
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
Step by step instructions to Deal with Your Time While Chasing after an Internet based Degree - 2
Spots to Go Hang Floating - 3
How Mars 'punches above its weight' to influence Earth's climate - 4
Record-breaking flu hospitalizations in New York in a single week: Health officials - 5
Opening Potential: Self-awareness and Long lasting Learning
The most effective method to Pick The Right Speakers
10 Activities to Lift Your Consume and Bust Your Stomach
Figure out How to Use Your Nursing Abilities for Better Compensation
Vote In favor of Your Number one Game Control center
Windows to the Previous: An Excursion Through the World's Notable Engineering
Blue Origin launches New Glenn rocket on company's first NASA-scale science mission
New research reveals urban raccoons across the US show early signs of domestication
The Most Vital Crossroads in Olympic History
Overhaul Your Rest: Tips for a Serene Evening













