Researchers say the CRISPR-based technique used could eventually be employed to treat more people with rare genetic diseases ...

With grim prognosis hanging overhead, doctors and scientists at universities and institutions across the U.S. worked ...

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

The only gene-editing therapy currently on the market is a CRISPR–Cas9-based treatment for two blood disorders, sickle-cell ...

Doctors have successfully used gene-editing therapy to treat an infant born with a rare disorder. Experts believe the ...

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 ...

A hot (if cautious) topic at the big gene therapy meeting: what Vinay Prasad's appointment might mean for approvals in rare ...

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

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 ...

Hopkins neuroscientist Richard Huganir is close to finding a potential life-changing treatment for kids with SYNGAP1-related ...