In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a ...

Scientists were able to create a bespoke treatment for KJ Muldoon’s rare genetic disorder within six months. It could be a ...

A team of doctors at the Children's Hospital of Philadelphia and Penn Medicine treated a baby with a gene editing technology ...

A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design ...

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.

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

A Delaware County infant with a rare metabolic disease, CPS1 deficiency, was treated at CHOP with a custom CRISPR therapy.

Aldevron and Integrated DNA Technologies – both part of the Danaher Corporation – have achieved a medical milestone with the ...

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 ...

G ene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has ...