Trial is started to see whether gene therapy can improve lung function in cystic fibrosis

BMJ 2012; 344 doi: (Published 19 March 2012) Cite this as: BMJ 2012;344:e2141
  1. Zosia Kmietowicz
  1. 1London

A trial of gene therapy that aims to repair the underlying fault in people with cystic fibrosis will begin this month, involving 130 adults and children in the United Kingdom.

The trial, described as the largest of its kind, is coordinated by the UK Cystic Fibrosis Gene Therapy Consortium and funded by a £3.1m (€3.7m; $4.9m) grant from the National Institute for Health Research and the Medical Research Council.

Cystic fibrosis, the commonest inherited disease caused by a single mutation in a gene, affects around 9500 people in the UK …

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