• Question: Is there a way to manipulate genes or DNA to stop genetic defects? How do you do it?

    Asked by to Lilly on 18 Mar 2014. This question was also asked by .
    • Photo: Lilian Hunt

      Lilian Hunt answered on 18 Mar 2014:


      At the moment not really, but almost! There are a lot of scientists working really hard to create ‘gene therapies’ where we manipulate the DNA in someone with a genetic disorder so they no longer have that mutation that causes it. There are some that are quite far down the line in human trials so could happen soon.

      We try and do it by delivering new DNA without the mutation into the cells of the the person with the genetic disorder. This can be really tricky as at the moment, we have to use things like viruses to deliver the DNA which can cause other problems. Also, trying to get the DNA to stay in the cells and replicate with the DNA already in that person can be difficult to, so often the changes aren’t permanent.

      Keep an eye out in the news as I think the most likely success stories will come from gene therapies to treat eye disorders. There have already been a few successful trials in this area 🙂

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