Therapies targeting the airways

ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY 

Gene therapy targets from the airway compartment 

Recombinant AAV serotype 2 vectors were developed for CFTR gene transfer. These vectors were designed to overcome the intrinsic packaging limitation of rAAV by fitting the relatively large coding sequence of CFTR (approximately 4.5 kb), with minimal promoter 

Extracellular barriers (DNA, enzymes, mucus) Paucity of receptors Proteosome-mediated degradation Inhibition of second-strand synthesis Airway clearance, anti-inflammatory, anti-protease pre-treatments Alternate serotypes, targeted capsid mutants Proteosome inhibitors (tripeptides, anthracyclines) Tyrosine kinase inhibitors 

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