US EditionFirst Personalized Gene Therapy Using Base Editing Shows Promising Results in Baby with Rare Disorder
- A baby named KJ Muldoon from Clifton Heights, Pennsylvania, is growing and thriving after receiving a personalized gene editing treatment for a rare genetic disease.
- KJ received a diagnosis of severe CPS1 deficiency shortly following his birth, a rare disorder affecting approximately one in every million infants and resulting in a 50% fatality rate among those affected.
- Within half a year, a research group affiliated with Children’s Hospital and Penn Medicine developed a CRISPR-based gene-editing treatment administered via lipid nanoparticle infusions, which KJ received between February and April.
- KJ, now nine months old, eats more normally, recovers better from illnesses, and takes less medication, though researchers caution it is still early to fully assess the therapy's effects.
- Experts see this case as the first step toward treating many rare genetic disorders, but note personalized therapies may take years to become widely available due to existing barriers and costs.
236 Articles
236 Articles
Washington Should Fund More Potential Science Miracles
A baby boy, born in Philadelphia in 2024 with a potentially fatal genetic deficiency, has become the first person treated with a custom gene-editing therapy. Doctors used CRISPR technology to rapidly create a personalized treatment for KJ, targeting the child’s specific mutation. The boy is meeting developmental milestones, his weight is in the 40th percentile, but his need for a liver transplant is still unknown, according to news reports. This…
From birth to gene-edited in 6 months: Custom therapy breaks speed limits
News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects an ultra-rare mutation in KJ that breaks a liver enzyme. That enzyme is required to convert ammonia, a byproduct of metabolism, to urea, a waste product released in urine. Without treatment, ammonia would build up to dangerous levels in KJ—and he would…
World's first successful tailor-made gene therapy saves baby born with rare disorder
Baby KJ Muldoon was born with a rare genetic condition that is often fatal, but doctors used custom CRISPR gene therapy to target the exact mutation in his DNA. His family shares their emotional journey in their first TV interview with CBS News.
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