- KONTAKT
- AFS
- Business
- Bussiness
- Car
- Career
- Celebrity
- Digital Products
- Education
- Entertainment
- Fashion
- Film
- Food
- Games
- General Health
- Health
- Health Awareness
- Healthy
- Healthy Lifestyle
- History Facts
- Household Appliances
- Internet
- Investment
- Law
- Lifestyle
- Loans&Mortgages
- Luxury Life Style
- movie
- Music
- Nature
- News
- Opinion
- panorama
- Pet
- Plant
- Politics
- promis
- Recommends
- Science
- Self-care
- services
- Smart Phone
- sport
- Sports
- Style
- Technology
- tire
- Travel
- US
- World

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.
NEUESTE BEITRÄGE
Ähnliche Artikel
Cannabis reclassification could 'open the floodgates' for research, scientists say
Factbox-Weight-loss drug developers line up to tap lucrative market as competition heats up
Flu season is ramping up, and some experts are "pretty worried"
FDA approves Wegovy pill for weight loss: What to know
Raw oysters linked to ongoing salmonella outbreak infecting 64 across 22 states: CDC
FDA approves Wegovy pill for weight loss
I’m a doctor. Here are 10 science-backed tips to help you get healthier.
Flu season is ramping up, and some experts are "pretty worried"
How stripping diversity, equity and inclusion from health care may make Americans sicker
Doctor's orders? ‘Belly laugh at least two to five days a week'














