PMID: 11916456Mar 28, 2002Paper

An adventure in biotechnology: the development of haemophilia A therapeutics -- from whole-blood transfusion to recombinant DNA to gene therapy

Biotechnology and Applied Biochemistry
Henry S Kingdon, Roger L Lundblad

Abstract

The past decade has seen an explosion in the number of therapeutic proteins available for a wide spectrum of diseases. Some of these proteins are obtained from human plasma. Examples of these therapeutic proteins are albumin, intravenous immunoglobulins and prothrombin complex concentrates. The majority of new therapeutic proteins are, however, derived via recombinant DNA technology. There are other examples where the first therapeutic preparation was a crude preparation derived from plasma or tissue and where subsequent development has resulted in a recombinant form of the therapeutic protein. This article focuses on the development of therapeutics for the treatment of haemophilia A (deficiency of Factor VIII activity). The progression from crude plasma fractions to monoclonal-purified preparations to the more recent development of therapeutic concentrates via recombinant DNA technology is described in some detail. Finally, the current status of gene therapy for haemophilia A is evaluated. Both technical issues as well as market forces are described, as both have had significant impact on the product-development process.

Citations

Jan 20, 2011·Journal of Biomaterials Science. Polymer Edition·Wen HeQinmei Wang
Feb 18, 2005·Journal of Internal Medicine·H G Klein
Apr 29, 2014·European Journal of Haematology·Massimo Morfini
Aug 30, 2018·The New England Journal of Medicine·Johnny MahlanguRebecca Kruse-Jarres
Nov 15, 2018·Haemophilia : the Official Journal of the World Federation of Hemophilia·Johannes OldenburgSylvia von Mackensen
Mar 10, 2017·Bioengineered·Kamilla SwiechDimas Tadeu Covas
Mar 1, 2009·ISBT Science Series·H G Klein, B J Bryant
Oct 22, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Sonia O'HaraJamie O'Hara
May 10, 2007·Archives of Pathology & Laboratory Medicine·Barbara J Bryant, Harvey G Klein

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