Abstract Alpha-1-antitrypsin (AAT) is an acute phase secretory glycoprotein that inhibits
neutrophil proteases like elastase and is considered as the archetype of a family of …

Genetic modification therapy is a promising therapeutic strategy for many diseases of the
lung intractable to other treatments. Lung gene therapy has been the subject of numerous …

Abstract α1-antitrypsin (AAT) is a circulating serine protease inhibitor secreted from the liver
and important in preventing proteolytic neutrophil elastase associated tissue damage …

Introduction Altering the human genetic code has been explored since the early 1990s as a
definitive answer for the treatment of monogenic and acquired diseases which do not …

Gene therapy is an alternative therapy in many respiratory diseases with genetic origin and
currently without curative treatment. After five decades of progress, many different vectors …

ABSTRACT Alpha-1 antitrypsin deficiency (AATD) is associated with premature onset of
emphysema resulting from low serum A1-PI levels. The only available pharmacological …

Cystic fibrosis (CF) is an autosomal recessive genetic disorder arising from mutations to the
cystic fibrosis transmembrane conductance regulator (CFTR) gene. Disruption to normal ion …

While adeno-associated viral (AAV) vectors are successfully used in a variety of in vivo gene
therapy applications, they continue to be hampered by the immune system. Here, we sought …

Alpha 1-antitrypsin deficiency (AATD) is the most common genetic cause of liver disease in
children and is associated with early-onset chronic liver disease in adults. AATD associated …

Abstract Introduction Alpha-1 antitrypsin (AAT) deficiency is an autosomal co-dominant
genetic condition that predisposes individuals to pulmonary and hepatic disease, and in …