
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
NATO official says members often aren't buying weapons together, and it's a mistake29.03.2026 - 2
French high-speed train slams into truck, killing TGV driver07.04.2026 - 3
Watch interstellar comet 3I/ATLAS make its closest approach to Earth in free livestream on Dec. 1817.12.2025 - 4
Reactions as Artemis II astronauts lift off on historic lunar mission01.04.2026 - 5
West Palm Beach Shorecrest, renderings of downtown waterfront condo07.04.2026 - 6
Thousands of Walgreens nasal spray bottles recalled. See which ones.26.11.2025 - 7
Vial marked 'Polonium 210' sparks scare during German Easter egg hunt05.04.2026 - 8
Hundreds of kilos of protected plant seized by Israel Nature and Parks Authority in Golan29.03.2026 - 9
NI economy losing momentum due to Iran crisis26.03.2026 - 10
People are getting their news from AI – and it’s altering their views19.12.2025 - 11
Tehran synagogue damaged by missile strike according to Iranian media07.04.2026 - 12
‘We are the alternative’: Anti-Hamas Gaza militia tells BBC group is receiving international support21.11.2025 - 13
Pleasant Cycle Courses All over the Planet06.06.2024 - 14
Remote Headphones: Improve Your Sound Insight25.09.2023 - 15
Trump administration launches new immigration crackdowns in New Orleans and Minneapolis. Here are all the cities it has targeted so far.03.12.2025
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

































