For the first time, scientists said they reached into the genome of a really sick kid and changed the unique misspelling in his DNA. It was a big deal, like seriously huge, and it was all published in the New England Journal of Medicine on Thursday. The boy, now 9.5 months old and called KJ, was diagnosed right after birth with this super rare disease that messed up his liver’s ability to deal with ammonia. And that ammonia can really mess up your brain or even kill you.
KJ had been stuck in the hospital, waiting until he was old enough to get a liver transplant. But then, at 6 months, the doctors decided to try this gene editing treatment on him. And guess what? It actually worked! They were able to relax his super strict low-protein diet and cut his ammonia-lowering meds in half. Plans for a transplant are on hold for now, but everyone’s still kind of holding their breath to see how things go.
The results are a glimmer of hope for thousands of patients with these deadly mutations that are just too rare for big drug companies to care about. But, you know, it’s not all rainbows and sunshine. There are some big questions about how to make these personalized treatments safe and fair for everyone. And, like, how do we even pay for all this genetic medicine stuff? It’s a lot to think about, man.
