• Question: Could we exploit natural epigenetic mechanisms to cure genetic diseases?

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

      Barbara Shih answered on 24 Mar 2015:

      Yes, potentially – For example, they are already looking into using drugs to induce epigenetic changes to treat Angelman syndrome, a genetic disorder resulted from the deletions or mutations in the maternal copy of a gene called UBE3A. Due to genetic imprinting, only the maternal copy of the gene is expressed in the brain, even if the individual have a normal paternal copy of the gene. Scientists attempted to unslience the paternal copy in the hope that it would compensate for the faulty maternal copy, and showed some levels of success in mice.
      (for more information:
      http://sfari.org/news-and-opinion/conference-news/2013/angelman-syndrome-drug-shows-promise-in-mouse-study )