Opal Sandy, aged 18 months, was born completely deaf due to condition auditory neuropathy

Opal Sandy, aged 18 months, was born completely deaf due to condition auditory neuropathy

  • witty_username@feddit.nlEnglish
    5·
    7 months ago

    Great achievement. Not much on the tech though. According to the ucam press release, they used an AAV1 viral delivery system. Given that the therapy is permanent, I would expect integration of the therapeutic gene into the patient’s genome (in the targeted tissue). Anyone have more on this?

    https://www.cam.ac.uk/research/news/baby-born-deaf-can-hear-after-breakthrough-gene-therapy

    Edit: I think this patent may be relevant
    https://www.patentsencyclopedia.com/app/20130095071

      • hallettjEnglish
        3·
        7 months ago

        I don’t know anything about the treatment. But to be hereditary the genome changes would have to affect gametes. If genes are only changed in other targeted tissue then the changes wouldn’t be inherited.

      • witty_username@feddit.nlEnglish
        3·
        7 months ago

        No
        The gene therapy is applied locally. They operate on the ear to enable access to the relevant tissue and then apply the therapy. So it should only affect cells in the ear.
        Additionally, it is unclear to me whether the therapeutic gene is integrated into the genome. It might not be.
        For transmission to offspring, you would need to edit germ line cells (sex cells) and you would need to integrate the gene into the genome.