In a medical breakthrough, a baby diagnosed with a rare and fatal genetic disorder is recovering well after receiving a first-of-its-kind personalized gene editing therapy.

KJ Muldoon, born with CPS1 deficiency, a condition affecting about one in a million infants, was treated using a cutting-edge base editing technique developed by doctors at Children's Hospital of Philadelphia and Penn Medicine.

The condition prevents the body from removing toxic ammonia, often requiring a liver transplant.

KJ's parents chose the experimental route over surgery, and within six months, doctors created a CRISPR-based treatment tailored to correct his faulty gene.

Delivered via lipid nanoparticles, the therapy flips a single DNA base to restore normal function without cutting the DNA strand.

Following multiple doses, KJ has shown significant improvement, now eating normally and requiring less medication. Though still in early stages, the success offers hope for the 350 million people globally suffering from rare diseases.

Researchers say this custom therapy could pave the way for similar treatments in the future, with time and cost efficiency improving as technology advances.

This may be a turning point in personalized medicine.