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Intervention Review

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Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease

  1. Tim WR Lee1,*,
  2. Kevin W Southern2

Editorial Group: Cochrane Cystic Fibrosis and Genetic Disorders Group

Published Online: 26 NOV 2013

Assessed as up-to-date: 19 SEP 2013

DOI: 10.1002/14651858.CD005599.pub4


How to Cite

Lee TWR, Southern KW. Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease. Cochrane Database of Systematic Reviews 2013, Issue 11. Art. No.: CD005599. DOI: 10.1002/14651858.CD005599.pub4.

Author Information

  1. 1

    A Floor, Clarendon Wing, Leeds General Infirmary, Leeds Regional Paediatric Cystic Fibrosis Centre, Leeds, West Yorkshire, UK

  2. 2

    University of Liverpool, Department of Women's and Children's Health, Liverpool, Merseyside, UK

*Tim WR Lee, Leeds Regional Paediatric Cystic Fibrosis Centre, A Floor, Clarendon Wing, Leeds General Infirmary, Great George Street, Leeds, West Yorkshire, LS1 3EX, UK. timlee@doctors.org.uk.

Publication History

  1. Publication Status: New search for studies and content updated (no change to conclusions)
  2. Published Online: 26 NOV 2013

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This is not the most recent version of the article. View current version (17 JUN 2016)

References

References to studies included in this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. References to ongoing studies
  4. Additional references
  5. References to other published versions of this review
Alton 1999 {published data only}
  • Alton EW, Stern M, Farley R, Jaffe A, Chadwick SL, Phillips J, et al. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. The Lancet 1999;353(9157):947-54. [CFGD Register: BD11a]
  • Davies MG, Stern M, Geddes DM, Alton EWFW. Reduction in sputum inflammatory cells and IL-8 following pulmonary administration of liposome mediated CFTR gene transfer in CF subjects [abstract]. Thorax 1998;53(Suppl 4):A14. [CFGD Register: BD11b]
  • Stern M, Phillips J, Jaffe A, Farley R, Chadwick S, Davies J. A double blind placebo controlled trial of pulmonary and nasal administration of liposome-mediated CFTR gene transfer in CF subjects [abstract]. American Journal of Respiratory and Critical Care Medicine 1998;157(3 Suppl):A564. [CFGD Register: BD11d]
  • Stern M, Phillips J, Jaffe A, Farley R, Chadwick S, Davies J, et al. A double blind placebo controlled trial of pulmonary and nasal administration of liposome-mediated CFTR gene transfer in CF subjects [abstract]. Thorax 1997;52(Suppl 6):A3. [CFGD Register: BD11c]
Moss 2004 {published data only}
  • Moss RB, Aitken M, Clancy J, Milla C, Rodman D, Spencer T, et al. A multicenter, double-blind, placebo controlled, phase II study of aerosolized TGAAVCF in cystic fibrosis patients with mild lung disease [abstract]. Pediatric Pulmonology 2002;34(Suppl 24):250-1. [CFGD Register: BD17a]
  • Moss RB, Rodman D, Spencer LT, Aitken ML, Zeitlin PL, Waltz D, et al. Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial. Chest 2004;125(2):509-21. [CFGD Register: BD17b]
Moss 2007 {published and unpublished data}
  • Moss RB, Heald AE, 25E01 SG. Phase 2B Trial of Aerosolized tgAAVCF for Cystic Fibrosis [abstract]. Journal of Cystic Fibrosis 2005;4 Suppl:S27. [CFGD Register: BD20b; ]
  • Moss RB, Heald AE, 25E01 Study Group. A randomized phase 2B study of aerosolized tgAAVCF for treatment of cystic fibrosis [abstract]. American Thoracic Society International Conference; 2005 May 20-25; San Diego, USA. 2005:A179. [CFGD Register: BD20c]
  • Moss RB, Milla C, Colombo J, Accurso F, Zeitlin PL, Clancy JP, et al. Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Human Gene Therapy 2007;18(8):726-32. [CFGD Register: BD20d]
  • Moss RB, Milla C, Colombo J, Clancy JP, Zeitlin P, Spencer T, et al. A multicenter, double-blind, placebo-controlled, phase 2b study of aerosolized TGAAVCF for the treatment of cystic fibrosis [abstract]. Pediatric Pulmonology 2004;38(Suppl 27):257. [CFGD Register: BD20a]

References to studies excluded from this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. References to ongoing studies
  4. Additional references
  5. References to other published versions of this review
Davies 2011 {published data only}
  • Alton E. Single dose of PGM169/GL67A in CF patients. http://public.ukcrn.org.uk/search/StudyDetail.aspx?StudyID=7094 Accessed on 22 September 2009. [: UKCRN ID 7094]
  • Alton E, Davies JC, Davies G, Lees B, Mohamedhossen MH, Soussi S, et al. Safety evaluation of a single escalating dose of pGM169/GL67A in the nose and lung of individuals with cystic fibrosis. www.rbht.nhs.uk/research/projects/?entryid31=161410&catid=651&p=1 Accessed 22 September 2009. [: Royal Brompton & Harefield NHS Trust Ref No.: 2007CF011B]
  • Davies JC, Davies G, Gill DR, Hyde SC, Boyd AC, Innes AJ, et al. Safety & expression of a single dose of lipid-mediated CFTR gene therapy to the upper & lower airways of patients with CF. Pediatric Pulmonology 2011;46(Suppl 34):Abstract 198.
Flotte 1996 {published data only}
  • Flotte T, Carter B, Conrad C, Guggino W, Reynolds T, Rosenstein B, et al. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Human Gene Therapy 1996;7(9):1145-59. [CFGD Register: BD6; MEDLINE: 96369511]
Gill 1997 {published data only}
  • Gill DR, Southern KW, Mofford KA, Seddon T, Huang L, Sorgi F, et al. A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Therapy 1997;4(3):199-209. [CFGD Register: BD4c; MEDLINE: 97281425]
  • Gill DR, Southern KW, Mofford KA, Sorgi F, Huang L, Higgins CF, et al. A Phase I clinical trial to assess the safety of DNA/liposome mediated gene transfer [abstract]. Tenth Annual North American Cystic Fibrosis Conference 1996;Suppl 13:264. [CFGD Register: BD4a; ]
  • Hyde SC, Southern KW, Mofford KA, Sorgi F, Huang L, Higgins CF, et al. Towards DNA/liposome gene therapy for cystic fibrosis: A phase I clinical trial [abstract]. Pediatric Pulmonology 1996;22(Suppl 13):265. [CFGD Register: BD4b]
Harvey 1999 {published data only}
  • Harvey BG, Leopold PL, Hackett NR, Grasso TM, Williams PM, Tucker AL, et al. Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus see comments. Journal of Clinical Investigation 1999;104(9):1245-55. [CFGD Register: BD13]
Hyde 2000 {published data only}
  • Hyde SC, Southern KW, Gileadi U, Fitzjohn EM, Mofford KA, Waddell BE, et al. Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis. Gene Therapy 2000;7(13):1156-65. [CFGD Register: BD15b]
  • Southern KW, Hyde SC, Fitzjohn EM, Waddell BE, Egan JJ, Cole JY, et al. Repeated nasal administration of liposome-mediated CFTR gene transfer reagents; the clinical and immunological consequences [abstract]. Pediatric Pulmonology 1997;24(Suppl 14):265. [CFGD Register: BD15a]
Joseph 2001 {published data only}
  • Joseph PM, O'Sullivan BP, Lapey A, Dorkin H, Oren J, Balfour R, et al. Aerosol and lobar administration of a recombinant adenovirus to individuals with cystic fibrosis. I. Methods, safety, and clinical implications. Human Gene Therapy 2001;12(11):1369-82. [CFGD Register: BD16]
Knowles 1995 {published data only}
  • Knowles MR, Hohneker KW, Zhou Z, Olsen JC, Noah TL, Hu PC, et al. A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. New England Journal of Medicine 1995;333(13):823-31. [CFGD Register: BD1]
Noone 2000 {published data only}
  • Noone PG, Hohneker KW, Gipson CA, Schwartzbach CJ, Efthimiou J, Johnson LG, et al. Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis [abstract]. Pediatric Pulmonology 1998;26(Suppl 17):260. [CFGD Register: BD10a]
  • Noone PG, Hohneker KW, Zhou Z, Johnson LG, Foy C, Gipson C, et al. Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis. Molecular Therapy 2000;1(1):105-14. [CFGD Register: BD10b]
Porteous 1997 {published data only}
  • Porteous DJ, Dorin JR, McLachlan G, Davidson Smith H, Davidson H, Stevenson BJ, 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 Therapy 1997;4(3):210-8. [CFGD Register: BD5b; MEDLINE: 97281426]
  • Porteous DJ, Dorin JR, McLachlan G, Davidson-Smith H, Davidson H, Stevenson BJ, et al. DOTAP catonic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis [abstract]. Pediatric Pulmonology 1996;22(Suppl 13):266. [CFGD Register: BD5a]
Wagner 1999 {published data only}
  • Wagner JA, Messner AH, Moran ML, Daifuku R, Kouyama K, Desch JK, et al. Safety and biological efficacy of an adeno-associated virus vector - cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus. Laryngoscope 1999;109(2 Pt 1):266-74. [CFGD Register: BD8b]
  • Wagner JA, Nepomuceno IB, Shah N, Messner AH, Moran ML, Norbash AM, et al. Maxillary sinusitis as a surrogate model for CF gene therapy clinical trials in patients with antrostomies. Journal of Gene Medicine 1999;1(1):13-21. [CFGD Register: BD8c]
  • Wagner JA, Reynolds T, Moran ML, Moss RB, Wine JJ, Flotte TR, et al. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus [letter]. Lancet 1998;351(9117):1702-3. [CFGD Register: BD8a]
Wagner 2002 {published data only}
  • Wagner JA, Messner AH, Friborg S, Reynolds T, Guggino WB, Moss RB, et al. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF in the maxillary sinus of CF patients [abstract]. Pediatric Pulmonology 1998;26(Suppl 17):260. [CFGD Register: BD9a]
  • Wagner JA, Messner AH, Moran ML, Guggino WB, Flotte TR, Wine JJ, et al. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in CF patients with antrostomies [abstract]. Pediatric Pulmonology 1999;28(Suppl 19):223-4. [CFGD Register: BD9b]
  • Wagner JA, Nepomuceno IB, Messner AH, Moran ML, Batson EP, Dimiceli S, et al. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Human Gene Therapy 2002;13(11):1349-59. [CFGD Register: BD9c]
Zabner 1996 {published data only}
  • Zabner J, Ramsey BW, Meeker DP, Aitken ML, Balfour RP, Gibson RL, et al. Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosis. Journal of Clinical Investigation 1996;97(6):1504-11. [CFGD Register: BD18]
Zabner 1997 {published data only}
  • Zabner J, Cheng SH, Meeker D, Launspach J, Balfour R, Perricone MA, et al. Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo. Journal of Clinical Investigation 1997;100(6):1529-37. [CFGD Register: BD7; MEDLINE: 97439775]
Zuckerman 1999 {published data only}
  • Zuckerman JB, Robinson CB, McCoy KS, Shell R, Sferra TJ, Chirmule N, et al. A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosis. Human Gene Therapy 1999;10(18):2973-85. [CFGD Register: BD14]

References to ongoing studies

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. References to ongoing studies
  4. Additional references
  5. References to other published versions of this review
Alton 2012 {published data only}
  • Clinical study protocol - a randomised, double-blind, placebo-controlled phase 2b clinical trial of repeated application of gene therapy in patients with cystic fibrosis. Short title: repeated application of gene therapy in CF patients. Version: 3. Date 26.03.2012 [online]. http://www.eme.ac.uk/funded_projects/PDFs/111425protocol.pdf 2013. [CFGD Register: BD180d; ]
  • Project brief: A randomised double-blind placebo controlled phase 2b clinical trial of repeated application of gene therapy in patients with cystic fibrosis [online]. http://www.eme.ac.uk/funded_projects/PDFs/111425info.pdf 2013. [CFGD Register: BD180c; ]
  • Alton EW, Ashby D, Boyd C, Cheng S, Cunningham S, Davies JC, et al. Update on the UK CF gene therapy trial [abstract]. Pediatric Pulmonology 2012;47 Suppl 35:309, Abstract no: 245. [CFGD Register: BD180a; ]
  • NCT01621867. A randomised, double-blind, placebo-controlled phase 2B clinical trial of repeated application of gene therapy in patients with cystic fibrosis. http://clinicaltrials.gov/show/NCT01621867 2013. [CFGD Register: BD180b; ]

Additional references

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. References to ongoing studies
  4. Additional references
  5. References to other published versions of this review
Bobadilla 2002
Boucher 2004
Conway 1994
  • Conway SP, Pond MN, Bowler I, Smith DL, Simmonds EJ, Joanes DN, et al. The chest radiograph in cystic fibrosis: A new scoring system compared with the Chrispin-Norman and Brasfield scores. Thorax 1994;49:860-2.
Elbourne 2002
  • Elbourne DR, Altman DG, Higgins JP, Curtin F, Worthington HV, Vail A. Meta-analyses involving cross-over trials: methodological issues. International Journal of Epidemiology 2002;31:140-9.
Ferrari 2002
FitzSimmons 1993
Frederiksen 1996
Griesenbach 2004
Griesenbach 2005
  • Griesenbach U, Boyd AC. Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy. Journal of Cystic Fibrosis 2005;4:89-100.
Griesenbach 2008
  • Griesenbach U. The UK gene therapy consortium research programme [abstract]. Journal of Cystic Fibrosis 2008;7(Suppl 3):S4.
Higgins 2003
Jüni 2001
Lee 2005
Matsui 1998
  • Matsui H, Grubb BR, Tarran R, Randell SH, Gatzy JT, Davis W, et al. Evidence for periciliary liquid layer depletion, not abnormal ion composition, in the pathogenesis of cystic fibrosis airways disease. Cell 1998;95(7):1005-15.
Ranganathan 2004
  • Ranganathan SC, Stocks J, Dezateux C, Bush A, Wade A, Carr S, et al. The evolution of airway function in early childhood following clinical diagnosis of cystic fibrosis. American Journal of Respiratory and Critical Care Medicine 2004;169(8):928-33.
Riordan 1989
Rowntree 2003
Southern 1996
Southern 2003

References to other published versions of this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. References to ongoing studies
  4. Additional references
  5. References to other published versions of this review
Lee 2007
Lee 2012