
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
- 1
It Looks Like a Tiny, Fluffy Dragon, But It's Really a Bird. Meet the Great Eared Nightjar09.01.2026 - 2
Flash flooding causes highways to close to and from Eilat06.12.2025 - 3
The most effective method to Keep up with Proficient Handshakes in a Computerized World19.10.2023 - 4
$2,000 tariff rebate checks? 50-year mortgages? Making sense of Trump's new 'affordability' proposals.12.11.2025 - 5
'The Real Housewives of Rhode Island' 1st teaser trailer unveiled: Which Bachelor Nation star is part of the cast? And when does it premiere?17.11.2025 - 6
Building an Individual Brand: Illustrations from Powerhouses25.09.2023 - 7
Bitcoin momentum builds in Abu Dhabi as global interest surges05.12.2025 - 8
COGAT discovers motor oil hidden inside UN's humanitarian aid to Gaza in smuggling attempt25.03.2026 - 9
Reality TV star Spencer Pratt, who lost his home in Palisades Fire, is running for mayor of Los Angeles08.01.2026 - 10
The Iconic, Instantly Recognizable Plastic Chair That's Known All Around The World05.04.2026 - 11
Kate Middleton and Prince William unveil annual family Christmas card photo with George, Charlotte and Louis18.12.2025 - 12
Kids may be more likely to get the new ‘Cicada’ variant of Covid-19, scientists say. Here’s what to know about BA.3.202.04.2026 - 13
Compassion and Association: Building Significant Connections01.01.1 - 14
South Korea launches Earth-observation satellite on homegrown Nuri rocket26.11.2025 - 15
'Pluribus' release date: Everything you need to know about the new series from 'Better Call Saul's' co-creator06.11.2025
Ähnliche Artikel
Artemis 2 captures historic 'Earthset' photo | Space photo of the day for April 7, 202607.04.2026
Tesla Germany Registrations Quadruple to 9,252 Vehicles in Best March Ever07.04.2026
What's going on with Katseye? The Manon Bannerman hiatus drama, explained.07.04.2026
Two IDF officers, civilian face indictment in alleged Gaza aid-truck smuggling scheme07.04.2026
West Palm Beach Shorecrest, renderings of downtown waterfront condo07.04.2026
Artemis II astronauts make long-distance call to the space station as they head home from the moon07.04.2026
Tuesday, April 7. Russia’s War On Ukraine: News And Information From Ukraine07.04.2026
The Eastern Bongo, Kenya’s Rare Forest Antelope on the Brink07.04.2026
U.K. blocks Kanye West from entering Britain to headline now canceled festival: What led to the ban07.04.2026
Russia confirms 16 Cameroonian soldiers killed in Ukraine war07.04.2026
First Phosphate advances battery-grade phosphate project as analysts highlight strategic Federal support07.04.2026
After fleeing past Hezbollah fighting, some Israelis on northern border vow to stay07.04.2026
'Unreal' solar eclipse: Artemis 2 crew just saw one of the rarest sights in spaceflight history07.04.2026
Chinese construction workers in Israel: 'I’d rather be bombed than live in poverty'07.04.2026
Broken toilet, T-shirts on windows and collecting saliva: The weirdness of daily life aboard Orion07.04.2026
Czech Republic caps fuel prices amid Iran war energy crisis07.04.2026
NASA releases stunning new images captured by the Artemis II moon mission, including 'Earthset' and a solar eclipse from space07.04.2026
Amid Iran war, 53 of Israel's future scientists showcase projects in Jerusalem contest07.04.2026
Iranian-linked drone attack kills Kurdish couple in northern Iraq07.04.2026
Thousands of small fish defy gravity to climb Congo waterfall07.04.2026
































