UK CF Gene Therapy Trial: Positive Results

In the wake of the initial disappointment seen with adenovirus-delivered gene therapy for cystic fibrosis (Marketletters passim), the results of a UK trial have suggested that liposome-delivered therapy may emerge as a safer alternative to viral vectors. Published in the first edition of the new journal Nature Medicine, the study shows that a single administration of liposomes containing functional copies of the cystic fibrosis transmembrane regulator (CFTR) gene to the nasal lining partially corrected the defect for up to a week. CF is caused by inheritance of a defective CFTR gene, the gene product of which is involved in the regulation of chloride ions out of mucosal membranes.

The researchers, from the Royal Brompton and St Mary's Hospitals in London, the Medical Research Council's Human Genetics Unit in Edinburgh and the University of Pittsburgh in the USA, report that the results are as good as can be achieved using viral vectors (although higher doses of genes are required) and the treatment is extremely well-tolerated. Studies looking at administration to the lung are now planned.