The two most common hereditary lung disorders in Caucasians, α 1-antitrypsin (α 1-AT)
deficiency and cystic fibrosis, have their major clinical manifestations in the lung. Rapid …

The common fatal hereditary disorders, alpha 1-antitrypsin (alpha 1AT) deficiency and cystic
fibrosis (CF), are clinical models for the common lung diseases, emphysema and chronic …

The past decade has brought significant advances in the field of gene therapy for both
inherited and acquired diseases, especially with regard to respiratory disease. Barriers to …

Given both the accessibility and the genetic basis of several pulmonary diseases, the lungs
and airways initially seemed ideal candidates for gene therapy. Several routes of access are …

Over the last decade, numerous investigators have pursued gene transfer as a definitive
treatment for inherited airway diseases, and although the promise of “gene therapy” appears …

The lung represents an attractive target organ for somatic gene therapy strategy in that,(1) it
is easily accessible by vectors,(2) most frequent hereditary disorders, cystic fibrosis (CF) and …

In vivo gene transfer to the lungs is possible either by an intravenous or an airway route of
administration. A plasmid containing the recombinant human alpha 1-antitrypsin (h alpha …

We evaluated the potential of lung-directed gene therapy for α1-antitrypsin (AAT) deficiency
using an adeno-associated virus type 6 (AAV6) vector containing a human AAT (hAAT) …

Over the 2 years covered here, there has been one clinical study in which a normal alpha-1
antitrypsin (AAT) gene was delivered to the nasal epithelium of AAT-deficient subjects using …

▪ Abstract Gene therapy as a treatment modality for pulmonary disorders has attracted
significant interest over the past decade. Since the initiation of the first clinical trials for cystic …