Skip to main content
NCBI home page
Journal of the Royal Society of Medicine logoLink to Journal of the Royal Society of Medicine
. 2002;95(Suppl 41):58–67.

New therapeutic approaches for cystic fibrosis lung disease.

Jane C Davies 1
PMCID: PMC1308638  PMID: 12216276

Full Text

The Full Text of this article is available as a PDF (188.3 KB).

Selected References

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

  1. Abman S. H., Ogle J. W., Harbeck R. J., Butler-Simon N., Hammond K. B., Accurso F. J. Early bacteriologic, immunologic, and clinical courses of young infants with cystic fibrosis identified by neonatal screening. J Pediatr. 1991 Aug;119(2):211–217. doi: 10.1016/s0022-3476(05)80729-2. [DOI] [PubMed] [Google Scholar]
  2. Alison M. R., Poulsom R., Jeffery R., Dhillon A. P., Quaglia A., Jacob J., Novelli M., Prentice G., Williamson J., Wright N. A. Hepatocytes from non-hepatic adult stem cells. Nature. 2000 Jul 20;406(6793):257–257. doi: 10.1038/35018642. [DOI] [PubMed] [Google Scholar]
  3. Alton E. W., Stern M., Farley R., Jaffe A., Chadwick S. L., Phillips J., Davies J., Smith S. N., Browning J., Davies M. G. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet. 1999 Mar 20;353(9157):947–954. doi: 10.1016/s0140-6736(98)06532-5. [DOI] [PubMed] [Google Scholar]
  4. Arispe N., Ma J., Jacobson K. A., Pollard H. B. Direct activation of cystic fibrosis transmembrane conductance regulator channels by 8-cyclopentyl-1,3-dipropylxanthine (CPX) and 1,3-diallyl-8-cyclohexylxanthine (DAX). J Biol Chem. 1998 Mar 6;273(10):5727–5734. doi: 10.1074/jbc.273.10.5727. [DOI] [PMC free article] [PubMed] [Google Scholar]
  5. Armstrong D. S., Grimwood K., Carzino R., Carlin J. B., Olinsky A., Phelan P. D. Lower respiratory infection and inflammation in infants with newly diagnosed cystic fibrosis. BMJ. 1995 Jun 17;310(6994):1571–1572. doi: 10.1136/bmj.310.6994.1571. [DOI] [PMC free article] [PubMed] [Google Scholar]
  6. Bennett W. D., Olivier K. N., Zeman K. L., Hohneker K. W., Boucher R. C., Knowles M. R. Effect of uridine 5'-triphosphate plus amiloride on mucociliary clearance in adult cystic fibrosis. Am J Respir Crit Care Med. 1996 Jun;153(6 Pt 1):1796–1801. doi: 10.1164/ajrccm.153.6.8665037. [DOI] [PubMed] [Google Scholar]
  7. Birrer P., McElvaney N. G., Rüdeberg A., Sommer C. W., Liechti-Gallati S., Kraemer R., Hubbard R., Crystal R. G. Protease-antiprotease imbalance in the lungs of children with cystic fibrosis. Am J Respir Crit Care Med. 1994 Jul;150(1):207–213. doi: 10.1164/ajrccm.150.1.7912987. [DOI] [PubMed] [Google Scholar]
  8. Birrer P. Proteases and antiproteases in cystic fibrosis: pathogenetic considerations and therapeutic strategies. Respiration. 1995;62 (Suppl 1):25–28. doi: 10.1159/000196490. [DOI] [PubMed] [Google Scholar]
  9. Brown C. R., Hong-Brown L. Q., Biwersi J., Verkman A. S., Welch W. J. Chemical chaperones correct the mutant phenotype of the delta F508 cystic fibrosis transmembrane conductance regulator protein. Cell Stress Chaperones. 1996 Jun;1(2):117–125. doi: 10.1379/1466-1268(1996)001<0117:ccctmp>2.3.co;2. [DOI] [PMC free article] [PubMed] [Google Scholar]
  10. Bryan R., Feldman M., Jawetz S. C., Rajan S., DiMango E., Tang H. B., Scheffler L., Speert D. P., Prince A. The effects of aerosolized dextran in a mouse model of Pseudomonas aeruginosa pulmonary infection. J Infect Dis. 1999 Jun;179(6):1449–1458. doi: 10.1086/314755. [DOI] [PubMed] [Google Scholar]
  11. Chadwick S. L., Kingston H. D., Stern M., Cook R. M., O'Connor B. J., Lukasson M., Balfour R. P., Rosenberg M., Cheng S. H., Smith A. E. Safety of a single aerosol administration of escalating doses of the cationic lipid GL-67/DOPE/DMPE-PEG5000 formulation to the lungs of normal volunteers. Gene Ther. 1997 Sep;4(9):937–942. doi: 10.1038/sj.gt.3300481. [DOI] [PubMed] [Google Scholar]
  12. Cheng P. W., Boat T. F., Cranfill K., Yankaskas J. R., Boucher R. C. Increased sulfation of glycoconjugates by cultured nasal epithelial cells from patients with cystic fibrosis. J Clin Invest. 1989 Jul;84(1):68–72. doi: 10.1172/JCI114171. [DOI] [PMC free article] [PubMed] [Google Scholar]
  13. Choo-Kang L. R., Zeitlin P. L. Type I, II, III, IV, and V cystic fibrosis transmembrane conductance regulator defects and opportunities for therapy. Curr Opin Pulm Med. 2000 Nov;6(6):521–529. doi: 10.1097/00063198-200011000-00011. [DOI] [PubMed] [Google Scholar]
  14. Clancy J. P., Bebök Z., Ruiz F., King C., Jones J., Walker L., Greer H., Hong J., Wing L., Macaluso M. Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis. Am J Respir Crit Care Med. 2001 Jun;163(7):1683–1692. doi: 10.1164/ajrccm.163.7.2004001. [DOI] [PubMed] [Google Scholar]
  15. Crystal R. G., McElvaney N. G., Rosenfeld M. A., Chu C. S., Mastrangeli A., Hay J. G., Brody S. L., Jaffe H. A., Eissa N. T., Danel C. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Genet. 1994 Sep;8(1):42–51. doi: 10.1038/ng0994-42. [DOI] [PubMed] [Google Scholar]
  16. Davies J. C., Geddes D. M., Alton E. W. Gene therapy for cystic fibrosis. J Gene Med. 2001 Sep-Oct;3(5):409–417. doi: 10.1002/jgm.200. [DOI] [PubMed] [Google Scholar]
  17. 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]
  18. DiMango E., Ratner A. J., Bryan R., Tabibi S., Prince A. Activation of NF-kappaB by adherent Pseudomonas aeruginosa in normal and cystic fibrosis respiratory epithelial cells. J Clin Invest. 1998 Jun 1;101(11):2598–2605. doi: 10.1172/JCI2865. [DOI] [PMC free article] [PubMed] [Google Scholar]
  19. Dong Y. H., Wang L. H., Xu J. L., Zhang H. B., Zhang X. F., Zhang L. H. Quenching quorum-sensing-dependent bacterial infection by an N-acyl homoserine lactonase. Nature. 2001 Jun 14;411(6839):813–817. doi: 10.1038/35081101. [DOI] [PubMed] [Google Scholar]
  20. Dorin J. R., Farley R., Webb S., Smith S. N., Farini E., Delaney S. J., Wainwright B. J., Alton E. W., Porteous D. J. A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Ther. 1996 Sep;3(9):797–801. [PubMed] [Google Scholar]
  21. Dosanjh A., Lencer W., Brown D., Ausiello D. A., Stow J. L. Heterologous expression of delta F508 CFTR results in decreased sialylation of membrane glycoconjugates. Am J Physiol. 1994 Feb;266(2 Pt 1):C360–C366. doi: 10.1152/ajpcell.1994.266.2.C360. [DOI] [PubMed] [Google Scholar]
  22. Drapkin P. T., O'Riordan C. R., Yi S. M., Chiorini J. A., Cardella J., Zabner J., Welsh M. J. Targeting the urokinase plasminogen activator receptor enhances gene transfer to human airway epithelia. J Clin Invest. 2000 Mar;105(5):589–596. doi: 10.1172/JCI8858. [DOI] [PMC free article] [PubMed] [Google Scholar]
  23. Eigen H., Rosenstein B. J., FitzSimmons S., Schidlow D. V. A multicenter study of alternate-day prednisone therapy in patients with cystic fibrosis. Cystic Fibrosis Foundation Prednisone Trial Group. J Pediatr. 1995 Apr;126(4):515–523. doi: 10.1016/s0022-3476(95)70343-8. [DOI] [PubMed] [Google Scholar]
  24. Elborn J. S., Shale D. J., Britton J. R. Cystic fibrosis: current survival and population estimates to the year 2000. Thorax. 1991 Dec;46(12):881–885. doi: 10.1136/thx.46.12.881. [DOI] [PMC free article] [PubMed] [Google Scholar]
  25. Engelhardt J. F., Yankaskas J. R., Ernst S. A., Yang Y., Marino C. R., Boucher R. C., Cohn J. A., Wilson J. M. Submucosal glands are the predominant site of CFTR expression in the human bronchus. Nat Genet. 1992 Nov;2(3):240–248. doi: 10.1038/ng1192-240. [DOI] [PubMed] [Google Scholar]
  26. Gan K. H., Veeze H. J., van den Ouweland A. M., Halley D. J., Scheffer H., van der Hout A., Overbeek S. E., de Jongste J. C., Bakker W., Heijerman H. G. A cystic fibrosis mutation associated with mild lung disease. N Engl J Med. 1995 Jul 13;333(2):95–99. doi: 10.1056/NEJM199507133330204. [DOI] [PubMed] [Google Scholar]
  27. Graham A., Hasani A., Alton E. W., Martin G. P., Marriott C., Hodson M. E., Clarke S. W., Geddes D. M. No added benefit from nebulized amiloride in patients with cystic fibrosis. Eur Respir J. 1993 Oct;6(9):1243–1248. [PubMed] [Google Scholar]
  28. Gussoni E., Soneoka Y., Strickland C. D., Buzney E. A., Khan M. K., Flint A. F., Kunkel L. M., Mulligan R. C. Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature. 1999 Sep 23;401(6751):390–394. doi: 10.1038/43919. [DOI] [PubMed] [Google Scholar]
  29. Harvey B. G., Leopold P. L., Hackett N. R., Grasso T. M., Williams P. M., Tucker A. L., Kaner R. J., Ferris B., Gonda I., Sweeney T. D. Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus. J Clin Invest. 1999 Nov;104(9):1245–1255. doi: 10.1172/JCI7935. [DOI] [PMC free article] [PubMed] [Google Scholar]
  30. Illek B., Zhang L., Lewis N. C., Moss R. B., Dong J. Y., Fischer H. Defective function of the cystic fibrosis-causing missense mutation G551D is recovered by genistein. Am J Physiol. 1999 Oct;277(4 Pt 1):C833–C839. doi: 10.1152/ajpcell.1999.277.4.C833. [DOI] [PubMed] [Google Scholar]
  31. Jaffé Adam, Rosenthal Mark. Macrolides in the respiratory tract in cystic fibrosis. J R Soc Med. 2002;95 (Suppl 41):27–31. [PMC free article] [PubMed] [Google Scholar]
  32. Johnson L. G., Boyles S. E., Wilson J., Boucher R. C. Normalization of raised sodium absorption and raised calcium-mediated chloride secretion by adenovirus-mediated expression of cystic fibrosis transmembrane conductance regulator in primary human cystic fibrosis airway epithelial cells. J Clin Invest. 1995 Mar;95(3):1377–1382. doi: 10.1172/JCI117789. [DOI] [PMC free article] [PubMed] [Google Scholar]
  33. 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]
  34. Khan T. Z., Wagener J. S., Bost T., Martinez J., Accurso F. J., Riches D. W. Early pulmonary inflammation in infants with cystic fibrosis. Am J Respir Crit Care Med. 1995 Apr;151(4):1075–1082. doi: 10.1164/ajrccm/151.4.1075. [DOI] [PubMed] [Google Scholar]
  35. Kitson C., Angel B., Judd D., Rothery S., Severs N. J., Dewar A., Huang L., Wadsworth S. C., Cheng S. H., Geddes D. M. The extra- and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epithelium. Gene Ther. 1999 Apr;6(4):534–546. doi: 10.1038/sj.gt.3300840. [DOI] [PubMed] [Google Scholar]
  36. Knowles M. R., Church N. L., Waltner W. E., Yankaskas J. R., Gilligan P., King M., Edwards L. J., Helms R. W., Boucher R. C. Aerosolized amiloride as treatment of cystic fibrosis lung disease: a pilot study. Adv Exp Med Biol. 1991;290:119–132. doi: 10.1007/978-1-4684-5934-0_14. [DOI] [PubMed] [Google Scholar]
  37. Knowles M. R., Robinson J. M., Wood R. E., Pue C. A., Mentz W. M., Wager G. C., Gatzy J. T., Boucher R. C. Ion composition of airway surface liquid of patients with cystic fibrosis as compared with normal and disease-control subjects. J Clin Invest. 1997 Nov 15;100(10):2588–2595. doi: 10.1172/JCI119802. [DOI] [PMC free article] [PubMed] [Google Scholar]
  38. Knowles M., Gatzy J., Boucher R. Increased bioelectric potential difference across respiratory epithelia in cystic fibrosis. N Engl J Med. 1981 Dec 17;305(25):1489–1495. doi: 10.1056/NEJM198112173052502. [DOI] [PubMed] [Google Scholar]
  39. Koch C., Høiby N. Pathogenesis of cystic fibrosis. Lancet. 1993 Apr 24;341(8852):1065–1069. doi: 10.1016/0140-6736(93)92422-p. [DOI] [PubMed] [Google Scholar]
  40. Konstan M. W., Byard P. J., Hoppel C. L., Davis P. B. Effect of high-dose ibuprofen in patients with cystic fibrosis. N Engl J Med. 1995 Mar 30;332(13):848–854. doi: 10.1056/NEJM199503303321303. [DOI] [PubMed] [Google Scholar]
  41. Krause D. S., Theise N. D., Collector M. I., Henegariu O., Hwang S., Gardner R., Neutzel S., Sharkis S. J. Multi-organ, multi-lineage engraftment by a single bone marrow-derived stem cell. Cell. 2001 May 4;105(3):369–377. doi: 10.1016/s0092-8674(01)00328-2. [DOI] [PubMed] [Google Scholar]
  42. Lang A. B., Schaad U. B., Rüdeberg A., Wedgwood J., Que J. U., Fürer E., Cryz S. J., Jr Effect of high-affinity anti-Pseudomonas aeruginosa lipopolysaccharide antibodies induced by immunization on the rate of Pseudomonas aeruginosa infection in patients with cystic fibrosis. J Pediatr. 1995 Nov;127(5):711–717. doi: 10.1016/s0022-3476(95)70158-3. [DOI] [PubMed] [Google Scholar]
  43. Ledson M., Gallagher M., Hart C. A., Walshaw M. Nebulized heparin in Burkholderia cepacia colonized adult cystic fibrosis patients. Eur Respir J. 2001 Jan;17(1):36–38. doi: 10.1183/09031936.01.17100360. [DOI] [PubMed] [Google Scholar]
  44. Matsui H., Grubb B. R., Tarran R., Randell S. H., Gatzy J. T., Davis C. W., Boucher R. C. Evidence for periciliary liquid layer depletion, not abnormal ion composition, in the pathogenesis of cystic fibrosis airways disease. Cell. 1998 Dec 23;95(7):1005–1015. doi: 10.1016/s0092-8674(00)81724-9. [DOI] [PubMed] [Google Scholar]
  45. Matthews W. J., Jr, Williams M., Oliphint B., Geha R., Colten H. R. Hypogammaglobulinemia in patients with cystic fibrosis. N Engl J Med. 1980 Jan 31;302(5):245–249. doi: 10.1056/NEJM198001313020501. [DOI] [PubMed] [Google Scholar]
  46. 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]
  47. Ono K., Takii T., Onozaki K., Ikawa M., Okabe M., Sawada M. Migration of exogenous immature hematopoietic cells into adult mouse brain parenchyma under GFP-expressing bone marrow chimera. Biochem Biophys Res Commun. 1999 Sep 7;262(3):610–614. doi: 10.1006/bbrc.1999.1223. [DOI] [PubMed] [Google Scholar]
  48. Oppenheimer E. H., Esterly J. R. Pathology of cystic fibrosis review of the literature and comparison with 146 autopsied cases. Perspect Pediatr Pathol. 1975;2:241–278. [PubMed] [Google Scholar]
  49. Parsons D. W., Grubb B. R., Johnson L. G., Boucher R. C. Enhanced in vivo airway gene transfer via transient modification of host barrier properties with a surface-active agent. Hum Gene Ther. 1998 Dec 10;9(18):2661–2672. doi: 10.1089/hum.1998.9.18-2661. [DOI] [PubMed] [Google Scholar]
  50. Petersen B. E., Bowen W. C., Patrene K. D., Mars W. M., Sullivan A. K., Murase N., Boggs S. S., Greenberger J. S., Goff J. P. Bone marrow as a potential source of hepatic oval cells. Science. 1999 May 14;284(5417):1168–1170. doi: 10.1126/science.284.5417.1168. [DOI] [PubMed] [Google Scholar]
  51. Pier G. B., Grout M., Zaidi T. S., Olsen J. C., Johnson L. G., Yankaskas J. R., Goldberg J. B. Role of mutant CFTR in hypersusceptibility of cystic fibrosis patients to lung infections. Science. 1996 Jan 5;271(5245):64–67. doi: 10.1126/science.271.5245.64. [DOI] [PMC free article] [PubMed] [Google Scholar]
  52. 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]
  53. Robinson M., Daviskas E., Eberl S., Baker J., Chan H. K., Anderson S. D., Bye P. T. The effect of inhaled mannitol on bronchial mucus clearance in cystic fibrosis patients: a pilot study. Eur Respir J. 1999 Sep;14(3):678–685. doi: 10.1034/j.1399-3003.1999.14c30.x. [DOI] [PubMed] [Google Scholar]
  54. Robinson M., Eberl S., Tomlinson C., Daviskas E., Regnis J. A., Bailey D. L., Torzillo P. J., Menache M., Bye P. T. Regional mucociliary clearance in patients with cystic fibrosis. J Aerosol Med. 2000 Summer;13(2):73–86. doi: 10.1089/089426800418604. [DOI] [PubMed] [Google Scholar]
  55. 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]
  56. Rubenstein R. C., Egan M. E., Zeitlin P. L. In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR. J Clin Invest. 1997 Nov 15;100(10):2457–2465. doi: 10.1172/JCI119788. [DOI] [PMC free article] [PubMed] [Google Scholar]
  57. Rubenstein R. C., Zeitlin P. L. A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function. Am J Respir Crit Care Med. 1998 Feb;157(2):484–490. doi: 10.1164/ajrccm.157.2.9706088. [DOI] [PubMed] [Google Scholar]
  58. Saiman L., Prince A. Pseudomonas aeruginosa pili bind to asialoGM1 which is increased on the surface of cystic fibrosis epithelial cells. J Clin Invest. 1993 Oct;92(4):1875–1880. doi: 10.1172/JCI116779. [DOI] [PMC free article] [PubMed] [Google Scholar]
  59. Schwartz D. A., Quinn T. J., Thorne P. S., Sayeed S., Yi A. K., Krieg A. M. CpG motifs in bacterial DNA cause inflammation in the lower respiratory tract. J Clin Invest. 1997 Jul 1;100(1):68–73. doi: 10.1172/JCI119523. [DOI] [PMC free article] [PubMed] [Google Scholar]
  60. Scott C. S., Retsch-Bogart G. Z., Henry M. M. Renal failure and vestibular toxicity in an adolescent with cystic fibrosis receiving gentamicin and standard-dose ibuprofen. Pediatr Pulmonol. 2001 Apr;31(4):314–316. doi: 10.1002/ppul.1047. [DOI] [PubMed] [Google Scholar]
  61. Singh P. K., Schaefer A. L., Parsek M. R., Moninger T. O., Welsh M. J., Greenberg E. P. Quorum-sensing signals indicate that cystic fibrosis lungs are infected with bacterial biofilms. Nature. 2000 Oct 12;407(6805):762–764. doi: 10.1038/35037627. [DOI] [PubMed] [Google Scholar]
  62. Smith J. J., Travis S. M., Greenberg E. P., Welsh M. J. Cystic fibrosis airway epithelia fail to kill bacteria because of abnormal airway surface fluid. Cell. 1996 Apr 19;85(2):229–236. doi: 10.1016/s0092-8674(00)81099-5. [DOI] [PubMed] [Google Scholar]
  63. Stern M., Caplen N. J., Browning J. E., Griesenbach U., Sorgi F., Huang L., Gruenert D. C., Marriot C., Crystal R. G., Geddes D. M. The effect of mucolytic agents on gene transfer across a CF sputum barrier in vitro. Gene Ther. 1998 Jan;5(1):91–98. doi: 10.1038/sj.gt.3300556. [DOI] [PubMed] [Google Scholar]
  64. 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]
  65. Tanaka G., Shigeta M., Komatsuzawa H., Sugai M., Suginaka H., Usui T. Effect of clarithromycin on Pseudomonas aeruginosa biofilms. Chemotherapy. 2000 Jan-Feb;46(1):36–42. doi: 10.1159/000007254. [DOI] [PubMed] [Google Scholar]
  66. 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]
  67. Welsh M. J., Smith A. E. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell. 1993 Jul 2;73(7):1251–1254. doi: 10.1016/0092-8674(93)90353-r. [DOI] [PubMed] [Google Scholar]
  68. Wilschanski M., Famini C., Blau H., Rivlin J., Augarten A., Avital A., Kerem B., Kerem E. A pilot study of the effect of gentamicin on nasal potential difference measurements in cystic fibrosis patients carrying stop mutations. Am J Respir Crit Care Med. 2000 Mar;161(3 Pt 1):860–865. doi: 10.1164/ajrccm.161.3.9904116. [DOI] [PubMed] [Google Scholar]
  69. Worgall S., Kikuchi T., Singh R., Martushova K., Lande L., Crystal R. G. Protection against pulmonary infection with Pseudomonas aeruginosa following immunization with P. aeruginosa-pulsed dendritic cells. Infect Immun. 2001 Jul;69(7):4521–4527. doi: 10.1128/IAI.69.7.4521-4527.2001. [DOI] [PMC free article] [PubMed] [Google Scholar]
  70. Zabner J., Ramsey B. W., Meeker D. P., Aitken M. L., Balfour R. P., Gibson R. L., Launspach J., Moscicki R. A., Richards S. M., Standaert T. A. Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosis. J Clin Invest. 1996 Mar 15;97(6):1504–1511. doi: 10.1172/JCI118573. [DOI] [PMC free article] [PubMed] [Google Scholar]

Articles from Journal of the Royal Society of Medicine are provided here courtesy of Royal Society of Medicine Press

RESOURCES