Gene Therapy Via Aerosol

Gene therapy delivered by aerosol for lung disease is the goal of intensive research. The primary focus is the treatment of CF. Cationic-lipid-mediated CFTR gene transfer can significantly influence the underlying chloride defect in the lungs of patients with CF [179]. There are many problems to be overcome before clinical applications are practical. Some of these are safety, successful transfer of sufficient genetic material to appropriate tissue, adequate gene expression, maintenance of expression over time, and efficacy of expression. Early work focused on adenovirus as a vector for transfection [180-184]. Complications caused by reactions to adenovirus caused investigators to search for other mechanisms, such as lipid-based particles [185-189]. The liposomal material chosen can significantly affect the mass of aerosol produced [190] and can also influence the effect of the nebulization process on the efficiency of gene transfer [191]. Targeting of specific airway generations is also a consideration, depending upon the gene and the disease of interest [192].

Much work remains in order to bring about the use of gene transfer for the successful management of CF. The final clinical approach may well involve aerosol delivery of vectors.

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