Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMV-CFTR-DOTAP

Gene Therapy

Volumn 3, Issue 12, 1996, Pages 1113-1123

  McLachlan, G ^a   Ho, L P ^b   Davidson Smith, H ^a   Samways, J ^b   Davidson, H ^a   Stevenson, B J ^a   Carothers, A D ^a   Alton, E W F W ^c   Middleton, P G ^c   Smith, S N ^c   Kallmeyer, G ^d   Michaelis, U ^d   Seeber, S ^d   Naujoks, K ^d   Greening, A P ^b   Innes, J A ^b   Dorin, J R ^a   Porteous, D J ^a  

^a WESTERN GENERAL HOSPITAL   (United Kingdom)

^b UNIVERSITY OF EDINBURGH   (United Kingdom)

^c NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

^d F HOFFMANN LA ROCHE LTD   (Switzerland)

Author keywords

Cystic fibrosis; Gene therapy; PcMV CFTR DOTAP

Indexed keywords

HALIDE; ION CHANNEL; LIPOSOME; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CYSTIC FIBROSIS; DOUBLE BLIND PROCEDURE; DRUG FORMULATION; DRUG STABILITY; ELECTROPHYSIOLOGY; FEMALE; FLUORESCENCE ANALYSIS; GENE TARGETING; GENE THERAPY; GENE TRANSFER; HUMAN; INTRANASAL DRUG ADMINISTRATION; INTRATRACHEAL DRUG ADMINISTRATION; ION TRANSPORT; MALE; MOLECULAR GENETICS; MOUSE; MOUSE MUTANT; NONHUMAN; NOSE; NOSE MUCOSA; PHASE 1 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; TRACHEA;

AEROSOLS; ANIMALS; CLINICAL TRIALS, PHASE I; COS CELLS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; CYTOMEGALOVIRUS; FATTY ACIDS, MONOUNSATURATED; GENE THERAPY; GENETIC VECTORS; HUMANS; LIPOSOMES; MEMBRANE POTENTIALS; MICE; NASAL MUCOSA; QUATERNARY AMMONIUM COMPOUNDS; QUINOLINIUM COMPOUNDS; RNA, MESSENGER; TRANSFECTION; VEHICLES;

EID: 12644286576     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (33)

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 et 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. Quinton PM. Cystic fibrosis: a disease in electrolyte transport. FASEB J 1990; 4: 2709-2717.
5. Stutts MJ et al. Cftr as a cAMP-dependent regulator of sodium channels. Science 1995; 269: 847-850.
6. Gabriel SE, Clarke LL, Boucher RC, Stutts MJ. CFTR and outwardly rectifying chloride channels are distinct proteins with a regulatory relationship. Nature 1993; 363: 263-266.
7. Schwiebert EM, Kizer N, Gruenert DC, Stanton BA. GTP-binding proteins inhibit cAMP activation of chloride channels in cystic fibrosis airway epithelial cells. Proc Natl Acad Sci USA 1992; 89: 10623-10627.
8. Wilson JM. Gene therapy for cystic fibrosis: challenges and future directions. J Clin Invest 1995; 96: 2547-2554.
9. Grubb BR et al. Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans. Nature 1994; 371: 802-806.
10. 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.
11. Gao X, Huang L. Cationic liposome-mediated gene transfer. Gene Therapy 1995; 2: 710-722.
12. Feigner PL et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987; 84: 7413-7417.
13. Drumm ML et al. Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell 1990; 62: 1227-1233.
14. Rich DP et al. Expression of cystic fibrosis transmembrane conductor regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells. Nature 1990; 347: 358-363.
15. McLachlan G et al. Evaluation in vitro and in vivo of carionic liposome-expression construct complexes for cystic fibrosis gene therapy. Gene Therapy 1995; 2: 614-622.
16. 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.
17. Dorin JR et al. Cystic fibrosis in the mouse by targeted inserhonal mutagenesis. Nature 1992; 359: 211-215.
18. Ratcliff R et al. Production of a severe cystic fibrosis mutation in mice by gene targeting. Nat Genet 1993; 4: 35-41.
19. Smith SN et al. Bioelectric characteristics of the exon 10 insertional cystic fibrosis mouse: comparison with humans. Am J Physiol 1995; 268 (Cell Physiology, 37): C297-307.
20. 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.
21. Hyde SC et al. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature 1993; 362: 250-255.
22. Caplen NJ et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1995; 1: 39-46.
23. Illsley NP, Verkman AS. Membrane chloride transport measured using a chloride-sensitive fluorescent indicator. Biochemistry 1987; 26: 1215-1219.
24. Alton EWFW et al. Nasal potential difference: a clinical diagnostic test for cystic fibrosis. Eur Resp J 1990; 3: 922-926.
25. Middleton PG, Geddes DM, Alton EWFW. Protocols for in vivo measurement for ion transport defects in cystic fibrosis nasal epithelium. Eur Resp J 1994; 7: 2050-2056.
26. Gao X, Huang L. A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem Biophys Res Commun 1991; 179: 280-285.
27. Nabel GJ, Chang A, Nabel EG. Clinical protocol: immunotherapy of malignancy by in vivo gene transfer into tumors. Hum Gene Ther 1992; 3: 399-410.
28. Caplen NJ et al. Gene therapy for cystic fibrosis in humans by liposome-mediated DNA transfer: the production of resources and the regulatory process. Gene Therapy 1994, 1 139-147.
29. Feigner JH et al. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J Biol Chem 1994; 269: 2550-2561.
30. Ledley FD. Non-viral gene therapy: the promise of genes as pharmaceutical products. Hum Gene Ther 1995; 6: 1129-1144.
31. Gorman CM, Moffat LF, Howard BH. Recombinant genomes which express chloramphenicol acetyltransferase in mammalian cells. Mol Cell Biol 1982; 2: 1044-1051.
32. Dorin JR et al. A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Therapy 1996; 3: 797-801.
33. Stamatatos L, Leventis R, Zuckerman MJ, Silvius JR. Interactions of cationic lipid vesicles with negatively charged phospholipid vesicles and biological membranes. Biochemistry 1988; 27: 3917-3925.