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

Alton, Eric W. F. W.; Boyd, A. Christopher; Seng H. Cheng; Cunningham, Steve; Davies, Jane C.; Gill, Deborah R.; Griesenbach, Uta; Higgins, Tracy; Hyde, Stephen C.; Innes, J. Alastair; Murray, Gordon D.; Porteous, David J.
November 2013
Thorax;Nov2013, Vol. 68 Issue 11, p1075
Academic Journal
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.


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