
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
From Overpowered to Coordinated: Individual Accounts of Cleaning up - 2
There are thousands of aligned holes in Peru. Archaeologists now think they know who made them - 3
Can scientists detect life without knowing what it looks like? Research using machine learning offers a new way - 4
'Euphoria' releases Season 3 trailer, premiere date: Watch Rue and Laurie finally face off - 5
Tremendous Spelunking: Cool Caverns All over the Planet
Step by step instructions to Shield Your Wellbeing Around 5G Pinnacles\
The Electric Bicycle Americans Can Confide in 2024
A Manual for the Right SUV for Seniors
Figurine of a woman and a goose offers peek at prehistoric beliefs
Best Amusement Park in Asia: Which One Is a Must-Visit
Home Machine Basics: An Exhaustive Purchasing Guide
Is 'Veronica Mars' about to be your new binge-watch? It's now streaming on Netflix.
Man triggers smoke bomb during failed crypto robbery
74 suicide warnings and 243 mentions of hanging: What ChatGPT said to a suicidal teen










