Cystic fibrosis gene therapy trials and tribulations

Molecular Therapy

Volumn 1, Issue 1, 2000, Pages 5-6

  Zeitlin, Pamela L ^a  

^a JOHNS HOPKINS HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CFTR PROTEIN, HUMAN; MESSENGER RNA; TRANSMEMBRANE CONDUCTANCE REGULATOR;

CLINICAL TRIAL; CYSTIC FIBROSIS; GENE EXPRESSION; GENE THERAPY; GENETICS; HUMAN; METABOLISM; METHODOLOGY; NOTE;

CLINICAL TRIALS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; GENE EXPRESSION; GENE THERAPY; HUMANS; RNA, MESSENGER;

EID: 0033642893     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1006/mthe.1999.0016     Document Type: Note

References (15)

1. Noone, P. G., Hohneker, K. W., Zhou, Z., Johnson, L. G., Foy, C., Gipson, C., Jones, K., Noah, T. L., Leigh, M. W., Schwartzbach, C., Efthimiou, J., Pearlman, R., Boucher, R. C., and Knowles, M. R. (2000). Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis. Mol. Ther. 1: 105-114.
2. Caplen, N. J., Alton, E. W., Middleton, P. G., Dorin, J. R., Stevenson, B. J., Gao, X., Durham, S. R., Jeffery, P. K., Hodson, M. E., and Coutelle, C. (1995). Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis [see comments]. Nat. Med. 1: 39-46. [published erratum appears in Nat. Med. 1(3): 272, 1995.]
3. Porteous, D. J., Dorin, J. R., McLachlan, G., Davidson-Smith, H., Davidson, H., Stevenson, B. J., Carothers, A. D., Wallace, W. A., Moralee, S., Hoenes, C., Kallmeyer, G., Michaelis, U., Naujoks, K., Ho, L. P., Samways, J. M., Imrie, M., Greening, A. P., and Innes, J. A. (1997). Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. 4: 210-218.
4. Zabner, J., Cheng, S. H., Meeker, D., Launspach, J., Balfour, R., Perricone, M. A., Morris, J. E., Marshall, J., Fasbender, A., Smith, A. E., and Welsh, M. J. (1997). Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo. J. Clin. Invest. 100: 1529-1537.
5. Alton, E. W., Stern, M., Farley, R., Jaffe, A., Chadwick, S. L., Phillips, J., Davies, J., Smith, S. N., Browning, J., Davies, M. G., Hodson, M. E., Durham, S. R., Li, D., Jeffery, P. K., Scallan, M., Balfour, R., Eastman, S. J., Cheng, S. H., Smith, A. E., Meeker, D., and Geddes, D. M. (1999). Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: A double-blind placebo-controlled trial. Lancet 353: 947-954.
6. Middleton, P. G., Geddes, D. M., and Alton, E. W. (1994). Protocols for in vivo measurement of the ion transport defects in cystic fibrosis nasal epithelium [see comments]. Eur. Respir. J. 7: 2050-2056.
7. Knowles, M. R., Paradiso, A. M., and Boucher, R. C. (1995). In vivo nasal potential difference: Techniques and protocols for assessing efficacy of gene transfer in cystic fibrosis. Hum. Gene Ther. 6: 445-455.
8. Wilschanski, M., Famini, C., Blau, H., Rivlin, J., Augarten, A., Avital, A., Kerem, B., and Kerem, E. (1999). A pilot trial of the effect of gentamicin on nasal potential difference measurements in cystic fibrosis patients carrying stop mutations. Am. J. Respir. Crit. Care Med., in press.
9. Rubenstein, R. C., and Zeitlin, P. L. (1998). A pilot clinical trial of sodium 4-phenylbutyrate (Buphenyl) in delta F508-homozygous cystic fibrosis patients: Evidence of restoration of nasal epithelial CFTR function. Am. J. Respir. Crit. Care Med. 157: 484-490.
10. Johnson, L. G., Olsen, J. C., Sarkadi, B., Moore, K. L., Swanstrom, R., and Boucher, R. C. (1992). Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat. Genet. 2: 21-25.
11. Matsui, H., Johnson, L. G., Randell, S. H., and Boucher, R. C. (1997). Loss of binding and entry of liposome-DNA complexes decreases transfection efficiency in differentiated airway epithelial cells. J. Biol. Chem. 272: 1117-1126.
12. Parsons, D. W., Grubb, B. R., Johnson, L. G., and Boucher, R. C. (1998). Enhanced in vivo airway gene transfer via transient modification of host barrier properties with a surfaceactive agent. Hum. Gene Ther. 9: 2661-2672.
13. Kitson, C., Angel, B., Judd, D., Rothery, S., Severs, N. J., Dewar, A., Huang, L., Wadsworth, S. C., Cheng, S. H., Geddes, D. M., and Alton, E. W. (1999). The extra-and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epithelium. Gene Ther. 6: 534-546.
14. Knoell, D. L., and Yiu, I. M. (1998). Human gene therapy for hereditary diseases: A review of trials. Am. J. Health Syst. Pharm. 55: 899-904.
15. Ayers, M. M., and Jeffery, P. K. (1988). Proliferation and differentiation in mammalian airway epithelium. Eur. Respir. J. 1: 58-80.