
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
EU delays signing of Mercosur free trade deal - 2
Underestimated Metropolitan Experience Urban communities On the planet - 3
Ways to track down the Right Criminal Legal counselor - 4
The Most Well known Online Entertainment Forces to be reckoned with of 2023 - 5
Court clears Beersheba assault suspect of link to Haymanut Kasau disappearance, extends detention
Avoid Slam: Clearing the Street for the Eventual fate of Standard Size Trucks
Vote in favor of your Number one Kind of Gems
Photos of amputees in Gaza, struggling to survive after losing limbs to Israeli airstrikes
Vote In favor of Your Number one Sort Of Blossoms
Go on A Careful spending plan: Modest Objections for Your List of must-dos
Electric discovery on Mars! Scientists find tiny lightning bolts coming from Red Planet dust clouds
Scientists find evidence that an asteroid contains tryptophan
Instructions to Guarantee Kids Foster Solid Dental Propensities
The Leonid meteor shower is peaking early this week. Here’s what to know













