A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis

Eric W F W Alton, A. Christopher Boyd, Seng H. Cheng, Steve Cunningham, Jane C. Davies*, Deborah R. Gill, Uta Griesenbach, Tracy Higgins, Stephen C. Hyde, J. Alastair Innes, Gordon D. Murray, David J. Porteous

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

The UK Cystic Fibrosis Gene Therapy Consortium has been working towards clinical gene therapy for patients with cystic fibrosis for several years. We have recently embarked on a large, multi-dose clinical trial of a nonviral, liposome-based formulation powered for the first time to detect clinical benefit. The article describes the details of the protocol.

Original languageEnglish
Pages (from-to)1075-1077
Number of pages3
JournalThorax
Volume68
Issue number11
DOIs
Publication statusPublished - 1 Nov 2013

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  • MRC HUB NETWORK WORK

    Murray, G.

    MRC

    1/01/0931/08/15

    Project: Research

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