First we had general-purpose gene editing to treat (and cure) diseases based on singular genetic mutations, such as sickle cell anemia. And that was already a big deal. Personalized gene editing kept being an illusive goal, but now it’s here. A baby (KJ) was successfully treated for a rare genetic disorder which left his body unable to remove toxic ammonia from his blood. It’s stil early days, but this could be the beginning of something big (and important).
KJ’s doctors will monitor him for years, and they can’t yet say how effective this gene-editing approach is. But they ~plan to launch a clinical trial to test such personalized treatments~ in children with similar disorders caused by “misspelled” genes that can be targeted with base editing.
