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A Randomised, Double-blind, Placebo-controlled Phase IIB Clinical Trial of Repeated Application of Gene Therapy in Patients with Cystic Fibrosis

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Journal Thorax
Date 2013 Mar 26
PMID 23525080
Citations 24
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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.

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