Prospects for gene therapy in cystic fibrosis

Archives of Disease in Childhood

Volumn 80, Issue 3, 1999, Pages 286-289

  Jaffe A ^a   Bush, A ^a   Geddes, D M ^b   Alton, E W F W ^b  

^a ROYAL BROMPTON AND HAREFIELD NHS FOUNDATION TRUST   (United Kingdom)

^b IMPERIAL COLLEGE SCHOOL OF MEDICINE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

TRANSMEMBRANE CONDUCTANCE REGULATOR;

ADENOVIRUS; BINDING SITE; COST BENEFIT ANALYSIS; CYSTIC FIBROSIS; GENE THERAPY; LUNG DEVELOPMENT; PATHOPHYSIOLOGY; PRIORITY JOURNAL; REVIEW; SURVIVAL RATE;

EID: 0033010167     PISSN: 00039888     EISSN: 14682044     Source Type: Journal    
DOI: 10.1136/adc.80.3.286     Document Type: Review

References (27)

1. 1 Kerem BS, Rommens JM, Buchanan JA, et al. Identification of the cystic fibrosis gene: genetic analysis. Science 1989;245:1073-80.
2. 2 O'Brodovich H. Epithelial ion transport in the fetal and perinatal lung. Am J Physiol 1991;261:C555-64.
3. 3 Tizzano EF, O'Brodovich H, Chitayat D, Benichou JC, Buchwald M. Regional expression of CFTR in developing human respiratory tissues. Am J Respir Cell Mol Biol 1994; 10:355-62.
4. 4 Jeffery PK, Gaillard D, Moret S. Human airway secretory cells during development and in mature airway epithelium. Eur Respir J 1992;5:93-104.
5. 5 Johnson LG, Olsen JC, Sarkadi B, Moore KL, Swanstrom R, Boucher RC. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 1992;2:21-5.
6. 6 Goldman MJ, Anderson GM, Stolzenberg ED, Kari UP, Zasloff M, Wilson JM. Human β-defensin-1 is a salt-sensitive antibiotic in lung that is inactivated in cystic fibrosis. Cell 1997;88:553-60.
7. 7 Pier GB, Grout M, Zaidi TS. Cystic fibrosis transmembrane conductance regulator is an epithelial cell receptor for clearance of Pseudomonas aeruginosa from the lung. Proc Natl Acad Sci USA 1997;94:12088-93.
8. 8 Davies JC, Stern M, Dewar A, et al. CFTR gene transfer reduces the binding of Pseudomonas aeruginosa to cystic fibrosis respiratory epithelium. Am J Respir Cell Mol Biol 1997;16:657-63.
9. 9 Alton EWFW, Geddes DM, Gill DR, et al. Towards gene therapy for cystic fibrosis: a clinical progress report. Gene Ther 1998;4:291-2.
10. 10 Wagner JA, Reynolds T, Moran ML, et al. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus [research letter]. Lancet 1998;351:1702-3.
11. 11 Crystal RG, Jaffe A, Brody S, et al. A phase 1 study, in cystic fibrosis patients, of the safety, toxicity, and biological efficacy of a single administration of a replication deficient, recombinant adenovirus carrying the cDNA of the normal cystic fibrosis transmembrane conductance regulator gene in the lung. Hum Gene Ther 1995;6:643-66.
12. 12 Alton EWFW, Stern M, Farley R, et al. A double blind placebo controlled trial of pulmonary and nasal administration of liposome-mediated CFTR gene transfer in CF subjects. Lancet. [In press.]
13. 13 Alton EWFW, Currie D, Logan-Sinclair R, Warner JO, Hodson ME, Geddes DM. Nasal potential difference: a clinical diagnostic test for cystic fibrosis. Eur Respir J 1990;3:922-6.
14. 14 Middleton PG, Geddes DM, Alton EWFW. Protocols for in vivo measurement of the ion transport defects in cystic fibrosis nasal epithelium. Eur Respir J 1994;7:2050-6.
15. 15 Stern M, Munkonge FM, Caplen NJ, et al. Quantitative fluorescence measurements of chloride secretion in native airway epithelium from CF and non-CF subjects. Gene Ther 1995;2:766-74.
16. 16 Kohn DB, Parkman R. Gene therapy for newborns. FASEB J 1997;11:635-9.
17. 17 Ethics Advisory Committee. Guidelines for the ethical conduct of medical research involving children. London: British Paediatric Association, 1992.
18. 18 Kass Eisler A, Falck Pedersen E, Elfenbein DH, Alvira M, Buttrick PM, Leinwand LA. The impact of developmental stage, route of administration and the immune system on adenovirus-mediated gene transfer. Gene Ther 1994;1:395-402.
19. 19 Huard J, Lochmuller H, Acsadi G, et al. Differential short-term transduction efficiency of adult versus newborn mouse tissues by adenoviral recombinants. Exp Mol Pathol 1995;62:131-43.
20. 20 Rubinstein RC, McVeigh U, Flotte TR, Guggino WB, Zeitlin PL. CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vector. Gene Ther 1997;4: 384-92.
21. 21 Zepeda M, Wilson JM. Neonatal cotton rats do not exhibit destructive immune responses to adenoviral vectors. Gene Ther 1996;3:973-9.
22. 22 Jaffe A, Judd D, Bush A, Cheng SH, Geddes DM, Alton EWFW. Cationic lipid-mediated gene transfer to the growing murine airway [abstract]. Pediatr Pulmonol 1998;17: S266.
23. 23 Sekhon HS, Larson JE. In utero gene transfer into the pulmonary epithelium. Nat Med 1995;1:1201-3.
24. 24 Douar AM, Adebakin S, Themis M, Pavirani A, Cook T, Coutelle C. Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus. Gene Ther 1997;4:883-90.
25. 25 Larson JE, Morrow SL, Happel L, Sharp JF, Cohen JC. Reversal of cystic fibrosis phenotype in mice by gene therapy in utero [research letter]. Lancet 1997;349:619-20.
26. 26 Coutelle C, Douar AM, Colledge WH, Froster U. The challenge of fetal gene therapy. Nat Med 1995;1:864-6.
27. 27 Mahadeva R, Webb K, Westerbeek RC, Carroll NR, Dodd ME, Bilton D. Clinical outcome in relation to care in centres specialising in cystic fibrosis: cross sectional study. BMJ 1998;316:1771-5.