A new gene therapy for an extremely rare disorder has offered two Canadian children a new chance at life, allowing them to move on their own after years of being trapped within their own bodies.
  • mtumishiA
    2•4 years ago

    Fascinating stuff imagined more than 25 years ago.

    How the new gene therapy works is that the genes needed to restore movement are actually put into AADC adenoviruses, and then injected deep into the brain. Once there, the DNA is taken up by cells, and starts correcting the defect.

    …

    Bankiewicz is a professor and the director of the Brain Health and Performance Center at the Ohio State University College of Medicine. He described the first ideas that would eventually become this gene therapy in a paper 25 years ago.

  • @mvuviA
    1•4 years ago

    Science and technology are not value neutral. It is driven by ideas of value, in this case profit:

    But although this new therapy is extremely promising, there are still numerous barriers to delivering this care, one of them being cost. Therapies for rare diseases aren’t profitable, so all this is funded by donations, fund raising and charities, with many other children waiting for treatment.