Summary
It is unarguable that results reported in the early trials of somatic gene therapy for CF are equivocal. However, the inappropriately negative response which these studies have created in some quarters should be qualified and countered. Remarkable progress has been made and independent strategies have been tested in the very few years since the CFTR gene was cloned. The inadequacies of the very early generation of adenoviruses were easily predicted, as were the inefficiencies of the first generation cationic liposomes. What is encouraging is, that these early studies have stimulated rapid re-evaluation and further development of second and third generation vectors, which can be tested in laboratory mice engineered to have the CFTR defect as a prelude to clinical trials. As with any new development of clinical medicine, the development of somatic gene therapy protocols for cystic fibrosis is likely to be incremental. Only when safe and effective methods of gene delivery have been developed, will it be appropriate to apply this form of treatment to young CF individuals prior to the onset of irreversible lung damage. Under these circumstances, there is every reason to believe that the rationale underlying CF somatic gene therapy will be realised.
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© 1999 Birkhäuser Verlag Basel
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Porteous, D.J., Innes, J.A. (1999). Gene Therapy for Cystic Fibrosis. In: Blankenstein, T. (eds) Gene Therapy. Birkhäuser Basel. https://doi.org/10.1007/978-3-0348-7011-5_10
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DOI: https://doi.org/10.1007/978-3-0348-7011-5_10
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