Journal
TRENDS IN MOLECULAR MEDICINE
Volume 20, Issue 2, Pages 105-115Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.molmed.2013.11.006
Keywords
alpha-1-antitrypsin deficiency; gene therapy; biomarkers
Funding
- Grifols
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The recognition of alpha-1-antitrypsin deficiency, its function, and its role in predisposition to the development of severe emphysema was a watershed in our understanding of the pathophysiology of the condition. This led to the concept and development of intravenous replacement therapy used worldwide to protect against lung damage induced by neutrophil elastase. Nevertheless, much remained unknown about the deficiency and its impact, although in recent years the genetic and clinical variations in manifestation have provided new insights into assessing impact, efficacy of therapy, and development of new therapeutic strategies, including gene therapy, and outcome measures, such as biomarkers and computed tomography. The current article reviews this progress over the preceding 50 years.
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