
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
Move. Cheer. Dance. Do the wave. How to tap into the collective joy of 'we mode'22.11.2025 - 2
I traveled to 13 countries in 2025. This small island nation surprised me the most.31.12.2025 - 3
The Response to Self-improvement: Embracing a Development Outlook07.07.2023 - 4
Drones, physics and rats: Studies show how the people of Rapa Nui made and moved the giant statues – and what caused the island’s deforestation26.11.2025 - 5
Southern Californians, your health insurance costs could rise in 202623.12.2025 - 6
New COVID-19 variant 'Cicada' is spreading. What to know about BA.3.2.25.03.2026 - 7
Israel Police arrest twenty-one as anti-war protests grow despite broad support for Iran war30.03.2026 - 8
Full SNAP benefits must be paid ‘promptly,’ USDA tells states as government reopens13.11.2025 - 9
Clovis Unified students spend spring break traveling through China02.04.2026 - 10
Factbox-Weight-loss drug developers line up to tap lucrative market as competition heats up23.12.2025 - 11
Dinosaur collagen used to create one-of-a-kind handbag02.04.2026 - 12
Stolen Renoir, Cézanne and Matisse were probably uninsured, market sources say02.04.2026 - 13
Transcript: NASA's Jared Isaacman on "Face the Nation with Margaret Brennan," April 5, 202605.04.2026 - 14
Thousands of Walgreens nasal spray bottles recalled. See which ones.26.11.2025 - 15
Top 15 Supportable Design Brands Coming out on top07.07.2023
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


































