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. 1999 Mar;80(3):286–289. doi: 10.1136/adc.80.3.286

Prospects for gene therapy in cystic fibrosis

A Jaffe 1, A Bush 1, D Geddes 1, E Alton 1
PMCID: PMC1717847  PMID: 10325715

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Selected References

These references are in PubMed. This may not be the complete list of references from this article.

  1. Alton E. W., Currie D., Logan-Sinclair R., Warner J. O., Hodson M. E., Geddes D. M. Nasal potential difference: a clinical diagnostic test for cystic fibrosis. Eur Respir J. 1990 Sep;3(8):922–926. [PubMed] [Google Scholar]
  2. Alton E. W., Geddes D. M., Gill D. R., Higgins C. F., Hyde S. C., Innes J. A., Porteous D. J. Towards gene therapy for cystic fibrosis: a clinical progress report. Gene Ther. 1998 Mar;5(3):291–292. doi: 10.1038/sj.gt.3300655. [DOI] [PubMed] [Google Scholar]
  3. Coutelle C., Douar A. M., Colledge W. H., Froster U. The challenge of fetal gene therapy. Nat Med. 1995 Sep;1(9):864–866. doi: 10.1038/nm0995-864. [DOI] [PubMed] [Google Scholar]
  4. Crystal R. G., Jaffe A., Brody S., Mastrangeli A., McElvaney N. G., Rosenfeld M., Chu C. S., Danel C., Hay J., Eissa T. A phase 1 study, in cystic fibrosis patients, of the safety, toxicity, and biological efficacy of a single administration of a replication deficient, recombinant adenovirus carrying the cDNA of the normal cystic fibrosis transmembrane conductance regulator gene in the lung. Hum Gene Ther. 1995 May;6(5):643–666. doi: 10.1089/hum.1995.6.5-643. [DOI] [PubMed] [Google Scholar]
  5. Davies J. C., Stern M., Dewar A., Caplen N. J., Munkonge F. M., Pitt T., Sorgi F., Huang L., Bush A., Geddes D. M. CFTR gene transfer reduces the binding of Pseudomonas aeruginosa to cystic fibrosis respiratory epithelium. Am J Respir Cell Mol Biol. 1997 Jun;16(6):657–663. doi: 10.1165/ajrcmb.16.6.9191467. [DOI] [PubMed] [Google Scholar]
  6. Douar A. M., Adebakin S., Themis M., Pavirani A., Cook T., Coutelle C. Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus. Gene Ther. 1997 Sep;4(9):883–890. doi: 10.1038/sj.gt.3300498. [DOI] [PubMed] [Google Scholar]
  7. Goldman M. J., Anderson G. M., Stolzenberg E. D., Kari U. P., Zasloff M., Wilson J. M. Human beta-defensin-1 is a salt-sensitive antibiotic in lung that is inactivated in cystic fibrosis. Cell. 1997 Feb 21;88(4):553–560. doi: 10.1016/s0092-8674(00)81895-4. [DOI] [PubMed] [Google Scholar]
  8. Huard J., Lochmüller H., Acsadi G., Jani A., Holland P., Guérin C., Massie B., Karpati G. Differential short-term transduction efficiency of adult versus newborn mouse tissues by adenoviral recombinants. Exp Mol Pathol. 1995 Apr;62(2):131–143. doi: 10.1006/exmp.1995.1015. [DOI] [PubMed] [Google Scholar]
  9. Jeffery P. K., Gaillard D., Moret S. Human airway secretory cells during development and in mature airway epithelium. Eur Respir J. 1992 Jan;5(1):93–104. [PubMed] [Google Scholar]
  10. Johnson L. G., Olsen J. C., Sarkadi B., Moore K. L., Swanstrom R., Boucher R. C. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet. 1992 Sep;2(1):21–25. doi: 10.1038/ng0992-21. [DOI] [PubMed] [Google Scholar]
  11. Kass-Eisler A., Falck-Pedersen E., Elfenbein D. H., Alvira M., Buttrick P. M., Leinwand L. A. The impact of developmental stage, route of administration and the immune system on adenovirus-mediated gene transfer. Gene Ther. 1994 Nov;1(6):395–402. [PubMed] [Google Scholar]
  12. Kerem B., Rommens J. M., Buchanan J. A., Markiewicz D., Cox T. K., Chakravarti A., Buchwald M., Tsui L. C. Identification of the cystic fibrosis gene: genetic analysis. Science. 1989 Sep 8;245(4922):1073–1080. doi: 10.1126/science.2570460. [DOI] [PubMed] [Google Scholar]
  13. Kohn D. B., Parkman R. Gene therapy for newborns. FASEB J. 1997 Jul;11(8):635–639. doi: 10.1096/fasebj.11.8.9240965. [DOI] [PubMed] [Google Scholar]
  14. Larson J. E., Morrow S. L., Happel L., Sharp J. F., Cohen J. C. Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet. 1997 Mar 1;349(9052):619–620. doi: 10.1016/S0140-6736(05)61567-X. [DOI] [PubMed] [Google Scholar]
  15. Mahadeva R., Webb K., Westerbeek R. C., Carroll N. R., Dodd M. E., Bilton D., Lomas D. A. Clinical outcome in relation to care in centres specialising in cystic fibrosis: cross sectional study. BMJ. 1998 Jun 13;316(7147):1771–1775. doi: 10.1136/bmj.316.7147.1771. [DOI] [PMC free article] [PubMed] [Google Scholar]
  16. Middleton P. G., Geddes D. M., Alton E. W. Protocols for in vivo measurement of the ion transport defects in cystic fibrosis nasal epithelium. Eur Respir J. 1994 Nov;7(11):2050–2056. [PubMed] [Google Scholar]
  17. O'Brodovich H. Epithelial ion transport in the fetal and perinatal lung. Am J Physiol. 1991 Oct;261(4 Pt 1):C555–C564. doi: 10.1152/ajpcell.1991.261.4.C555. [DOI] [PubMed] [Google Scholar]
  18. Pier G. B., Grout M., Zaidi T. S. Cystic fibrosis transmembrane conductance regulator is an epithelial cell receptor for clearance of Pseudomonas aeruginosa from the lung. Proc Natl Acad Sci U S A. 1997 Oct 28;94(22):12088–12093. doi: 10.1073/pnas.94.22.12088. [DOI] [PMC free article] [PubMed] [Google Scholar]
  19. Rubenstein R. C., McVeigh U., Flotte T. R., Guggino W. B., Zeitlin P. L. CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vector. Gene Ther. 1997 May;4(5):384–392. doi: 10.1038/sj.gt.3300417. [DOI] [PubMed] [Google Scholar]
  20. Sekhon H. S., Larson J. E. In utero gene transfer into the pulmonary epithelium. Nat Med. 1995 Nov;1(11):1201–1203. doi: 10.1038/nm1195-1201. [DOI] [PubMed] [Google Scholar]
  21. Stern M., Munkonge F. M., Caplen N. J., Sorgi F., Huang L., Geddes D. M., Alton E. W. Quantitative fluorescence measurements of chloride secretion in native airway epithelium from CF and non-CF subjects. Gene Ther. 1995 Dec;2(10):766–774. [PubMed] [Google Scholar]
  22. Tizzano E. F., O'Brodovich H., Chitayat D., Bènichou J. C., Buchwald M. Regional expression of CFTR in developing human respiratory tissues. Am J Respir Cell Mol Biol. 1994 Apr;10(4):355–362. doi: 10.1165/ajrcmb.10.4.7510983. [DOI] [PubMed] [Google Scholar]
  23. Wagner J. A., Reynolds T., Moran M. L., Moss R. B., Wine J. J., Flotte T. R., Gardner P. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet. 1998 Jun 6;351(9117):1702–1703. doi: 10.1016/S0140-6736(05)77740-0. [DOI] [PubMed] [Google Scholar]
  24. Zepeda M., Wilson J. M. Neonatal cotton rats do not exhibit destructive immune responses to adenoviral vectors. Gene Ther. 1996 Nov;3(11):973–979. [PubMed] [Google Scholar]

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