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

For the first time, a futuristic gene therapy has helped a sick baby. The new technique allowed doctors to modify the DNA of a baby...

International researchers have called the treatment a promising medical milestone.

Experimental gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions

Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can someday help millions who have been left behind by genetic medicine because their diseases are so rare.

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.