• Question: How close do you think we are to using gene therapy, in an effactive and safe manner?

    Asked by jackyboy1999 to Clare, Mariana, Robert, Susanne on 21 Nov 2012.
    • Photo: Robert Insall

      Robert Insall answered on 21 Nov 2012:


      Hey Jackyboy

      I think we are very close indeed, for a very limited range of diseases etc. – but the trouble is, we aren’t that good at it yet. Fore some diseases (for example cytsic fibrosis) just fixing the gene should fix the disease. For cancer the problem is much more to do with the balance of genes, so we can’t just fill poeple up with gene therapy and expect it to work…

    • Photo: Mariana Campos

      Mariana Campos answered on 21 Nov 2012:


      I think so. Check this out: “A treatment which corrects errors in a person’s genetic code has been approved for commercial use in Europe for the first time.”. http://www.bbc.co.uk/news/health-20179561.
      But as Robert said, this is close to become reality for a very limited range of diseases, that have genetic alterations that are simple and that we understand.

    • Photo: Susanne Muekusch

      Susanne Muekusch answered on 21 Nov 2012:


      It has been successfully done for diseases like SCID (severe combined immunodeficiency). However, there is a risk to develop leukemia as a consequence of the treatment. That is why I am not so sure about the “safe” in your question. But still, it is a great achievement. I agree with Clare and Robert that it will work for some diseases only.

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