That leads to a buildup of ammonia that destroys the liver and can cause neurological damage with lifelong consequences. His ...

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

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

In a medical first that could revolutionize treatment for thousands of rare genetic diseases, doctors have successfully used ...

Acwa Power has signed Memorandums of Understanding (MoUs) worth $500 million with US companies, building on its existing ...

A race was on to understand what was wrong. A blood test revealed toxic levels of ammonia building up in KJ’s body, ...

KJ received the first dose through an infusion in February, followed by two additional doses in March and April. Doctors ...

Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a ...

An infant named KJ has made history as the first patient to be treated with an in vivo CRISPR gene editing therapy designed ...

Breakthrough therapy may pave the way for personalized cures and a cheaper path to treating ultra-rare conditions.

“This is the first step towards the use of gene editing therapies to treat a wide variety of rare genetic disorders for which ...