For decades, the dream of fixing harmful mutations in mitochondrial DNA felt out of reach. Scientists have long known these ...

A new study suggests a gene mutation could have a protective effect against Alzheimer's. The research looked specifically at ...

Microglia replacement therapy helps treat people with a rare genetic condition called ALSP, suggesting the approach could ...

The discovery may explain why cutting edge CAR T-cell therapy has managed to revolutionized treatment for blood cancer but ...

Rare genetic diseases are challenging for patients and their families—made all the more overwhelming because symptoms tend to ...

Mutations are changes in the genetic sequence, and they are a main cause of diversity among organisms. These changes occur at many different levels, and they can have widely differing consequences.

A mutation is a heritable change in the nucleotide sequence of an organism's DNA that ultimately serves as a source of genetic diversity. A single base change can create a devastating genetic ...