Background The efficacy of α1 proteinase inhibitor (A1PI) augmentation treatment for α1
antitrypsin deficiency has not been substantiated by a randomised, placebo-controlled trial. CT-…

A 60-year-old white man presents for evaluation of progressive dyspnea. He is a former
smoker with a 20-pack-year smoking history and a 10-year history of chronic obstructive …

Recombinant adeno-associated virus (rAAV) vectors offer promise for the gene therapy of α
1 -antitrypsin (AAT) deficiency. In our prior trial, an rAAV vector expressing human AAT (…

Human neutrophilic polymorphonuclear leukocyte (PMN) elastase was purified by affinity
chromatography to greater than 95 per cent homogeneity as judged by disc-gel electrophoresis…

Background and study objectives α 1 -Antitrypsin (AAT) deficiency is common but underrecognized.
A 1994 mail survey showed a long delay between the onset of symptoms and the …

Background: The diagnosis and clinical management of adults with alpha-1 antitrypsin
deficiency (AATD) have been the subject of ongoing debate, ever since the publication of the first …

The metabolic turnover of mature elastin fibers in adult animals is relatively slow. Although
only small amounts of elastin are degraded normally, increased degradation and …

Background α 1 -Antitrypsin deficiency (AATD) is usually underrecognized, with only 5% of
cases being diagnosed in the estimated 100,000 affected individuals in the United States. To …

Recombinant adeno-associated virus (rAAV) vectors have shown promise for the treatment
of several diseases; however, immune-mediated elimination of transduced cells has been …

Background: Severe α 1 -antitrypsin (AAT) deficiency is an autosomal recessive genetic
condition associated with an increased but variable risk for chronic obstructive pulmonary …