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.
Publication types
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Clinical Trial, Phase II
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Comparative Study
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Randomized Controlled Trial
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Research Support, Non-U.S. Gov't
MeSH terms
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Cystic Fibrosis / therapy*
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Cystic Fibrosis Transmembrane Conductance Regulator / genetics
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Cystic Fibrosis Transmembrane Conductance Regulator / therapeutic use*
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Double-Blind Method
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Follow-Up Studies
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Genetic Therapy / methods*
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Humans
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Prospective Studies
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Treatment Outcome
Substances
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Cystic Fibrosis Transmembrane Conductance Regulator