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Infant With Incurable Disease is First to Successfully Receive Personalized Gene Therapy Treatment
- On May 15, 2025, medical teams from Children’s Hospital and the University of Pennsylvania successfully administered a tailored gene-editing treatment to infant KJ Muldoon, addressing his severe CPS1 deficiency.
- KJ's rare metabolic disorder prevents ammonia removal from protein breakdown and usually requires liver transplants with high infant mortality, prompting urgent gene therapy development.
- Researchers rapidly designed a CRISPR-based treatment targeting KJ's specific mutation, creating a bespoke therapy infused intravenously in late February and followed by two doses.
- Following therapy, KJ now tolerates more protein intake with reduced medication needs, and Dr. Ahrens-Nicklas said the therapy was “manufactured for an individual patient.”
- Though initial results are promising and mark a landmark in precision medicine, experts caution that long-term effects, regulatory changes, and economic challenges remain for widespread use.
13 Articles
13 Articles
Infant With Incurable Disease is First to Successfully Receive Personalized Gene Therapy Treatment
In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children’s Hospital of Philadelphia and Penn Medicine. The infant, KJ, was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase deficiency (CPS1). After spending […] The post Infant With Incurable Disease is First to Successfully Receive Personaliz…
Scientists have developed a revolutionary new genetic editing system capable of integrating whole genes into human DNA. This could, in the long run, offer a concrete hope for patients suffering from complex genetic diseases caused by a multitude of different mutations. Up to now, genetic editing tools were often limited to correcting one or a few of the [...]
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