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
      1·
      17 days ago

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

    • JasSmith@sh.itjust.worksEnglish
      0·
      18 days ago

      Yeah I don’t see how this won’t happen now. As soon as parents are given the option to prevent systemic genetic diseases like congenital blindness, the next question will be, “do you want them to be a bit smarter?” Very few parents are going to say no.

      • ZILtoid1991@lemmy.worldEnglish
        1·
        18 days ago

        The catastrophe will be when we realize yet again that the human genome is not like a config file.

      • muusemuuse@lemm.eeEnglish
        1·
        18 days ago

        The problem is intelligence doesn’t automatically mean better life, especially not in a country like the US.

        • JasSmith@sh.itjust.worksEnglish
          01·
          17 days ago

          It is the single greatest correlate for life outcomes. Higher income, longer lifespan, lower addiction, higher employment, higher wealth, lower crime, better physical health on every metric, and lower rates of fatherlessness. These effects all compound the next generation too. There is nothing else in sociology which comes even close to IQ in predicting life outcomes. Not income, race, location, education, or fatherlessness.

          Of course nothing guarantees a “better” life.

          • Lumiluz@slrpnk.netEnglish
            1·
            17 days ago

            Well that’s just bullshit and I looked it up to be sure it was.

            Income is the best life outcome. I did find that high IQ is more correlated with depression and suicide however.

      • Kayel@aussie.zoneEnglish
        0·
        18 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
          1·
          18 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.