• Question: What are your views on the effects that gene therapy can have on medicine, and do you belief that the medicinal effects outweigh the social drawbacks ?

    Asked by connorf2212 to Ryan, Marianne on 7 Jun 2019.
    • Photo: Marianne King

      Marianne King answered on 7 Jun 2019:


      I think gene therapy has huge potential, particularly for diseases that are caused by mutations in only one gene. Rett Syndrome for example is caused by mutations in the MeCP2 gene, which affects brain development and so many other things. It has been shown that in mice with the same mutation, replacing the gene with a healthy MeCP2 gene almost abolished all their symptoms. Diseases caused by problems in multiple genes might be more complicated, but you never know what science and research will be able to do in the future. I believe issues could arise if we start to meddle in human genes for no good reason, for example if you want your child to have brown hair. This I think would be a very negative slope and should be avoided. But gene therapy for helping to treat disease is something I think is really exciting.

    • Photo: Ryan Beveridge

      Ryan Beveridge answered on 10 Jun 2019:


      Gene therapy will transform medicine. The ability to introduce new genes into cells that either have defective genes or completely new ones has already changed the way we look at medicine.

      There are two examples that, for me, are truly extraordinary. The first is CAR-T cells, this technique is used to train a patients own cells to see cancer and kill it. As cancer is a disease of self, that means its part of your own body gone wrong, your own immune system that normally deals with sickness doesn’t often do very well with cancer. During CAR-T therapy doctors take your blood, purify the important immune cells and teach them to see the cancer by giving them a new receptor. They then grow them up so there are enough of them to be useful and inject them back into the patient. The results from clinical trials are amazing and its started being used in clinics just last year.

      The other is from an Italian group that have found a way to help children whose skin does not attach well at all, it can fall off just by touching it. That is because they don’t have the gene that allows the skin to stay put. What these scientists did was take skin samples from the child, introduce a working copy of the gene and, as with CAR-T therapy, grow them up. However, this time they are growing sheets of skin that can be grafted onto the child. Again the results were amazing, the poor little boy had hardly any skin left when they started but is now out of hospital and doing well.
      These are just two examples but their are lots and lots of new applications! One of the biggest challenges will be being able to make enough of the materials required for gene therapy at a cost that allows it to be used widely.

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