• Question: if you are a carrier of cystic fibrosis is there any way of getting rid of that gene?

    Asked by Dani Evaristo to Thomas, Ellen on 18 Jun 2016.
    • Photo: Thomas Biggans

      Thomas Biggans answered on 18 Jun 2016:

      Yes there is a way to remove the gene from cells. This isn’t my area of expertise but from what I know scientists are developing gene therapies that can change or delete specific genes that cause diseases such as cystic fibrosis. However, like all therapies, there are risks involved and there could be side effects, you also have to consider the cost. So at the moment I don’t think a carrier would be consider for this gene therapy which is still being trialed in patients who suffer from cystic fibrosis.

Comments

  • Photo: Elliot

    Elliot commented on :

    The question wasn’t directed to me, but I thought I’d insert a couple of points!

    There are some cool trials looking at using gene therapy for people who actually suffer from cystic fibrosis, so those are people who have two copies of the faulty gene. If we can fix a copy of the gene, or insert a healthy copy of the gene, we might be able to stop the harmful effects of the disease. This makes trying gene therapy worth it in people who are already suffering from the disease.

    Carriers have one faulty gene, and one healthy gene, which is enough to keep them from getting the disease. Since carriers don’t suffer any harmful effects, even if there was a way for us to fix the broken copy, it wouldn’t be worth the risk of any side effects of such a treatment.

    It might make sense to try fixing the gene in the germ cells (so the egg cells and the sperm cells) or in embryos to prevent a situation where you get two faulty copies of the gene. But I think it would be more straightforward to just test embryos for the disease when they are only a few cells big and select for embryos without faulty copies of the gene – however, there are many people think that selecting embryos like that is not very ethical. I think it would be a good thing if it prevents the disease. Do you have any thoughts?