Biotechnology: alternatives to human plasma-derived therapeutic proteins.
Proteins derived from human plasma have become critically important therapeutic products since their introduction in the 1940s. In the last 20 years, the tools of molecular biology have provided alternatives to the administration of the natural products. Recombinant analogues of Factor VIII and Factor IX are commercially available, and recombinant forms of other plasma proteins are under development. Genetic engineering also provides the opportunity to modify a natural protein to improve the efficiency with which it can be produced in vitro, or to change its therapeutic profile. More efficient production systems, such as transgenic plants or animals, may yield less costly therapies and a wider availability of products that are now in limited supply. Finally, gene therapy offers the prospect of permanently correcting conditions arising from deficiencies in any one of several plasma proteins, freeing individuals from the need to undergo periodic treatments with exogenous proteins.
Lynch
T J
TJ
Division of Hematology, U.S. Food and Drug Administration, Rockville, MD, USA.
D001709:Biotechnology; D001798:Blood Proteins / Q000627:therapeutic use*; D005164:Factor IX / Q000627:therapeutic use; D005169:Factor VIII / Q000627:therapeutic use; D005818:Genetic Engineering; D015316:Genetic Therapy; D006801:Humans; D011994:Recombinant Proteins / Q000627:therapeutic use*; D014841:von Willebrand Factor / Q000627:therapeutic use
D001798:Blood Proteins; D011994:Recombinant Proteins; D014841:von Willebrand Factor; D005169:Factor VIII; D005164:Factor IX