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U.S. Department of Health and Human Services

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Amino Acids 2006 Jun;30(4):317-32

Recombinant human alpha-1 proteinase inhibitor: towards therapeutic use.

Karnaukhova E, Ophir Y, Golding B

Karnaukhova E, US FDA, Div Hematol, Ctr Biol Evaluat & Res, 8800 Rockville Pike,Natl Inst Hlth Bldg 29, Bethesda, MD 20892 USA US FDA, Div Hematol, Ctr Biol Evaluat & Res, Bethesda, MD 20892 USA


Human alpha-1-proteinase inhibitor is a well-characterized protease inhibitor with a wide spectrum of anti-protease activity. Its major physiological role is inhibition of neutrophil elastase in the lungs, and its deficiency is associated with progressive ultimately fatal emphysema. Currently in the US, only plasma-derived human alpha-1-proteinase inhibitor is available for augmentation therapy, which appears to be insufficient to meet the anticipated clinical demand. Moreover, despite effective viral clearance steps in the manufacturing process, the potential risk of contamination with new and unknown pathogens still exists. In response, multiple efforts to develop recombinant versions of human alpha-1-proteinase inhibitor, as an alternative to the plasma-derived protein, have been reported. Over the last two decades, various systems have been used to express the human gene for alpha-1-proteinase inhibitor. This paper reviews the recombinant versions of human alpha-1-proteinase inhibitor produced in various hosts, considers current major safety and efficacy issues regarding recombinant glycoproteins as potential therapeutics, and the factors that are impeding progress in this area(1).

Category: Journal Article, Review
PubMed ID: #16773239
Includes FDA Authors from Scientific Area(s): Biologics
Entry Created: 2011-10-04 Entry Last Modified: 2012-08-29