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

A sweeping ancient-DNA analysis of more than 22,000 genomes from Europe and the Near East shows that natural selection ...

Everyday DNA mutations can help us understand immune function and aging—and even mitigate harm caused by some inherited ...

A recently published study from Fred Hutch Cancer Center dramatically expands the number of FDA-approved cancer drugs ...

By comparing genetic data from 23 million newborns to rare disease models, researchers found some models’ estimates were 10 ...

An analysis of ancient DNA and modern disease risk suggests some immune genes may reduce allergy risk rather than increase it ...

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) can have the same genetic cause, a discovery that won ...

Genome editing is revolutionizing the medical landscape by offering new therapeutic possibilities for genetic diseases, which collectively affect hundreds ...

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

The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time ...

Tracking how blood cancers develop over time has uncovered important genetic differences between patients whose disease stays ...

A trio of common amino acids may hold the key to unlocking far more powerful gene therapies. Researchers found that adding ...