Imperial College London
Alternate

ProfessorUtaGriesenbach

Faculty of Medicine, National Heart & Lung Institute

Professor of Molecular Medicine
 
 
 
//

Contact

 

+44 (0)20 7594 7927u.griesenbach

 
 
//

Assistant

 

Miss Samia Soussi +44 (0)20 7594 7980

 
//

Location

 

Emmanuel Kaye BuildingRoyal Brompton Campus

//

Summary

 

Publications

Citation

BibTex format

@article{Griesenbach:2015:10.1089/hum.2015.027,
author = {Griesenbach, U and Pytel, K and Alton, EWFW},
doi = {10.1089/hum.2015.027},
journal = {Human Gene Therapy},
pages = {226--275},
title = {Cystic Fibrosis gene therapy in the UK and elsewhere},
url = {http://dx.doi.org/10.1089/hum.2015.027},
volume = {26},
year = {2015}
}
Download

RIS format (EndNote, RefMan)

TY  - JOUR
AB  - The cystic fibrosis transmembrane conductance regulator (CFTR) gene was identified in 1989. This opened the door for the development of cystic fibrosis (CF) gene therapy, which has been actively pursued for the last 20 years. Although 26 clinical trials involving approximately 450 patients have been carried out, the vast majority of these trials were short and included small numbers of patients; they were not designed to assess clinical benefit, but to establish safety and proof-of-concept for gene transfer using molecular end points such as the detection of recombinant mRNA or correction of the ion transport defect. The only currently published trial designed and powered to assess clinical efficacy (defined as improvement in lung function) administered AAV2-CFTR to the lungs of patients with CF. The U.K. Cystic Fibrosis Gene Therapy Consortium completed, in the autumn of 2014, the first nonviral gene therapy trial designed to answer whether repeated nonviral gene transfer (12 doses over 12 months) can lead to clinical benefit. The demonstration that the molecular defect in CFTR can be corrected with small-molecule drugs, and the success of gene therapy in other monogenic diseases, is boosting interest in CF gene therapy. Developments are discussed here.
AU  - Griesenbach,U
AU  - Pytel,K
AU  - Alton,EWFW
DO  - 10.1089/hum.2015.027
EP  - 275
PY  - 2015///
SN  - 1043-0342
SP  - 226
TI  - Cystic Fibrosis gene therapy in the UK and elsewhere
T2  - Human Gene Therapy
UR  - http://dx.doi.org/10.1089/hum.2015.027
UR  - http://hdl.handle.net/10044/1/25873
VL  - 26
ER  -
Download