The Pharmaceutical Journal
Vol 271 No 7270 p484
11 October 2003

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New England Journal of Medicine abstract (more)

Topical gentamicin corrects cystic fibrosis mutation

Applying topical gentamicin to the nasal epithelium of patients with cystic fibrosis can correct one of the mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that causes the disease. Researchers found that, after treatment, electrophysiological abnormalities caused by the CFTR defect resolved in 21 per cent of patients and chloride or sodium transport was restored in 68 per cent (New England Journal of Medicine 2003;349:1433).