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Gene therapy breakthrough for cystic fibrosis


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Gene therapy breakthrough for cystic fibrosis


 

Date: 06/07/2015


“Cystic fibrosis hope as new gene therapy improves condition,” The Daily Telegraph reports. Researchers have, for the first time, managed to successfully "smuggle" healthy copies of genes into the lungs of people with cystic fibrosis.

Cystic fibrosis is a genetic condition caused by a mutated gene called CFTR. The mutation causes the lungs and digestive system to become clogged up with sticky mucus.

The goal of gene therapy for cystic fibrosis is to replace the faulty CFTR gene with a working one.

Previous attempts of using a virus to deliver the working gene proved unsuccessful, as the lungs’ defence system against infection stopped the virus from entering.

In this new study, the researchers tried a different approach – the gene was encased in a bubble of fat, which was then delivered to the lungs via a nebuliser.

When compared to placebo, the nebuliser-delivered approach showed a modest, but significant, improvement in lung function (3.7%).

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Source: NHS


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