Article

Outcome measures for cystic fibrosis gene therapy clinical trials

2013-01-01

Abstract excerpt

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 currently used methods to detect these changes may be optimised to enhance sensitivity and allow quantification, and...

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Literature Corpus work: b5204667-cba1-5fda-ab50-6cf5391db42f
DOI: 10.25560/28414

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