Lancet. 1999 Mar 20;353(9157):947-54.

Double-blind randomised placebo controlled trial, parallel design

Mean age 26.9 years. Confirmed CF, FEV1 >70%, sterile. 16 participants (all males).

Single dose of CFTR DNA+liposome, or liposome alone nebulised to lungs.

Adverse events, gene expression, CFTR protein expression, airway potential difference.

Seven of the eight patients receiving the active complex reported mild influenza-like symptoms that resolved within 36 h. Six of eight patients in both the active and placebo groups reported mild airway symptoms over a period of 12 h following pulmonary administration. No specific treatment was required for either event. Pulmonary administration resulted in a significant (p<0.05) degree of correction of the chloride abnormality in the patients receiving active treatment but not in those on placebo when assessed by in-vivo potential difference and chloride efflux. Bacterial adherence was also reduced. We detected no alterations in the sodium transport abnormality. A similar pattern occurred following nasal administration.

Cationic-lipid-mediated CFTR gene transfer can significantly influence the underlying chloride defect in the lungs of patients with cystic fibrosis.

A, E, K vitamins supplementation

Antioxidants in cystic fibrosis

Calorie supplements in cystic fibrosis

Omega-3 fatty acids, zinc and probiotics supplements in cystic fibrosis

Pancreatic enzyme replacement therapy in cystic fibrosis

Vitamin D supplementation