Gene Delivery to Airways
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Experimental evidence for introducing functional genes into tissues and organs has opened new therapeutic approaches to reverse genetic or acquired pathologies. Gene therapy strategies applied to airway disorders (such as cystic fibrosis (CF), cancer and α1-antitrypsin (α1-AT) deficiency) have recently been implemented. At present the direct delivery of beneficial genes to diseased airway cells is accomplished using either replication defective viral vectors, based on the adenovirus, adeno-associated virus (AAV), retroviras genome, or nonviral synthetic vectors, such as cationic lipids complexing plasmid DNA (for review see ). Virus-based vectors efficiently transfer foreign genes to different target airway cells. However, safety considerations and limitations, such as host immune response, may limit their application to certain diseases. On the other hand, synthetic vectors are interesting candidates owing to their putative immunotolerance and low toxicity. However, their gene transfer efficiency needs to be increased. Several clinical protocols involving patients with cystic fibrosis, α1 AT deficiency and lung cancer have been approved and conducted in the USA and in Europe. The vast majority involves CF patients.
KeywordsCystic Fibrosis Cystic Fibrosis Transmembrane Conductance Regulator Recombinant Adenovirus Nasal Epithelium Cystic Fibrosis Transmembrane Conductance Regulator Gene
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