
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
4 Dazzling And Well known Island Objections In US - 2
Avoid Slam: Exploring the Pickup Truck Transformation - 3
Opening Achievement: 8 Methodologies for Compelling Using time productively - 4
Why this Iranian island looks like Mars after it rains - 5
I'm an 83-year-old yoga instructor. I'm not your typical grandma — I still work to feel fulfilled and supplement my Social Security.
Jeff Bezos’ Blue Origin launches landmark Mars mission in New Glenn rocket’s first big test
Kids with smartphones by age 12 are at higher risk of health issues, study finds
Famous Versatile Brands: Your Decision
The Secret Side of Italy: 12 Underrated Destinations Locals Don’t Want Tourists to Find
6 Famous Urban communities for Shopping on the planet
Vote in favor of the Web-based Work out schedule to Keep You Fit and Sound
Would you ever turn to AI for companionship? 6% of Americans say they could — or already have.
It's been 20 years since MTV's golden couple split. These producers saw it all unravel.
Top Music and Dance Celebration: Which One Gets You Going?













