Abstract
Although gene therapy is still in its infancy, the lung has received a good deal of attention as a target organ. This reflects the fact that the lung appears to be a favorable site for gene therapy; it is a relatively simple organ with the target cells in most instances lining its lumen, vectors can be delivered to a portion of the lung or the whole lung without a surgical procedure, and the success of delivery and efficacy of a vector can be assessed non-invasively. Another measure of the perceived practicality of lung gene therapy is the relatively large number of applications dealing with this organ that have been submitted in the United States to the Recombinant Advisory Committee (RAC), a regulatory agency of the National Institutes of Health that publicly reviews all such protocols. In addition to having important applications for lung disease, there is the clear hope that important lessons can be learned from trials in the lung that will aid in the design of gene therapy treatments for other organs.
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Wadsworth, S.C., Smith, A.E. (1995). Cystic fibrosis and lung diseases. In: Dickson, G. (eds) Molecular and Cell Biology of Human Gene Therapeutics. Molecular and Cell Biology of Human Diseases Series, vol 20. Springer, Dordrecht. https://doi.org/10.1007/978-94-011-0547-7_12
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DOI: https://doi.org/10.1007/978-94-011-0547-7_12
Publisher Name: Springer, Dordrecht
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