• Question: What Treatments Are currently going for cystic fibrosis??

    Asked by muqy101 to Matt on 10 Mar 2015.
    • Photo: Matthew Moore

      Matthew Moore answered on 10 Mar 2015:

      Hi!

      Sorry I missed this during the chat.

      Cystic Fibrosis (CF) is caused by a mutation in the CFTR gene and is a recessive disorder. This means that to have cystic fibrosis you must inherit two mutated (faulty) versions. If you inherit a working and fault version then you won’t have CF as the working one will be able to function still!

      At the moment the leading cause of death in CF is bacterial infection of the lung and in particular, Pseudomonas aeruginosa which I work on. Antibiotics especially have extended the lifespan from about 5 years to about 40 normally. Other than treatment for infection a lot of physiotherapy to clear thick mucus from the lungs is required.

      Gene therapies were developed which literally place a working version of the gene into the cells, this would ‘cure’ CF, it would completely treat it as the patient would have a working version of the protein that the CFTR gene produces. Unfortunately the lung is a complex environment and gene therapies have had little success, once scientists discovered how to better get this gene into the cells in the lung then this will work a lot better!

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