A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis

Gene Therapy

Volumn 4, Issue 3, 1997, Pages 199-209

  Gill, D R ^a,j   Southern, K W ^b   Mofford, K A ^a   Seddon, T ^c   Huang, L ^d   Sorgi, F ^d   Thomson, A ^a   MacVinish, L J ^e   Ratclif, R ^e   Bilton, D ^f   Lane, D J ^g   Littlewood, J M ^b   Webb, A K ^h   Middleton, P G ⁱ   Colledge, W H ^e   Cuthbert, A W ^e   Evans, M J ^e   Higgins, C F ^a   Hyde, S C ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b ST JAMES S UNIVERSITY HOSPITAL   (United Kingdom)

^c IMPERIAL CANCER RESEARCH FUND   (United Kingdom)

^d UNIVERSITY OF PITTSBURGH   (United States)

^e UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^f PAPWORTH HOSPITAL   (United Kingdom)

^g CHURCHILL HOSPITAL   (United Kingdom)

^h WYTHENSHAWE HOSPITAL   (United Kingdom)

ⁱ WESTMEAD HOSPITAL   (Australia)

^j UNIVERSITY OF OXFORD   (United Kingdom)

more..

Author keywords

Airway; Cationic liposome; CFTR; Clinical trial; Gene therapy

Indexed keywords

CHLORIDE ION; COMPLEMENTARY DNA; LIPOSOME; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ARTICLE; BIOPSY; CHLORIDE CURRENT; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CYSTIC FIBROSIS; DOUBLE BLIND PROCEDURE; FEMALE; FLUORESCENCE MICROSCOPY; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HUMAN; HUMAN TISSUE; MALE; NOSE MUCOSA; PLASMID; POTENTIAL DIFFERENCE; PRIORITY JOURNAL;

EID: 8244238394     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300391     Document Type: Article

References (29)

1. Riordan JR et al. Identification of the cystic fibrosis gene: cloning and characterisation of complementary DNA. Science 1989; 245: 1066-1073.
2. Boat TF et al. Cystic fibrosis. In: Beaudet AL et al (eds). The Metabolic Basis of Inherited Disease. Mc Graw-Hill: New York, 1989, pp 2649-2680.
3. Dodge JA et al. Cystic fibrosis in the United Kingdom, 1968-1988: incidence, population and survival. Paediatr Perinat Epidemiol 1993; 7: 157-166.
4. Drumm ML et al. Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell 1990; 62: 1227-1233.
5. Rich DP et al. Expression of the cystic fibrosis transmembrane conductance regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells. Nature 1990; 347: 358-363.
6. Alton EWFW et al. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat Genet 1993; 5: 135-142.
7. Hyde SC et al. Correction of the ion-channel defect in CF transgenic mice by gene therapy. Nature 1993; 362: 250-255.
8. Zabner J et al. Adenovirus-mediated gene transfer transiently corrects the chloride defect in nasal epithelia in patients with CF. Cell 1993; 75: 1-20.
9. Hay JG et al. Modification of nasal potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vector. Hum Gene Ther 1995; 6: 1487-1496.
10. Zabner J et al. Repeat administration of an adenovrius vector encoding CFTR to the nasal epithelium of patients with CF. J Clin Invest 1996; 97: 1504-1511.
11. Knowles MR et al. A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. New Engl J Med 1995; 333: 823-831.
12. Gao X, Huang L. A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem Biophys Res Commun 1991; 179: 280-285.
13. Nabel GJ et al. Direct gene transfer with DNA-liposome complexes in melanoma: expression, biologic activity and lack of toxicity in humans. Proc Natl Acad Sci USA 1993; 90: 11307-11311.
14. Caplen NJ et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med 1995; 1: 39-46.
15. Stern M et al. Quantitative fluorescence measurements of chloride secretion in native airway epithelium from CF and non-CF subjects. Gene Therapy 1995; 2: 766-774.
16. Middleton PG et al. Nasal application of the cationic liposome DC-Chol:DOPE does not alter ion transport lung function or bacterial growth. Eur Resp J 1994; 7: 442-445.
17. Middleton PG, Geddes DM, Alton EWFW. Protocols for in vivo measurement of the ion transport defects in cystic fibrosis epithelium. Eur Resp J 1994; 7: 2050-2056.
18. Logan JJ et al. Cationic lipids for reporter gene and CFTR transfer to rat pulmonary epithelium. Gene Therapy 1995; 2: 38-49.
19. MacVinish LJ et al. Correction of airway epithelial function in vivo by transfection with novel plasmid in cystic fibrosis mice. J Physiol (in press).
20. Crystal RG et al. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with CF. Nat Genet 1994; 3: 42-51.
21. Southern KW. Gene therapy for cystic fibrosis: current issues. Br J Hosp Med 1996; 55: 495-499.
22. Knowles MR, Paradiso AM, Boucher RC. In vivo nasal potential difference: techniques and protocols for assessing efficacy of gene transfer in cystic fibrosis. Hum Gene Ther 1995; 6: 445-455.
23. Knowles MR, Gatzy J, Boucher RC. Increased bioelectric potential difference across respiratory eipthelia in cystic fibrosis. New Engl J Med 1981; 305: 1489-1495.
24. Chinet TC. Use of in vivo nasal transepithelial difference to evaluate efficacy in CF gene therapy phase I trials. Eur Respir J 1994; 7: 1917-1920.
25. Johnson LG et al. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 1992; 2: 21-25.
26. Johnson LG et al. Normalisation 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; 95: 1377-1382.
27. Verkman AS. Development and biological applications of chloride-sensitive fluorescent indicators. Am J Physiol 1990; 259: C375-C388.
28. Sorgi FL, Huang L. Large-scale production of DC-Chol cationic liposomes by microfluidisatlon. Int J Pharm (in press).
29. Shwachman H, Kulczycki LL. Long term study of one hundred and five patients with cystic fibrosis. Am J Dis Child 1958; 96: 6-15.