Skip to main content
Archives of Disease in Childhood logoLink to Archives of Disease in Childhood
. 1993 Apr;68(4):437–440. doi: 10.1136/adc.68.4.437

Gene therapy for cystic fibrosis.

C Coutelle 1, N Caplen 1, S Hart 1, C Huxley 1, R Williamson 1
PMCID: PMC1029257  PMID: 8503661

Full text

PDF
437

Selected References

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

  1. Barasch J., Kiss B., Prince A., Saiman L., Gruenert D., al-Awqati Q. Defective acidification of intracellular organelles in cystic fibrosis. Nature. 1991 Jul 4;352(6330):70–73. doi: 10.1038/352070a0. [DOI] [PubMed] [Google Scholar]
  2. Bear C. E., Li C. H., Kartner N., Bridges R. J., Jensen T. J., Ramjeesingh M., Riordan J. R. Purification and functional reconstitution of the cystic fibrosis transmembrane conductance regulator (CFTR). Cell. 1992 Feb 21;68(4):809–818. doi: 10.1016/0092-8674(92)90155-6. [DOI] [PubMed] [Google Scholar]
  3. Boucher R. C., Stutts M. J., Knowles M. R., Cantley L., Gatzy J. T. Na+ transport in cystic fibrosis respiratory epithelia. Abnormal basal rate and response to adenylate cyclase activation. J Clin Invest. 1986 Nov;78(5):1245–1252. doi: 10.1172/JCI112708. [DOI] [PMC free article] [PubMed] [Google Scholar]
  4. Colledge W. H., Ratcliff R., Foster D., Williamson R., Evans M. J. Cystic fibrosis mouse with intestinal obstruction. Lancet. 1992 Sep 12;340(8820):680–680. doi: 10.1016/0140-6736(92)92223-3. [DOI] [PubMed] [Google Scholar]
  5. Cotten M., Wagner E., Zatloukal K., Phillips S., Curiel D. T., Birnstiel M. L. High-efficiency receptor-mediated delivery of small and large (48 kilobase gene constructs using the endosome-disruption activity of defective or chemically inactivated adenovirus particles. Proc Natl Acad Sci U S A. 1992 Jul 1;89(13):6094–6098. doi: 10.1073/pnas.89.13.6094. [DOI] [PMC free article] [PubMed] [Google Scholar]
  6. Culver K. W., Anderson W. F., Blaese R. M. Lymphocyte gene therapy. Hum Gene Ther. 1991 Summer;2(2):107–109. doi: 10.1089/hum.1991.2.2-107. [DOI] [PubMed] [Google Scholar]
  7. Curiel D. T., Wagner E., Cotten M., Birnstiel M. L., Agarwal S., Li C. M., Loechel S., Hu P. C. High-efficiency gene transfer mediated by adenovirus coupled to DNA-polylysine complexes. Hum Gene Ther. 1992 Apr;3(2):147–154. doi: 10.1089/hum.1992.3.2-147. [DOI] [PubMed] [Google Scholar]
  8. Dorin J. R., Dickinson P., Alton E. W., Smith S. N., Geddes D. M., Stevenson B. J., Kimber W. L., Fleming S., Clarke A. R., Hooper M. L. Cystic fibrosis in the mouse by targeted insertional mutagenesis. Nature. 1992 Sep 17;359(6392):211–215. doi: 10.1038/359211a0. [DOI] [PubMed] [Google Scholar]
  9. Drumm M. L., Pope H. A., Cliff W. H., Rommens J. M., Marvin S. A., Tsui L. C., Collins F. S., Frizzell R. A., Wilson J. M. Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell. 1990 Sep 21;62(6):1227–1233. doi: 10.1016/0092-8674(90)90398-x. [DOI] [PubMed] [Google Scholar]
  10. Gregory R. J., Cheng S. H., Rich D. P., Marshall J., Paul S., Hehir K., Ostedgaard L., Klinger K. W., Welsh M. J., Smith A. E. Expression and characterization of the cystic fibrosis transmembrane conductance regulator. Nature. 1990 Sep 27;347(6291):382–386. doi: 10.1038/347382a0. [DOI] [PubMed] [Google Scholar]
  11. Handyside A. H., Pattinson J. K., Penketh R. J., Delhanty J. D., Winston R. M., Tuddenham E. G. Biopsy of human preimplantation embryos and sexing by DNA amplification. Lancet. 1989 Feb 18;1(8634):347–349. doi: 10.1016/s0140-6736(89)91723-6. [DOI] [PubMed] [Google Scholar]
  12. Immunotherapy of malignancy by in vivo gene transfer into tumors. Hum Gene Ther. 1992 Aug;3(4):399–410. doi: 10.1089/hum.1992.3.4-399. [DOI] [PubMed] [Google Scholar]
  13. 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]
  14. 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]
  15. Miller A. D. Human gene therapy comes of age. Nature. 1992 Jun 11;357(6378):455–460. doi: 10.1038/357455a0. [DOI] [PubMed] [Google Scholar]
  16. Quinton P. M., Reddy M. M. Regulation of absorption in the human sweat duct. Adv Exp Med Biol. 1991;290:159–172. doi: 10.1007/978-1-4684-5934-0_17. [DOI] [PubMed] [Google Scholar]
  17. Rich D. P., Anderson M. P., Gregory R. J., Cheng S. H., Paul S., Jefferson D. M., McCann J. D., Klinger K. W., Smith A. E., Welsh M. J. Expression of cystic fibrosis transmembrane conductance regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells. Nature. 1990 Sep 27;347(6291):358–363. doi: 10.1038/347358a0. [DOI] [PubMed] [Google Scholar]
  18. Riordan J. R., Rommens J. M., Kerem B., Alon N., Rozmahel R., Grzelczak Z., Zielenski J., Lok S., Plavsic N., Chou J. L. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989 Sep 8;245(4922):1066–1073. doi: 10.1126/science.2475911. [DOI] [PubMed] [Google Scholar]
  19. Rommens J. M., Iannuzzi M. C., Kerem B., Drumm M. L., Melmer G., Dean M., Rozmahel R., Cole J. L., Kennedy D., Hidaka N. Identification of the cystic fibrosis gene: chromosome walking and jumping. Science. 1989 Sep 8;245(4922):1059–1065. doi: 10.1126/science.2772657. [DOI] [PubMed] [Google Scholar]
  20. Rosenfeld M. A., Siegfried W., Yoshimura K., Yoneyama K., Fukayama M., Stier L. E., Päkkö P. K., Gilardi P., Stratford-Perricaudet L. D., Perricaudet M. Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo. Science. 1991 Apr 19;252(5004):431–434. doi: 10.1126/science.2017680. [DOI] [PubMed] [Google Scholar]
  21. Rosenfeld M. A., Yoshimura K., Trapnell B. C., Yoneyama K., Rosenthal E. R., Dalemans W., Fukayama M., Bargon J., Stier L. E., Stratford-Perricaudet L. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell. 1992 Jan 10;68(1):143–155. doi: 10.1016/0092-8674(92)90213-v. [DOI] [PubMed] [Google Scholar]
  22. Snouwaert J. N., Brigman K. K., Latour A. M., Malouf N. N., Boucher R. C., Smithies O., Koller B. H. An animal model for cystic fibrosis made by gene targeting. Science. 1992 Aug 21;257(5073):1083–1088. doi: 10.1126/science.257.5073.1083. [DOI] [PubMed] [Google Scholar]
  23. Stewart M. J., Plautz G. E., Del Buono L., Yang Z. Y., Xu L., Gao X., Huang L., Nabel E. G., Nabel G. J. Gene transfer in vivo with DNA-liposome complexes: safety and acute toxicity in mice. Hum Gene Ther. 1992 Jun;3(3):267–275. doi: 10.1089/hum.1992.3.3-267. [DOI] [PubMed] [Google Scholar]
  24. Strauss W. M., Jaenisch R. Molecular complementation of a collagen mutation in mammalian cells using yeast artificial chromosomes. EMBO J. 1992 Feb;11(2):417–422. doi: 10.1002/j.1460-2075.1992.tb05070.x. [DOI] [PMC free article] [PubMed] [Google Scholar]
  25. Trapnell B. C., Chu C. S., Paakko P. K., Banks T. C., Yoshimura K., Ferrans V. J., Chernick M. S., Crystal R. G. Expression of the cystic fibrosis transmembrane conductance regulator gene in the respiratory tract of normal individuals and individuals with cystic fibrosis. Proc Natl Acad Sci U S A. 1991 Aug 1;88(15):6565–6569. doi: 10.1073/pnas.88.15.6565. [DOI] [PMC free article] [PubMed] [Google Scholar]
  26. Wagner E., Zatloukal K., Cotten M., Kirlappos H., Mechtler K., Curiel D. T., Birnstiel M. L. Coupling of adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes. Proc Natl Acad Sci U S A. 1992 Jul 1;89(13):6099–6103. doi: 10.1073/pnas.89.13.6099. [DOI] [PMC free article] [PubMed] [Google Scholar]
  27. Wang C. Y., Huang L. pH-sensitive immunoliposomes mediate target-cell-specific delivery and controlled expression of a foreign gene in mouse. Proc Natl Acad Sci U S A. 1987 Nov;84(22):7851–7855. doi: 10.1073/pnas.84.22.7851. [DOI] [PMC free article] [PubMed] [Google Scholar]
  28. Whitsett J. A., Dey C. R., Stripp B. R., Wikenheiser K. A., Clark J. C., Wert S. E., Gregory R. J., Smith A. E., Cohn J. A., Wilson J. M. Human cystic fibrosis transmembrane conductance regulator directed to respiratory epithelial cells of transgenic mice. Nat Genet. 1992 Sep;2(1):13–20. doi: 10.1038/ng0992-13. [DOI] [PubMed] [Google Scholar]
  29. Yoshimura K., Rosenfeld M. A., Nakamura H., Scherer E. M., Pavirani A., Lecocq J. P., Crystal R. G. Expression of the human cystic fibrosis transmembrane conductance regulator gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer. Nucleic Acids Res. 1992 Jun 25;20(12):3233–3240. doi: 10.1093/nar/20.12.3233. [DOI] [PMC free article] [PubMed] [Google Scholar]

Articles from Archives of Disease in Childhood are provided here courtesy of BMJ Publishing Group

RESOURCES