Repeated application of gene therapy in cystic fibrosis patients Background and study aims Cystic fibrosis (CF) is a common genetically inherited disease caused by mutations in the CFTR gene. Mutations in this gene lead to thick sticky mucus clogging the lungs, in...

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Longitudinal assessment of clinical measurements in patients with cystic fibrosis in preparation for a clinical trial of CFTR gene therapy Not provided at time of registration

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