Outcome measures for cystic fibrosis gene therapy clinical trials

Background: Cystic fibrosis (CF) is a life-shortening, chronic respiratory disease caused by mutations in the CFTR gene. Novel therapeutic agents such as gene therapy aim to correct CFTR and to demonstrate evidence of molecular, functional and (ultimately) clinical efficacy. It was hypothesised that...

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Bibliographic Details
Main Author: Davies, Gwyneth
Other Authors: Davies, Jane C. ; Alton, Eric W. ; Bush, Andrew ; Rosenthal, Mark
Published: Imperial College London 2013
Subjects:
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Online Access:http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.676746