
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
Exhaustive Experiences into Prudent Senior Living in the UK - 2
Russia accidentally destroys its only way of sending astronauts to space - 3
Step by step instructions to Explore Assessment Ramifications of Disc Rates - 4
Native artists in Texas and Mexico shared their vision of the universe for 4,000 years, ancient murals suggest - 5
Staggering Spots to Stargaze All over the Planet
Nature's Treats: 10 Organic products That Lift Prosperity
Picking the Right Pot for Your Plants: An Aide for Plant Devotees
Nordic people know how to beat the winter blues. Here's how to find light in the darkest months
Top Music and Dance Celebration: Which One Gets You Going?
Shah Capital pushes for Novavax sale, warns of proxy fight
Lahav 433 head Asst.-Ch. Meni Benjamin named as police officer investigated for breach of trust
Lift Your Style: Famous Hairdos for Ladies
They want better health care — so they're turning to crypto startups
Figure out How to Pick the Right Dental specialist for Your Dental Inserts













