Every few months for the past three years, Jeff Vierstra has been receiving infusions in his spine that target and disable a mutated gene that made it likely he would develop ALS.

A newly identified gene mutation may help explain why schizophrenia patients struggle to update their understanding of ...

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.

Columbia's Silence ALS program is testing spinal antisense therapy in a high‑risk patient; early EMG tests normalized.

Some of your most important life partners are the mitochondria that power all your cells. You and these little cellular ...

UF scientist develops safer gene therapy restoring vision in children with rare inherited eye diseases; pivotal trials begin ...

The PLN gene mutation causes heart failure and sudden cardiac death. Experts say West Michigan's Dutch community could be ...

Carbone called BAP1 a “paradox” because of its seeming ability to both promote and slow cancer, depending on the ...

A new study from the Massachusetts Institute of Technology suggests that a specific gene mutation prevents patients with ...

Circulating tumor DNA (ctDNA), small pieces of DNA released from dying tumor cells into the bloodstream, can be easily and non-invasively identified and examined through a simple blood test. This ...