
The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas.
KJ Muldoon is walking and getting ready to celebrate the holiday season at home with his parents and three siblings.
KJ was born last year with a genetic disorder called carbamoyl-phosphate synthetase 1 deficiency. Also known as CPS1 deficiency, the metabolic condition affects only 1 in 1.3 million babies and often leads to life-threatening outcomes.
When he was 6 months old, doctors began giving KJ a groundbreaking new treatment -- a personalized CRISPR gene editing therapy at Children's Hospital of Philadelphia.
Doctors save baby's life with first-ever gene fix for deadly rare disease
The gene-editing therapy works like a "molecular spell-checker,” finding and fixing the specific genetic error causing his condition.
After three infusions and after spending the first 10 months of his life in a hospital, KJ was discharged and sent home in June.
Baby saved by gene-editing therapy 'graduates' from hospital, goes home
Since then, KJ has continued to grow and thrive with his family, celebrating his first birthday at home and taking part in one of the family’s favorite rituals -- cheering for their beloved Philadelphia Eagles.
Doctors hope new technology like CRISPR gene-editing therapies can be the key to treating more than 7,000 rare diseases, such as sickle cell disease, cystic fibrosis, Huntington’s disease and muscular dystrophy, that affect approximately 30 million people across the country.
LATEST POSTS
- 1
Can ICE agents detain U.S. citizens? What powers do they have to arrest people? Your most common questions answered. - 2
Chinese astronauts’ return to Earth delayed over fears spaceship damaged by debris - 3
Lily Allen 2026 'West End Girl' Tour: How to get tickets, prices, presale info and more - 4
Russia Establishing Long-Range Drone Bases In Belarus, Warns Ukraine - 5
Poland identifies two Ukrainian suspects in railway sabotage blast
If everyone on Earth sat in the ocean at once, how much would sea level rise?
Italy's Beloved Trevi Fountain Hides A Unique Secret That Can Be Explored Underground
35 million tons of food go to waste yearly in the US. Experts share tips to help stop it
'Yellowstone' made him a fan favorite. His biggest role isn't the one you see.
Eating ultra-processed foods could raise precancerous polyp risk for women under 50, according to research
Rachael Ray is navigating grief this holiday season. She doesn't have time for 'negative energy' on the internet.
Satellite space quiz: What's orbiting Earth?
Going with Children: Tips for Tranquil Family Get-aways
Two UN peacekeepers killed in explosion in Lebanon













