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Cystic Fibrosis gene therapy in the UK and elsewhere
File(s)
hum%2E2015%2E027.pdf (282.83 KB)
Published version
Author(s)
Griesenbach, U
Pytel, K
Alton, EWFW
Type
Journal Article
Abstract
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.
Date Issued
2015-04-02
Date Acceptance
2015-02-23
Citation
Human Gene Therapy, 2015, 26 (5), pp.226-275
URI
DOI
ISSN
1043-0342
Publisher
Mary Ann Liebert
Start Page
226
End Page
275
Journal / Book Title
Human Gene Therapy
Volume
26
Issue
5
Copyright Statement
© Uta Griesenbach et al. 2015; Published by Mary Ann Liebert, Inc. This Open Access article is distributed under the terms of the Creative Commons License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited.
License URL
Publication Status
Published