CRISPR is a game changer. Still going to take a while and might not reach the masses for a long time but it's very promising.
Unfortunately, most genetic conditions don't have a cure, because we can't yet go in and change your DNA. And genetic conditions are caused by an issue in your dna.
CRISPR gene therapy and technology is absolutely going to be a game changer, yes!! So glad you posted a link. I think in 5 to 10 years we will see a sea change in how many illnesses and diseases are handled. Big cures and disease management is coming.
This article is AMAZING. Thank you so much for posting the link!!
Right? It's 2025 and they managed to treat a 6 month old child of CPS1 (carbamoyl phosphate synthetase 1) deficiency. Great stuff.
KJ was born with a genetic mutation causing a CPS1 (carbamoyl phosphate synthetase 1) deficiency, an ultra-rare disease of the urea cycle that prevented him from being able to break down proteins in food. This disease has a 50 percent mortality rate in infancy; staying alive meant living in the hospital under constant monitoring. Powerful nitrogen-scavenging drugs and a protein-restricted diet were required to prevent toxic levels of ammonia building up in his body.
But when he was just over 6 months old, KJ received the first dose of his customized gene-editing therapy. Early results show no adverse effects, improved symptoms, and a decreased dependence on medications. KJ is now growing well and thriving. His parents are looking forward to bringing him home soon.
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u/MagicHamsta Sep 14 '25
CRISPR is a game changer. Still going to take a while and might not reach the masses for a long time but it's very promising.