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

Doctors announced this week that they have treated a newborn baby with a rare genetic disease using the world’s first personalized gene editing therapy. Geoff Bennett discussed the treatment and its ...

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

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

A range of other rare inherited diseases are now likely to be curable by similar procedures, the research team said ...

In the Phase 2 study of patients with chronic kidney disease, the highest dose of the drug lowered by 86% the levels of ...

All this is possible because KJ is the first-ever recipient of a gene therapy designed to treat only ... what was stacked up against him and how bad of a prognosis it was in the very beginning ...

The treatment, described in a new study published in The New England Journal of Medicine and presented at the American Society of Gene and Cell Therapy meeting ... 50% with no bad side effects.

On first glance, Dr. Vinay Prasad’s medical credentials sound impressive. But look closer and you’ll find a narcissistic ...