross Blood Transfusion Service, Amsterdam, The Netherlands.
D000818:Animals; D001779:Blood Coagulation Factors / Q000235:genetics* / Q000600:supply & distribution; D015316:Genetic Therapy; D006801:Humans; D015202:Protein Engineering; D011994:Recombinant Proteins / Q000235:genetics / Q000600:supply & distribution
D001779:Blood Coagulation Factors; D011994:Recombinant Proteins