First Personalized Gene Therapy Using Base Editing Shows Promising Results in Baby with Rare Disorder

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…

Baby gets world's first personalised gene therapy treatment

Nine-and-a-half-month-old KJ Muldoon has a rare metabolic condition that meant he spent the first months of his life in hospital on a very restrictive diet.

First-Ever Custom CRISPR Therapy Saves Infant With Deadly Genetic Disorder

KJ was born with a metabolic disorder that kills up to 50% of infants.

Baby with rare life-threatening disorder makes medical history

Researchers hope what they learn from KJ will help other rare disease patients

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.