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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.

Original publication

DOI

10.1136/thoraxjnl-2013-203309

Type

Journal article

Journal

Thorax

Publication Date

11/2013

Volume

68

Pages

1075 - 1077

Keywords

Cystic Fibrosis, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Double-Blind Method, Follow-Up Studies, Genetic Therapy, Humans, Prospective Studies, Treatment Outcome