Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis

Gene Therapy

Volumn 4, Issue 3, 1997, Pages 210-218

  Porteous, D J ^a   Dorin, J R ^a   McLachlan, G ^a   Davidson Smith, H ^a   Davidson, H ^a   Stevenson, B J ^a   Carothers, A D ^a   Wallace, W A H ^b   Moralee, S ^b   Hoenes, C ^c   Kallmeyer, G ^c   Michaelis, U ^c   Naujoks, K ^c   Ho, L P ^b   Samways, J M ^b   Imrie, M ^b   Greening, A P ^b   Innes, J A ^b  

^a WESTERN GENERAL HOSPITAL   (United Kingdom)

^b UNIVERSITY OF EDINBURGH   (United Kingdom)

^c F HOFFMANN LA ROCHE LTD   (Switzerland)

Author keywords

Cationic liposome; Cystic fibrosis; Gene therapy

Indexed keywords

DNA; LIPOSOME; MESSENGER RNA; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ADOLESCENT; ADULT; ANIMAL TISSUE; ARTICLE; BIOPSY; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CYSTIC FIBROSIS; DOUBLE BLIND PROCEDURE; FEMALE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HUMAN; INFLAMMATORY CELL; ION CONDUCTANCE; ION TRANSPORT; MALE; MULTICENTER STUDY; NOSE MUCOSA; POLYMERASE CHAIN REACTION; POTENTIAL DIFFERENCE; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; TRANSGENE;

EID: 8244245761     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300390     Document Type: Article

References (25)

1. Kerem B et al. Identification of the cystic fibrosis gene: genetic analysis. Science 1989; 245: 1073-1080.
2. Collins FS. Cystic fibrosis. molecular biology and therapeutic implications. Science 1992; 256: 774-779.
3. Welsh MJ, Tsui L, Boat TF, Beaudet AL. Cystic fibrosis. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds). The Metabolic and Molecular Bases of Inherited Disease, Vol III, 7th edn. McGraw Hill: New York, 1995, pp 3799-3876.
4. Sheppard DN et al. Mutations in CFTR associated with mild-disease-form CI-channels with altered pore properties. Nature 1993; 362: 160-164.
5. Gan K et al. A cystic fibrosis mutation associated with mild lung disease. New Engl J Med 1995; 333: 96-99.
6. Zabner JL, Couture A, Smith AE, Welsh MJ. Correction of cAMP-stmulated fluid secretion in cystic fibrosis airway epithelia: efficiency of adenovirus-mediated gene transfer in vitro. Hum Gene Ther 1994; 5: 585-593.
7. Rosenfeld MA et al. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 1992; 68: 143-155.
8. Engelhardt J, Litzky L, Wilson J. Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a. Hum Gene Ther 1994; 5: 1217-1229.
9. Engelhardt J et al. Adenovirus-mediated transfer of the CFTR gene to lung of nonhuman primates: biological efficacy study. Hum Gene Ther 1993; 4: 759-769.
10. Bout A et al. Lung gene therapy: in vivo adenovirus-mediated gene transfer to rhesus monkey airway epithelium. Hum Gene Ther 1994; 5: 3-10.
11. Wilson JM. Gene therapy for cystic fibrosis: challenges and future directions. J Clin Invest 1995; 96: 2547-2554.
12. Yei S et al. Adenovirus-mediated gene transfer for cystic fibrosis. quantitative evaluation of repeated in vivo vector administration to the lung. Gene Therapy 1994; 1: 192-200.
13. Grubb BR et al. Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans. Nature 1994; 371: 802-806.
14. Crystal RG et al. Administration of an adenovirus containing human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Genet 1994; 8: 42-51.
15. 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.
16. Hyde SC et al. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature 1993; 362: 250-255.
17. McLachlan G et al. Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMV-CFTR-DOTAP. Gene Therapy 1996; 3: 1113-1123.
18. 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.
19. Caplen NJ et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med 1995; 1: 39-16.
20. McLachlan G et al. Evaluation in vitro and in vivo of cationic liposome-expression construct complexes for cystic fibrosis gene therapy. Gene Therapy 1995; 2: 614-622.
21. Brugal G et al. HOME: highly optimised microscope environment. Cytometry 1992; 13: 109-116.
22. Zabner J et al. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 1993; 75: 207-216.
23. Zabner J et al. 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; 97: 1504-1511.
24. Rayner RJ et al. Inflammatory markers in cystic fibrosis. Respir Med 1991; 85: 139-145.
25. Dean TP et al. IL-8 concentrations are elevated in bronchoalveolar lavage, sputum and sera of children with cystic fibrosis. Paediatr Res 1993; 34: 159-161.