[Somatic gene therapy of mucoviscidosis using recombinant adenovirus: the start of a long course].
The identification of the gene on which mutations cause cystic fibrosis has opened the way to the concept of gene therapy of this disease. Somatic gene therapy for cystic fibrosis should be directed to the epithelial cells of the bronchi where the biological defect is the most deleterious. In order to have the normal DNA coding sequence penetrate the bronchial epithelial cells of a patient, a gene vector is necessary, and recombinant adenoviruses have been the subject of extensive studies in this regard. Data are available from many laboratories as well as from several phase I clinical trials. Successes and limits of this approach are reviewed here. The advantage of non-viral vectors, in particular cationic liposomes, as an alternative to recombinant adenoviruses is briefly discussed. Significant advances still need to be made before gene therapy becomes a reality in cystic fibrosis. These future improvements should include the construction of more efficient gene vectors, and also the development of better tools for the evaluation of a biological effect of gene therapy in individual patients.
Rochat
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Département de médecine, Hôpitaux Universitaires de Genève.
D000256:Adenoviridae / Q000235:genetics / Q000276:immunology; D000818:Animals; D001980:Bronchi / Q000502:physiology; D003550:Cystic Fibrosis / Q000628:therapy*; D004848:Epithelium / Q000502:physiology; D018390:Gene Targeting; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D008081:Liposomes; D011994:Recombinant Proteins / Q000235:genetics / Q000276:immunology
D008081:Liposomes; D011994:Recombinant Proteins
Thérapie génique somatique de la mucoviscidose par adénovirus recombinants: les débuts d'un long parcours.