A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

Eric Wfw Alton; David K Armstrong; Deborah Ashby; Katie J Bayfield; Diana Bilton; Emily V Bloomfield; A Christopher Boyd; June Brand; Ruaridh Buchan; Roberto Calcedo; Paula Carvelli; Mario Chan; Seng H Cheng; David S Collie; Steve Cunningham; Heather E Davidson; Gwyneth Davies; Jane C Davies; Lee A Davies; Maria H Dewar; Ann Doherty; Jackie Donovan; Natalie S Dwyer; Hala I Elgmati; Rosanna F Featherstone; Jemyr Gavino; Sabrina Gea-sorli; Duncan M Geddes; James Sr Gibson; Deborah R Gill; Andrew P Greening; Uta Griesenbach; David M Hansell; Katharine Harman; Tracy E Higgins; Samantha L Hodges; Stephen C Hyde; Laura Hyndman; J Alastair Innes; Joseph Jacob; Nancy Jones; Brian F Keogh; Maria P Limberis; Paul Lloyd-evans; Alan W Maclean; Michelle C Manvell; Dominique Mccormick; Michael Mcgovern; Gerry Mclachlan; Cuixiang Meng; M Angeles Montero; Hazel Milligan; Laura J Moyce; Gordon D Murray; Andrew G Nicholson; Tina Osadolor; Javier Parra-leiton; David J Porteous; Ian A Pringle; Emma K Punch; Kamila M Pytel; Alexandra L Quittner; Gina Rivellini; Clare J Saunders; Ronald K Scheule; Sarah Sheard; Nicholas J Simmonds; Keith Smith; Stephen N Smith; Najwa Soussi; Samia Soussi; Emma J Spearing; Barbara J Stevenson; Stephanie G Sumner-jones; Minna Turkkila; Rosa P Ureta; Michael D Waller; Marguerite Y Wasowicz; James M Wilson; Paul Wolstenholme-hogg

doi:10.3310/eme03050

A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

Eric Wfw Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield, Diana Bilton, Emily V Bloomfield, A Christopher Boyd, June Brand, Ruaridh Buchan, Roberto Calcedo, Paula Carvelli, Mario Chan, Seng H Cheng, David S Collie, Steve Cunningham, Heather E Davidson, Gwyneth Davies, Jane C Davies, Lee A Davies, Maria H DewarAnn Doherty, Jackie Donovan, Natalie S Dwyer, Hala I Elgmati, Rosanna F Featherstone, Jemyr Gavino, Sabrina Gea-sorli, Duncan M Geddes, James Sr Gibson, Deborah R Gill, Andrew P Greening, Uta Griesenbach, David M Hansell, Katharine Harman, Tracy E Higgins, Samantha L Hodges, Stephen C Hyde, Laura Hyndman, J Alastair Innes, Joseph Jacob, Nancy Jones, Brian F Keogh, Maria P Limberis, Paul Lloyd-evans, Alan W Maclean, Michelle C Manvell, Dominique Mccormick, Michael Mcgovern, Gerry Mclachlan, Cuixiang Meng, M Angeles Montero, Hazel Milligan, Laura J Moyce, Gordon D Murray, Andrew G Nicholson, Tina Osadolor, Javier Parra-leiton, David J Porteous, Ian A Pringle, Emma K Punch, Kamila M Pytel, Alexandra L Quittner, Gina Rivellini, Clare J Saunders, Ronald K Scheule, Sarah Sheard, Nicholas J Simmonds, Keith Smith, Stephen N Smith, Najwa Soussi, Samia Soussi, Emma J Spearing, Barbara J Stevenson, Stephanie G Sumner-jones, Minna Turkkila, Rosa P Ureta, Michael D Waller, Marguerite Y Wasowicz, James M Wilson, Paul Wolstenholme-hogg

Research output: Book/Report › Commissioned report

Original language	English
Publisher	NHS National Institute for Health Research
Commissioning body	Secretary of State for Health
Number of pages	240
Volume	3
ISBN (Electronic)	ISSN: 2050-4373
ISBN (Print)	ISSN: 2050-4365
DOIs	https://doi.org/10.3310/eme03050
Publication status	Published - Jul 2016

Publication series

Name	Efficacy and Mechanism Evaluation
Publisher	NIHR Journals Library
No.	5
Volume	3
ISSN (Print)	2050-4365

Access to Document

10.3310/eme03050

FullReport-eme03050Final published version, 36.6 MB

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Cystic fibrosis lung gene therapy models for delivery and markers.
Collie, D., Greening, A., Innes, A. & Porteous, D.
MRC
1/11/00 → 31/10/05
Project: Research

Cite this

Alton, E. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., Boyd, A. C., Brand, J., Buchan, R., Calcedo, R., Carvelli, P., Chan, M., Cheng, S. H., Collie, D. S., Cunningham, S., Davidson, H. E., Davies, G., Davies, J. C., Davies, L. A., ... Wolstenholme-hogg, P. (2016). A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis. (Efficacy and Mechanism Evaluation; Vol. 3, No. 5). NHS National Institute for Health Research. https://doi.org/10.3310/eme03050

Alton, Eric Wfw ; Armstrong, David K ; Ashby, Deborah ; Bayfield, Katie J ; Bilton, Diana ; Bloomfield, Emily V ; Boyd, A Christopher ; Brand, June ; Buchan, Ruaridh ; Calcedo, Roberto ; Carvelli, Paula ; Chan, Mario ; Cheng, Seng H ; Collie, David S ; Cunningham, Steve ; Davidson, Heather E ; Davies, Gwyneth ; Davies, Jane C ; Davies, Lee A ; Dewar, Maria H ; Doherty, Ann ; Donovan, Jackie ; Dwyer, Natalie S ; Elgmati, Hala I ; Featherstone, Rosanna F ; Gavino, Jemyr ; Gea-sorli, Sabrina ; Geddes, Duncan M ; Gibson, James Sr ; Gill, Deborah R ; Greening, Andrew P ; Griesenbach, Uta ; Hansell, David M ; Harman, Katharine ; Higgins, Tracy E ; Hodges, Samantha L ; Hyde, Stephen C ; Hyndman, Laura ; Innes, J Alastair ; Jacob, Joseph ; Jones, Nancy ; Keogh, Brian F ; Limberis, Maria P ; Lloyd-evans, Paul ; Maclean, Alan W ; Manvell, Michelle C ; Mccormick, Dominique ; Mcgovern, Michael ; Mclachlan, Gerry ; Meng, Cuixiang ; Montero, M Angeles ; Milligan, Hazel ; Moyce, Laura J ; Murray, Gordon D ; Nicholson, Andrew G ; Osadolor, Tina ; Parra-leiton, Javier ; Porteous, David J ; Pringle, Ian A ; Punch, Emma K ; Pytel, Kamila M ; Quittner, Alexandra L ; Rivellini, Gina ; Saunders, Clare J ; Scheule, Ronald K ; Sheard, Sarah ; Simmonds, Nicholas J ; Smith, Keith ; Smith, Stephen N ; Soussi, Najwa ; Soussi, Samia ; Spearing, Emma J ; Stevenson, Barbara J ; Sumner-jones, Stephanie G ; Turkkila, Minna ; Ureta, Rosa P ; Waller, Michael D ; Wasowicz, Marguerite Y ; Wilson, James M ; Wolstenholme-hogg, Paul. / A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis. NHS National Institute for Health Research, 2016. 240 p. (Efficacy and Mechanism Evaluation; 5).

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title = "A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis",

author = "Alton, {Eric Wfw} and Armstrong, {David K} and Deborah Ashby and Bayfield, {Katie J} and Diana Bilton and Bloomfield, {Emily V} and Boyd, {A Christopher} and June Brand and Ruaridh Buchan and Roberto Calcedo and Paula Carvelli and Mario Chan and Cheng, {Seng H} and Collie, {David S} and Steve Cunningham and Davidson, {Heather E} and Gwyneth Davies and Davies, {Jane C} and Davies, {Lee A} and Dewar, {Maria H} and Ann Doherty and Jackie Donovan and Dwyer, {Natalie S} and Elgmati, {Hala I} and Featherstone, {Rosanna F} and Jemyr Gavino and Sabrina Gea-sorli and Geddes, {Duncan M} and Gibson, {James Sr} and Gill, {Deborah R} and Greening, {Andrew P} and Uta Griesenbach and Hansell, {David M} and Katharine Harman and Higgins, {Tracy E} and Hodges, {Samantha L} and Hyde, {Stephen C} and Laura Hyndman and Innes, {J Alastair} and Joseph Jacob and Nancy Jones and Keogh, {Brian F} and Limberis, {Maria P} and Paul Lloyd-evans and Maclean, {Alan W} and Manvell, {Michelle C} and Dominique Mccormick and Michael Mcgovern and Gerry Mclachlan and Cuixiang Meng and Montero, {M Angeles} and Hazel Milligan and Moyce, {Laura J} and Murray, {Gordon D} and Nicholson, {Andrew G} and Tina Osadolor and Javier Parra-leiton and Porteous, {David J} and Pringle, {Ian A} and Punch, {Emma K} and Pytel, {Kamila M} and Quittner, {Alexandra L} and Gina Rivellini and Saunders, {Clare J} and Scheule, {Ronald K} and Sarah Sheard and Simmonds, {Nicholas J} and Keith Smith and Smith, {Stephen N} and Najwa Soussi and Samia Soussi and Spearing, {Emma J} and Stevenson, {Barbara J} and Sumner-jones, {Stephanie G} and Minna Turkkila and Ureta, {Rosa P} and Waller, {Michael D} and Wasowicz, {Marguerite Y} and Wilson, {James M} and Paul Wolstenholme-hogg",

year = "2016",

month = jul,

doi = "10.3310/eme03050",

language = "English",

isbn = "ISSN: 2050-4365",

volume = "3",

series = "Efficacy and Mechanism Evaluation",

publisher = "NHS National Institute for Health Research",

number = "5",

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Alton, EW, Armstrong, DK, Ashby, D, Bayfield, KJ, Bilton, D, Bloomfield, EV, Boyd, AC, Brand, J, Buchan, R, Calcedo, R, Carvelli, P, Chan, M, Cheng, SH, Collie, DS , Cunningham, S, Davidson, HE, Davies, G, Davies, JC, Davies, LA, Dewar, MH, Doherty, A, Donovan, J, Dwyer, NS, Elgmati, HI, Featherstone, RF, Gavino, J, Gea-sorli, S, Geddes, DM, Gibson, JS, Gill, DR, Greening, AP, Griesenbach, U, Hansell, DM, Harman, K, Higgins, TE, Hodges, SL, Hyde, SC, Hyndman, L, Innes, JA, Jacob, J, Jones, N, Keogh, BF, Limberis, MP, Lloyd-evans, P, Maclean, AW, Manvell, MC, Mccormick, D, Mcgovern, M, Mclachlan, G, Meng, C, Montero, MA, Milligan, H, Moyce, LJ, Murray, GD, Nicholson, AG, Osadolor, T, Parra-leiton, J, Porteous, DJ, Pringle, IA, Punch, EK, Pytel, KM, Quittner, AL, Rivellini, G, Saunders, CJ, Scheule, RK, Sheard, S, Simmonds, NJ, Smith, K, Smith, SN, Soussi, N, Soussi, S, Spearing, EJ, Stevenson, BJ, Sumner-jones, SG, Turkkila, M, Ureta, RP, Waller, MD, Wasowicz, MY, Wilson, JM & Wolstenholme-hogg, P 2016, A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis. Efficacy and Mechanism Evaluation, no. 5, vol. 3, vol. 3, NHS National Institute for Health Research. https://doi.org/10.3310/eme03050

A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis. / Alton, Eric Wfw; Armstrong, David K; Ashby, Deborah; Bayfield, Katie J; Bilton, Diana; Bloomfield, Emily V; Boyd, A Christopher; Brand, June; Buchan, Ruaridh; Calcedo, Roberto; Carvelli, Paula; Chan, Mario; Cheng, Seng H; Collie, David S ; Cunningham, Steve; Davidson, Heather E; Davies, Gwyneth; Davies, Jane C; Davies, Lee A; Dewar, Maria H; Doherty, Ann; Donovan, Jackie; Dwyer, Natalie S; Elgmati, Hala I; Featherstone, Rosanna F; Gavino, Jemyr; Gea-sorli, Sabrina; Geddes, Duncan M; Gibson, James Sr; Gill, Deborah R; Greening, Andrew P; Griesenbach, Uta; Hansell, David M; Harman, Katharine; Higgins, Tracy E; Hodges, Samantha L; Hyde, Stephen C; Hyndman, Laura; Innes, J Alastair; Jacob, Joseph; Jones, Nancy; Keogh, Brian F; Limberis, Maria P; Lloyd-evans, Paul; Maclean, Alan W; Manvell, Michelle C; Mccormick, Dominique; Mcgovern, Michael; Mclachlan, Gerry; Meng, Cuixiang; Montero, M Angeles; Milligan, Hazel; Moyce, Laura J; Murray, Gordon D; Nicholson, Andrew G; Osadolor, Tina; Parra-leiton, Javier; Porteous, David J; Pringle, Ian A; Punch, Emma K; Pytel, Kamila M; Quittner, Alexandra L; Rivellini, Gina; Saunders, Clare J; Scheule, Ronald K; Sheard, Sarah; Simmonds, Nicholas J; Smith, Keith; Smith, Stephen N; Soussi, Najwa; Soussi, Samia; Spearing, Emma J; Stevenson, Barbara J; Sumner-jones, Stephanie G; Turkkila, Minna; Ureta, Rosa P; Waller, Michael D; Wasowicz, Marguerite Y; Wilson, James M; Wolstenholme-hogg, Paul.

NHS National Institute for Health Research, 2016. 240 p. (Efficacy and Mechanism Evaluation; Vol. 3, No. 5).

Research output: Book/Report › Commissioned report

TY - BOOK

T1 - A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

AU - Alton, Eric Wfw

AU - Armstrong, David K

AU - Ashby, Deborah

AU - Bayfield, Katie J

AU - Bilton, Diana

AU - Bloomfield, Emily V

AU - Boyd, A Christopher

AU - Brand, June

AU - Buchan, Ruaridh

AU - Calcedo, Roberto

AU - Carvelli, Paula

AU - Chan, Mario

AU - Cheng, Seng H

AU - Collie, David S

AU - Cunningham, Steve

AU - Davidson, Heather E

AU - Davies, Gwyneth

AU - Davies, Jane C

AU - Davies, Lee A

AU - Dewar, Maria H

AU - Doherty, Ann

AU - Donovan, Jackie

AU - Dwyer, Natalie S

AU - Elgmati, Hala I

AU - Featherstone, Rosanna F

AU - Gavino, Jemyr

AU - Gea-sorli, Sabrina

AU - Geddes, Duncan M

AU - Gibson, James Sr

AU - Gill, Deborah R

AU - Greening, Andrew P

AU - Griesenbach, Uta

AU - Hansell, David M

AU - Harman, Katharine

AU - Higgins, Tracy E

AU - Hodges, Samantha L

AU - Hyde, Stephen C

AU - Hyndman, Laura

AU - Innes, J Alastair

AU - Jacob, Joseph

AU - Jones, Nancy

AU - Keogh, Brian F

AU - Limberis, Maria P

AU - Lloyd-evans, Paul

AU - Maclean, Alan W

AU - Manvell, Michelle C

AU - Mccormick, Dominique

AU - Mcgovern, Michael

AU - Mclachlan, Gerry

AU - Meng, Cuixiang

AU - Montero, M Angeles

AU - Milligan, Hazel

AU - Moyce, Laura J

AU - Murray, Gordon D

AU - Nicholson, Andrew G

AU - Osadolor, Tina

AU - Parra-leiton, Javier

AU - Porteous, David J

AU - Pringle, Ian A

AU - Punch, Emma K

AU - Pytel, Kamila M

AU - Quittner, Alexandra L

AU - Rivellini, Gina

AU - Saunders, Clare J

AU - Scheule, Ronald K

AU - Sheard, Sarah

AU - Simmonds, Nicholas J

AU - Smith, Keith

AU - Smith, Stephen N

AU - Soussi, Najwa

AU - Soussi, Samia

AU - Spearing, Emma J

AU - Stevenson, Barbara J

AU - Sumner-jones, Stephanie G

AU - Turkkila, Minna

AU - Ureta, Rosa P

AU - Waller, Michael D

AU - Wasowicz, Marguerite Y

AU - Wilson, James M

AU - Wolstenholme-hogg, Paul

PY - 2016/7

Y1 - 2016/7

U2 - 10.3310/eme03050

DO - 10.3310/eme03050

M3 - Commissioned report

SN - ISSN: 2050-4365

VL - 3

T3 - Efficacy and Mechanism Evaluation

BT - A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

PB - NHS National Institute for Health Research

ER -

Alton EW, Armstrong DK, Ashby D, Bayfield KJ, Bilton D, Bloomfield EV et al. A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis. NHS National Institute for Health Research, 2016. 240 p. (Efficacy and Mechanism Evaluation; 5). https://doi.org/10.3310/eme03050

A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

Publication series

Access to Document

Cystic fibrosis lung gene therapy models for delivery and markers.

Cite this