
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
How to identify animal tracks, burrows and other signs of wildlife in your neighborhood - 2
$2,000 tariff rebate checks? 50-year mortgages? Making sense of Trump's new 'affordability' proposals. - 3
Find the Lively Food Markets of South America - 4
Experience Arranging: Planning for Epic Excursions - 5
Watch the Geminid meteor shower peak tonight from the comfort of home with this free livestream
NASA's SPHEREx telescope completes its 1st cosmic map of the entire sky and it's stunning!
A top Marine shares his secrets to keeping fit at 50
PA accuses Israel of 'human trafficking' after planeload of Gazans arrives in South Africa
We may be witnessing the messy death of a star in real time
See the moon shine with Saturn in the southern sky after sunset Dec. 26
When does Spotify Wrapped come out? The music streamer says 'soon.'
Find the Effect of Web-based Entertainment on Society: Exploring the Computerized Scene
Jesse Jackson hospitalized, under observation for a neurodegenerative condition
This Week In Space podcast: Episode 187 — An Inspired Enterprise












