The one-off treatment highlights the promise of a new type of gene editing and the challenges of using it to treat extremely ...

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.

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

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

An infant in Philadelphia has been successfully treated with a customized Crispr gene-editing therapy for a rare and deadly ...

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

A blood test revealed toxic levels of ammonia building up in KJ’s body, threatening to damage his brain — a sign of an ...

The researchers have conducted a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing technique aimed at ...

A baby born with a rare and dangerous genetic disease is thriving after getting an experimental gene editing treatment made ...

Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing ...

The case is detailed today in a study published by The New England Journal of Medicine and was presented at the American ...