• Question: Can you reengineer your dna

    Asked by 549genm52 to David, Gemma, Juhi, Matt, Stéphane, Yinka on 13 Mar 2018.
    • Photo: Stéphane Berneau

      Stéphane Berneau answered on 13 Mar 2018:

      In humans, it is difficult to re-engineer DNA once you are adults.
      Moreover, in human cells, DNA is stored in the nucleus, protected by a (nuclear) membrane which makes difficult to inject DNA into a cell for it to go to the nucleus.
      Cystic fibrosis is a lung disease. A new therapeutics involved breathing virus that has been genetically modified. Their DNA was replaced by the gene corrupted in cystic fibrosis patients.
      The virus will inject its DNA in the cytoplasm of pulmonary cells and help restore their function.
      However, maybe it is not present in the nucleus, this extra DNA will be degraded and not included in cell division. After few mitotic cell divisions, the viral-shared DNA will not be present and patients will have to come back for another inhalation.

      More info about cystic fibrosis:

    • Photo: Gemma Chandratillake

      Gemma Chandratillake answered on 13 Mar 2018:

      Yes, but it is easier to do this by growing engineered cells outside your body and then putting those engineered cells back. Here is a good example: http://www.sciencemag.org/news/2017/11/boy-rare-disease-gets-new-skin-thanks-gene-corrected-stem-cells

      If you want to do genetic engineering on the cells inside your body, the tricky thing is to get the correct DNA and the tools to do the engineering into the right cells in your body. This is done by using viruses (such as AAV) as vehicles to deliver both the correct DNA and the molecular tools that do the engineering to the correct cells in the body. There has been a recent report of such in vivo genetic engineering that got a lot of attention: http://www.sciencemag.org/news/2017/11/human-has-been-injected-gene-editing-tools-cure-his-disabling-disease-here-s-what-you
      This is a huge are of interest, the thing people are worried about is making sure that they only engineer the bit of DNA that they intend to, i.e. they are worried in case there are off-target effects that cause harm to the patient, but this is really exciting for patients with genetic diseases.

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