Adenoviral vectors modified for increased and persistent expression of the cystic fibrosis transmembrane conductance regulator gene in human airway epithelia

摘要

Disclosed are recombinant viral vectors for gene transfer to the epithelium of patients. Preferred embodiments include adenoviral vectors designed for the efficient transfer of the DNA encoding the cystic fibrosis transmembrane conductance regulator (CFTR) to human airways in vivo. The present invention provides vectors for the enhanced expression and persistence of a gene such as CFTR, which may allow lower vector doses and/or longer intervals between readministration and which comprise, in operable linkage, the keratin 18 (K18) promoter/enhanier element, an intron, the transgene and a stability enhancing element.