Cystic fibrosis is among the most common, known and studied genetic diseases. It affects over 100,000 people worldwide and ...

The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current CF therapies.

As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” ...

An experimental gene therapy for a rare form of genetic deafness has successfully restored hearing in children and adults, ...

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 ...

A genetic mutation that helps animals like yaks and Tibetan antelopes survive at high altitudes may hold the key to repairing nerve damage in conditions such as cerebral paralysis and multiple ...

Humanity is gradually conquering incurable genetic diseases that were once considered untreatable, thanks to a series of ...

Researchers at Weill Cornell Medicine report that a rare gene mutation that delays Alzheimer’s disease does so by damping inflammatory signaling in brain-resident immune cells in a preclinical study.

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 ...

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 ...

You might like the sound of this news. In a landmark move, the Food and Drug Administration on Thursday approved the first ...

For roughly one in ten people living with cystic fibrosis, the drugs that have transformed survival for most CF patients are ...