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A glimmer of hope for the thousands of families affected by cystic fibrosis has emerged from a clinical trial showing that gene therapy produced a modest but significant improvement among children suffering from the most common inherited disease of white Europeans.
Scientists in Britain said it was the first time in more than 20 years of research on the cystic fibrosis gene that anyone has shown definite advantages of gene therapy - the deliberate attempt to correct the defective gene behind cystic fibrosis, which primarily affects the lungs.
However, the members of the UK Cystic Fibrosis Gene Therapy Consortium believe they have now made the key initial breakthrough that allows them to proceed with further clinical trials involving more active forms of the gene-therapy treatment.“We are looking to undertake follow-up studies assessing higher, more frequent doses as well as combinations with other treatments,” Professor Alton said.
The latest trial involved 40-minute inhalations once a month for year with a nebulising spray containing fatty droplets or “liposomes” wrapped around a healthy, synthetic copy of the cystic fibrosis gene.
One in 25 Caucasians are carriers of a CF gene mutation.
If a person who is a CF carrier has partner who is also a carrier, they have a 1 in 4 chance of having a child with CF.
This causes incomplete digestion and malabsorption which can affect the growth of the baby and child.
The liposomes are designed to be absorbed by the cells lining the airways of cystic fibrosis patients, where the gene stimulates the production of the healthy protein in the cell membranes that prevents the the lungs from clogging with sticky mucus.
Results of the trial published in the journal The Lancet Respiratory Medicine revealed that there was about a 5-per-cent improvement in lung function among the treated patients, which rose to about 6 per cent in the worst-affected sub-group.
Cystic fibrosis is caused by a faulty gene that a child inherits from both their parents.
The faulty gene affects the movement of salt and water in and out of cells.
This, along with recurrent infections, can result in a build-up of thick, sticky mucus in the body's tubes and passageways – particularly the lungs and digestive system.