• Question: How does human genetic modification affect the patient?

    Asked by natxmas18 to Judith, Heather, Gemma, Ed, Bobby on 1 Feb 2019.
    • Photo: Judith Sleeman

      Judith Sleeman answered on 1 Feb 2019:

      If, by human genetic modification, you mean gene editing: if that is developed and allowed, the idea would be to correct a faulty gene that causes a disease, without altering any of the other genes in the patient. So, in theory, they would be the same person but without the disease. It would probably have to be done in an early embryo, though, as editing large numbers of cells in a person who already had a disease would be very challenging and might not reverse damage already done.

      If you mean gene therapy, there are already some approaches to this that are quite well developed for diseases such as Cystic Fibrosis and Spinal Muscular Atrophy. For these, the gene is put into cells of the patient, where it can work, but it doesn’t become part of the patient’s DNA so isn’t passed on to their children. It’s used like a drug.