A man from Norway has likely been cured of HIV after receiving a stem cell transplant from his brother, who has two copies of ...

A newly identified gene mutation may help explain why schizophrenia patients struggle to update their understanding of reality. The mutation disrupts a brain circuit involved in flexible ...

As we age, our cells accumulate genetic changes—mutations—some of which open the door to cancer. Scientists call these ...

Every few months for the past three years, Jeff Vierstra has been receiving infusions in his spine that target and disable a ...

A surprising discovery from high-altitude animals like yaks and Tibetan antelopes could reshape how we treat nerve damage in humans. Scientists found that a genetic mutation helping these animals ...

In 2025, baby KJ Muldoon became the first person to receive a personalized gene editing treatment, which likely saved his ...

A 63-year-old man in Norway seems to have gotten one of the best gifts ever given by a brother: a cure to his lifelong HIV ...

Though many studies approach the developmental disorder Rett syndrome as a single condition arising from general loss of ...

Scientists have corrected an extremely rare and life-threatening genetic disease of the liver in mouse models and human ...

Imagine getting only a few hours of sleep and being raring to go the next day. A new study published in the journal Proceedings of the National Academy of Sciences shows the discovery of a gene ...

University of Virginia School of Medicine scientists have used a next-generation form of gene editing to fix the underlying cause of a severe form of epilepsy in lab mice.