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Clinical Trial
. 2013 Nov;68(11):1075-7.
doi: 10.1136/thoraxjnl-2013-203309. Epub 2013 Mar 22.

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  1 , 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
Affiliations
Clinical Trial

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 et al. Thorax. 2013 Nov.

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 non-viral, liposome-based formulation powered for the first time to detect clinical benefit. The article describes the details of the protocol.

Keywords: Cystic Fibrosis.

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