US EditionFirst Personalized Gene Therapy Using Base Editing Shows Promising Results in Baby with Rare Disorder
- In 2025, researchers from Penn Medicine and the Children's Hospital of Philadelphia developed and administered a personalized gene editing treatment to baby KJ Muldoon in Clifton Heights, Pennsylvania, addressing his severe CPS1 deficiency.
- KJ received a diagnosis soon after being born of a rare and often deadly genetic disorder that causes hazardous ammonia accumulation, leading to the rapid creation of a CRISPR-based treatment.
- The customized treatment corrected KJ's faulty gene using CRISPR delivered by lipid nanoparticles starting in February 2025, enabling him to tolerate more protein and recover from common illnesses.
- Dr. Kiran Musunuru noted that this marks an initial milestone in applying gene editing techniques to develop treatments for numerous uncommon genetic conditions that currently lack effective medical options.
- This successful case marks a promising advance for personalized gene therapy and suggests that individualized treatments could become scalable and help many rare disease patients.
332 Articles
332 Articles
Behind the Headlines, Episode 17: Bespoke CRISPR Therapy, Executive Order Fallout, and More
Jamie Baumgartner, Jonathan Grinstein, and John Wilkerson go behind the headlines to discuss the implications of personalized gene-editing therapies, more HHS policy and funding updates, and the latest tariff-related investments.
In the United States, a baby with a rare and life-threatening disease was treated 7 months after being born with a gene therapy specially designed for him. A big step towards personalized medicine analyzed by the General Manager of Genethon, Frédéric Revah.
Metro family embraces life with baby battling rare, fatal genetic disorder
OLATHE, Kan. — Five months ago, Christina and Adam Hannan were celebrating the arrival of their sixth child, a beautiful baby girl named Annalise. But the joy of her birth quickly turned to heartbreak. Annalise wasn’t crying. She wasn’t moving. Something was wrong. KC-area emergency management on staying prepared ahead of severe weather Doctors diagnosed her with congenital hypomyelinating neuropathy, a condition so rare that fewer than 50…
Gene editing helped a desperately ill Delaware County baby thrive. Scientists say it could someday treat millions
Researchers described the case in a new study, saying the baby is among the first to be successfully treated with a custom therapy that seeks to fix a tiny but critical error in his genetic code that kills half of affected infants. A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. Researchers described the case in a new study, saying he’s among th…
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