
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
Some super-smart dogs can pick up new words just by eavesdropping - 2
A Past filled with Old Civilizations: The World's Most established Societies - 3
Careful Connections: Building Association and Trust - 4
Christmas 2025 skywatching guide: What you can see in the night sky on Dec. 25 - 5
Study casts doubt on potential for life on Jupiter's moon Europa
Popular Film Areas: A Worldwide Manual for Film Enchantment
10 Energizing Vocations in the Innovation Business
Family Holiday spots
Share your pick for the miniature headphones that you generally suggest!
Astronauts' brains change shape and position after time in space, study finds
Father and son spending Christmas together after health scares
Longtime United Launch Alliance CEO Tory Bruno resigns from space company. 'Finished the mission I came to do.'
Robyn returns to music with 'Dopamine,' her 1st single in 7 years: 'Came to save music once again'
Paratroopers kill terrorist who threw rocks at Israeli citizens, soldiers near Ofra in West Bank












