Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis
作者： Joseph Zabner , Larry A. Couture , Richard J. Gregory , Scott M. Graham , Alan E. Smith
DOI: 10.1016/0092-8674(93)80063-K
关键词:
摘要: To evaluate the potential of direct transfer cystic fibrosis transmembrane conductance regulator (CFTR) cDNA for treatment (CF), we administered an E1-deficient adenovirus, encoding CFTR, to a defined area nasal airway epithelium three individuals with CF. This corrected Cl- transport defect that is characteristic CF-affected epithelia. After treatment, there was decrease in elevated basal transepithelial voltage, and normal response cAMP agonist restored. We found no evidence viral replication or virus-associated adverse effects, even at highest dose tested (25 MOI). These data represent small step achieving long-term improvement CF lung function by gene therapy.
elsevier.com
sci-hub.se 参考文章(30)
F.L. Graham, L. Prevec, Adenovirus-based expression vectors and recombinant vaccines. Vaccines#R##N#New Approaches to Immunological Problems. ,vol. 20, pp. 363- 390 ,(1992) , 10.1016/B978-0-7506-9265-6.50022-1
David Dickson, UK scientists test liposome gene therapy technique Nature. ,vol. 365, pp. 4- 4 ,(1993) , 10.1038/365004A0
Paul M. Quinton, Cystic fibrosis: a disease in electrolyte transport The FASEB Journal. ,vol. 4, pp. 2709- 2710 ,(1990) , 10.1096/FASEBJ.4.10.2197151
J. H. Widdicombe, M. J. Welsh, W. E. Finkbeiner, Cystic fibrosis decreases the apical membrane chloride permeability of monolayers cultured from cells of tracheal epithelium. Proceedings of the National Academy of Sciences of the United States of America. ,vol. 82, pp. 6167- 6171 ,(1985) , 10.1073/PNAS.82.18.6167
H. S. Ginsberg, L. L. Moldawer, P. B. Sehgal, M. Redington, P. L. Kilian, R. M. Chanock, G. A. Prince, A mouse model for investigating the molecular pathogenesis of adenovirus pneumonia. Proceedings of the National Academy of Sciences of the United States of America. ,vol. 88, pp. 1651- 1655 ,(1991) , 10.1073/PNAS.88.5.1651
Larry G. Johnson, John C. Olsen, Balazs Sarkadi, Karen L. Moore, Ronald Swanstrom, Richard C. Boucher, Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis Nature Genetics. ,vol. 2, pp. 21- 25 ,(1992) , 10.1038/NG0992-21
Michael J. Welsh, Matthew P. Anderson, Devra P. Rich, Herbert A. Berger, Gerene M. Denning, Lynda S. Ostedgaard, David N. Sheppard, Seng H. Cheng, Richard J. Gregory, Alan E. Smith, Cystic fibrosis transmembrane conductance regulator: A chloride channel with novel regulation Neuron. ,vol. 8, pp. 821- 829 ,(1992) , 10.1016/0896-6273(92)90196-K
Mitchell L. Drumm, Heidi A. Pope, William H. Cliff, Johanna M. Rommens, Sheila A. Marvin, Lap-Chee Tsui, Francis S. Collins, Raymond A. Frizzell, James M. Wilson, Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell. ,vol. 62, pp. 1227- 1233 ,(1990) , 10.1016/0092-8674(90)90398-X
Ewald R. Weibel, Morphometry of the Human Lung ,(2013)
B. C. Trapnell, C. S. Chu, P. K. Paakko, T. C. Banks, K. Yoshimura, V. J. Ferrans, M. S. Chernick, R. G. Crystal, Expression of the cystic fibrosis transmembrane conductance regulator gene in the respiratory tract of normal individuals and individuals with cystic fibrosis Proceedings of the National Academy of Sciences of the United States of America. ,vol. 88, pp. 6565- 6569 ,(1991) , 10.1073/PNAS.88.15.6565