• Question: What are the advantages and limitations of new gene therapy/modification drugs, such as Glybera?

    Asked by Henry to Barbara on 24 Mar 2015.
    • Photo: Barbara Shih

      Barbara Shih answered on 24 Mar 2015:

      The advantages include:
      – the virus used for the therapy (adeno-associated virus serotype 1) does not appear to cause disease
      – can be delivered to non-dividing cells
      – because the viral DNA is free floating rather than being combined with the human chromomesomes, there is a smaller risk of the virus randomly insert its DNA into human genes (the problem with virus, such as retrovirus, randomly insert its DNA into human gene is that their insertion could be in the middle of important genes, causing mutations that lead to cancer.
      – the virus induce mild immune response

      However, the disadvantages include:
      – the virus can only insert genes less than 4800 base pairs gene, which is pretty small
      – patients would need to take immunosuppressive drugs, which can have a range of side effects (such as more prone to infections, cancer cells able to evade detection from the immune system…etc.)