• Question: do you think there is ever going to be a cure for cystic fibrosis ?

    Asked by ellie to Matt on 6 Mar 2015.
    • Photo: Matthew Moore

      Matthew Moore answered on 6 Mar 2015:

      Hi Ellie,

      Whether there could be a cure for cystic fibrosis is an interesting question and in short, yes there could be!

      Cystic fibrosis is an autosomal recessive disorder which means that somebody has to inherit two versions (alleles) of the mutated (faulty) gene. This means that a functioning version of the gene could actually be inserted and successfully ‘cure’ cystic fibrosis, the protein affected (CFTR) would now work normally.

      However, gene therapies for cystic fibrosis have been around for a while now and haven’t been successful in achieving this. Just because something works in the laboratory doesn’t mean it will work in a patient unfortunately. The lung is a complex environment and better methods of delivering the functional gene are being researched, which would effectively treat cystic fibrosis!

      For now, bacterial infections of the lung in cystic fibrosis are the primary cause of death. Careful treatment of infections massively increases life expectancy, which used to be about 5 years old and is now about 40.

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