Techno
For the first time, a CRISPR drug treats a child’s unique mutation

WAyen days after Kj I was born in Philadelphia in August 2024, it was clear that there was something wrong. He did not eat and sleep too much. Blood tests have revealed levels in the sky of ammonia, which is a toxic substance that usually expels the body. The genome sequence confirmed that he has a rare genetic disease called Carbamoyl-Pehosphate Synthetase 1 (CPS1) The deficiency, which is often killed in childhood, which is not good newborn treatment. Then one of his doctors suggested a fundamental thing: genetics is specially designed for him.