Use of liposomes in the treatment of cystic fibrosis

Gene Therapy in Lung Disease

Volumn , Issue , 2002, Pages 383-396

  Stern, Myra ^a   Alton, Eric W F W ^a  

^a NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 85018359650     PISSN: None     EISSN: None     Source Type: Book    
DOI: None     Document Type: Chapter

References (45)

1. Ferrari S, Moro E, Pentenazzo A, et al. Exgen 500 is an efficient vector for gene delivery to lung epithelial cells in vitro and in vivo. Gene Ther 1997; 4:1100-1106.
2. Rudolph C, Lausier J, Naundorf S, et al. In vivo delivery to the lung using polyethyl- enimine and fractured polyamidoamine dendrimers. J Gene Med 2000; 2:269-278.
3. Sternberg B, Sorgi FL, Huang L. New structures in complex formation between DNA and cationic liposomes visualized by freeze-fracture electron microscopy. FEBS Lett 1994; 356:361-366.
4. Felgner PL, Gadek TR, Holm M, et al. Lipofection: A highly efficient lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987; 84:7413-7417.
5. Felgner JH, Kumar R, Sridhar CN, et al. Enhanced gene delivery and mechanism studies with A novel series of cationic lipid formulations. J Biol Chem 1994; 269: 2550-2561.
6. Leventis R, Silvius JR. Interactions of mammalian cells with lipid dispersions containing novel metabolizable cationic amphiphiles. Biochim Biophys Acta 1990; 1023:124-132.
7. Gao X, Huang L. A novel cationic liposome for efficient transfection of mammalian cells. Biochem Biophys Res Commun 1991; 179:280-285.
8. Lee ER, Marshall J, Siegel et al. Detailed analysis of structure and formulations of cationic lipids for efficient gene transfer to the lung. Hum Gene Ther 1996; 7:1701-1717.
9. Labat-Moleur F, Steffan AM, Brisson C, et al. An electron microscopy study into the mechanism of gene transfer with polyamines. Gene Ther 1996; 3:1010-1017.
10. Kitson C, Angel B, Judd D, et al. The extra- and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epithelium. Gene Ther 1999; 6:534-546.
11. Xu Y, Szoka F. Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection. Biochemistry 1996; 35:5616-5623.
12. Wilson GL, Dean BS, Wang G, et al. Nuclear import of plasmid DNA in digitonin- permeabilised cells requires both cytoplasmic factors and specific DNA sequences. J Biol Chem 1999; 274:22025-22032.
13. Riordan JR, Rommens JM, Kerem BS, et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science 1989; 245:1066-1073.
14. Welsh MJ. Abnormal regulation of ion channels in cystic fibrosis. FASEB J 1990; 4:2718-2725.
15. Knowles MR, Gatzy JT, Boucher RC. Increased bioelectric potential difference across respiratory epithelia in cystic fibrosis. N Engl J Med 1981; 305:1489-1495.
16. Drumm M, Pope HA, Cliff WH, et al. Correction of the CF defect in vitro by retrovirus-mediated gene transfer. Cell 1999; 62:1227-1233.
17. Rich DP, Anderson MP, Gregory RJ, et al. Expression of cystic fibrosis transmembrane conductance regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells. Nature 1990; 347:358-363.
18. Hyde SC, Gill DR, Higgins CF, et al. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature 1993; 362:250-255.
19. Alton EWFW, Middleton PG, Caplen NJ, et al. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mice. Nat Genet 1993; 5:135-142.
20. Zabner J, Couture RA, Gregory RJ, 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.
21. Crystal RG, McElvanehy LG, Rosenfeld MA, et al. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Genet 1994; 8:42-51.
22. Boucher RC, Knowles MR, Johnson LG, et al. Gene therapy for cystic fibrosis using E1-deleted adenovirus: A phase I trial in the nasal cavity. Hum Gene Ther 1994; 5: 615-619.
23. Caplen NJ, Alton EW, Middleton PG, et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1995; 1:39-46.
24. Gill DR, Southern KW, Mofford KA, et al. A placebo controlled study of liposome- mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther 1997; 4:199-209.
25. Porteus DJ, Dorin JR, McLachlan G, et al. Evidence for safety and efficacy of DOTAP cationic liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther 1997; 4:210-218.
26. Zabner J, Cheng S, Meeker D, et al. Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelium in vivo. J Clin Invest 1997; 100:1529-1537.
27. Alton EWFW, Stern M, Farley R, et al. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: A double-blind placebo- controlled trial. Lancet 1999; 353:947-954.
28. Ruiz FE, Clancy JP, Perricone MA, et al. A clinical inflammatory syndrome attributable to aerosolised lipid-DNA administration in cystic fibrosis. Hum Gene Ther 2001; 12:751-61.
29. Hyde SC, Southern KW, Gileadi U, et al. Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis. Gene Ther 2000; 7:1156-1165.
30. Noone PG, Hohneker KW, Zhou Z, et al. Safety and efficacy of A lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis. Mol Med 2000; 1:105-114.
31. Middleton PG, Geddes DM, Alton EWFW. Protocols for in vivo measurement of the ion transport defects in cystic fibrosis epithelium. Eur Respir J 1994; 7:2050-2056.
32. Stern M, Munkonge F, Caplen NJ et al. Quantitative fluorescent measurements of chloride secretion in native airway epithelium from CF and non-CF subjects. Gene Ther 1995; 2:766-774.
33. Chadwick SL, Kingston HD, Stern M, et al. Safety of A single aerosol administration of escalating doses of the cationic lipid 67/DOPE/DMPE-PEG500 formulation to the lungs of normal volunteers. Gene Ther 1997; 4:937-942.
34. Davies J, Stern M, Dewar A, et al. CFTR gene transfer reduces the binding of pseudomonas aeruginosa to cytic fibrosis epithelium. Am J Respir Cell Mol Biol 1997; 16:657-663.
35. Schwartz DA, Quinn TJ, Thorne PS, et al. CpG motifs in bacterial DNA cause inflammation in the lower respiratory tract. J Clin Invest 1997; 100:68-73.
36. Stern M, Caplen NJ, Browning JE, et al. The effect of mucolytic agents on gene transfer across A CF sputum barrier in vitro. Gene Ther 1998; 5:91-98.
37. Matsui H, Johnson LG, Randell SH, et al. Loss of binding and entry of liposome- DNA complexes decreases transfection efficiency in differentiated airway epithelial cells. J Biol Chem 1997; 272:1117-1126.
38. Wagner E, Zenke M, Cotten M, et al. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc Natl Acad Sci USA 1990; 87:3410-3414.
39. Cotton M, Langle-Rouault R, Kirlappos H, et al. Transferrin-polycation-mediated introduction of DNA into human leukemic cells: stimulation by agents that affect the survival of transfected DNA or modulate transferrin receptor levels. Proc Natl Acad Sci USA 1990; 87:4033-4037.
40. Kollen WJ, Mulberg AE, Wei X, et al. High efficiency transfer of cystic fibrosis transmembrane conductance regulator into cystic fibrosis airway cells in culture using lactosylated polylysine as A vector. Hum Gene Ther 1999; 10:615-622.
41. Truong-Le VL, August JT, Leong KW. Controlled gene delivery by DNA-gelatin nanospheres. Hum Gene Ther 1998; 8:817-825.
42. Blessing T, Remy JS, Behr JP. Monomolecular collapse of plasmid DNA inot stable virus-like particles. Proc Natl Acad Sci USA 1998; 95:1427-1431.
43. Yonemitsu Y, Kaneda Y, Muraishi A, et al. HVJ (Sendai virus)-cationic liposomes: A novel and potentially effective liposome-mediated technique for gene transfer to the airway epithelium. Gene Ther 1997; 4:631-638.
44. Johnson LG, Olsen JC, Sarkadi B. et al. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 1992; 2:21-25.
45. Johnson LG, Boyles SE, Wilson J, 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; 95:1377-82