Gene-editing breakthrough has potential to treat array of devastating genetic diseases soon after birth, scientists say

Doctors in the US have become the first to treat a baby with a customised gene-editing therapy after diagnosing the child with a severe genetic disorder that kills about half of those affected in early infancy.

KJ was born with severe CPS1 deficiency, a condition that affects only one in 1.3 million people. Those affected lack a liver enzyme that converts ammonia, from the natural breakdown of proteins in the body, into urea so it can be excreted in urine. This causes a build-up of ammonia that can damage the liver and other organs, such as the brain.

Writing in the New England Journal of Medicine, the doctors described the painstaking process of identifying the specific mutations behind KJ’s disorder, designing a gene-editing therapy to correct them, and testing the treatment and fatty nanoparticles needed to carry it into the liver. The therapy uses a powerful procedure called base editing which can rewrite the DNA code one letter at a time.

    • Match!!@pawb.socialEnglish
      5·
      1 day ago

      the trumpists are attempting eugenics the old fashioned way, gattaca would be an upgrade

    • azuth@sh.itjust.worksEnglish
      6·
      3 days ago

      A baby (and more people after) not dying is an afterthought to you, you are mostly concerned with a fucking SciFi flick not coming true somehow.

      Grow up, seriously.

      This tech will save and improve a lot of peoples’ lives. It will be also be used frivolously by the rich to ‘improve’ (or actually fuck up) themselves just like they use current medical tech. Meanwhile some poorer people that need it to survive will be denied it.

      These are political issues, long existing, not problems with this medical development.

      • Kayel@aussie.zoneEnglish
        5·
        2 days ago

        I would suggest they’re more in touch with the science. It haunts science, we know we must do it for the benefit of humanity, but with the knowledge - the powerful and capital will use it for themselves.

        • azuth@sh.itjust.worksEnglish
          2·
          2 days ago

          I would suggest they’re more in touch with the science.

          How so?

          It haunts science, we know we must do it for the benefit of humanity, but with the knowledge - the powerful and capital will use it for themselves.

          The solution is not halting technological advancement(especially medical technology) but to try and change our political systems.

      • Kayel@aussie.zoneEnglish
        5·
        2 days ago

        Based on what? The US discussing legalising genetic experimentation for military uses is the pathway to Gattaca.

        Not saying you’re wrong, curious about your time-line

        • ouRKaoS@lemmy.todayEnglish
          6·
          2 days ago

          Just a general timeline for technology to become ubiquitous. Starts small, for the rich or “for nerds”; begins to take over, now seen as “normal”; eventually you’re the weird one for not having it.

          Microwave ovens & the Internet are the two I can think of in my lifetime, I’d say an average of about 20 years for the cycle to complete, so about a generation.

    • Nangijala@feddit.dkEnglish
      5·
      2 days ago

      You know what? You’re right. Let’s cancel medicine because it is eugenics. Let nature take care of those who are too weak to live. Fuck em.

      Fuck vaccines, fuck lifesaving surgery, fick meds, fuck gene-editing that can save a baby from a life of suffering due to severe genetic disorders.

      Fuck them all. We wouldn’t want to change anything for anyone because that might make some ignorant Karen think that medicine is eugenics.

    • Match!!@pawb.socialEnglish
      3·
      1 day ago

      they aren’t even altering the kid’s gonads, just the liver, so his ability to pass on that gene is unaffected for future generations