Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

An estimated 170,000 Australians were diagnosed with cancer in 2025. Many people know the causes of cancer are partly genetic. But how do your genes, which contribute so much of what makes you you, ...

A gene borrowed from the naked mole rat, an animal famous for extraordinary longevity and disease resistance, helped mice live a little longer and stay healthier, pointing to a surprising biological ...

Current gene therapies to treat sickle cell disease are complex, time-consuming, and are sometimes linked to serious side effects like infertility or blood cancer. To address these challenges, Johns ...

Researchers have discovered that mutations in the FOXJ3 gene act as a "master switch" failure, disrupting how the brain ...

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.

Scientists at The University of Texas at Austin have developed a revolutionary gene-editing method using bacterial retrons that can correct multiple disease-causing mutations at once. Unlike ...

In preclinical work recently published in Nature Communications, Fred Hutch Cancer Center scientists used a genetic “chain reaction” to transform herpes simplex virus DNA during an HSV infection. The ...

While working on her PhD at the Scripps Institution of Oceanography, Malia Moore focused on how local seagrasses could potentially be used for coastal restoration.