PHILADELPHIA (WPVI) -- An infant, who was born with a deadly disease, is now thriving, thanks to a medical first performed at ...

In a groundbreaking achievement, doctors have successfully treated a baby boy with a life-threatening genetic disorder using ...

For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as ...

The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific ...

Fargo’s Aldevron helped create a custom gene-editing therapy that cured a baby of urea cycle disorder, a rare and potentially ...

A tailored CRISPR base-editing therapy was given for the first time to an infant who was born with a rare genetic disease, ...

A newborn boy, KJ, was diagnosed at birth with a rare and potentially fatal genetic disease. He received a novel, on-demand CRISPR therapy in record time.

KJ Muldoon was born with a devastating medical problem. Racing to solve it, doctors may have launched a 'roadmap' for a new type of treatment.

In a study published May 15 in the New England Journal of Medicine and presented at the American Society of Gene and Cell ...

The baby boy received the bespoke therapy within a few months of birth and is doing well, according to a presentation at ASGCT.

They were also able to confirm defects in the process cells use to make ribosomes. Importantly, this study showed that different RNA exosomopathy mutations have unique molecular signatures that ...