New Strategies in Drug Development Focusing on the Anti-Protease-Protease Balance in Alpha-1 Antitrypsin Deficiency

ISSN: 1875-614X (Online)
ISSN: 1871-5230 (Print)

Volume 16, 3 Issues, 2017

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Anti-Inflammatory & Anti-Allergy Agents in Medicinal Chemistry

Formerly: Current Medicinal Chemistry - Anti-Inflammatory and Anti-Allergy Agents

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Claudiu T. Supuran
Neurofarba Department
University of Florence

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New Strategies in Drug Development Focusing on the Anti-Protease-Protease Balance in Alpha-1 Antitrypsin Deficiency

Anti-Inflammatory & Anti-Allergy Agents in Medicinal Chemistry, 9(4): 314-329.

Author(s): Emer P. Reeves, Sonya Cosgrove, David A. Bergin, Catherine M. Greene and Noel G McElvaney.

Affiliation: Department of Medicine, Royal College of Surgeons in Ireland, Education and Research Centre, Beaumont Hospital, Dublin 9, Ireland.


Alpha-1 antitrypsin (AAT) is the most abundant proteinase inhibitor within the circulation and AAT deficiency is a genetic disorder characterised by serum levels of less than 11μmol/L. The Z mutation is the most common AAT allele associated with the disease and causes the most severe plasma deficiency, as the mutant protein polymerizes and accumulates within the endoplasmic reticulum of hepatocytes. The retained polymers are associated with cirrhosis and reduced serum levels of AAT contribute to the development of chronic pulmonary disease in AAT deficient individuals. This article will review the importance of AAT as a serine anti-protease, the clinical manifestations of AAT deficiency and specific treatment of the disease. Current therapies including AAT replacement and treatment with synthetic or alternative protease inhibitors are reviewed, along with possible future therapies including those focusing on targeting AAT polymer formation or based on gene therapy.


Alpha-1 antitrypsin, alpha-1 antitrypsin deficiency, protease imbalance, replacement therapy, gene therapy, Alpha-1 antitrypsin (AAT), Z mutation, plasma deficiency, cirrhosis, hepatocytes, Serpins, glycosylated, endoplasmic reticulum, protease, neutrophil elastase, neutrophil chemoattractant, pleiomorphic, emphysema, panniculitis, Pseudomonas aeruginosa, Escherichia coli, Klebsiella pneumonia, matrix metalloproteinases, proteoglycans, thrombomodulin, cathepsin D, Toll-like receptors, interleukin, lipopolysaccharides, nuclear factor kappa B, augmentation-therapy, ulcerations, epidermolysis bullosa acquisita, bullosa pemphigoid, Aerosolized AAT, chemokine receptor 1, ofpulmonary hypertension, bleomycin, chemoattractant-1, proteinase 3, cathepsin G, selenoprotein S, phenylbutyric acid, transgenic protein, maleimidopolyethylene glycol, cytomegalovirus, peptide nucleic acids, Computed tomography, Trimethylamine-N-oxide.

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Article Details

Volume: 9
Issue Number: 4
First Page: 314
Last Page: 329
Page Count: 16
DOI: 10.2174/1871523011009040314
Price: $58
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