In a medical first, doctors raced to create a bespoke CRISPR gene therapy for a boy born with a deadly genetic disease and delivered it to him a mere six and a half months after birth.
The CRISPR treatment was administered safely, and the infant is now growing well and thriving, his doctors say. They hope that the treatment will make a liver transplant, which is usually required for survival, unnecessary for him.
The boy inherited two mutated genes that prevented him from breaking down the proteins in his food, which leads to a build-up of ammonia that destroys the liver and...