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.

Every few months for the past three years, Jeff Vierstra has been receiving infusions in his spine that target and disable a ...

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

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

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

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

From the time we are conceived and through old age, genetic mutations accumulate in all our tissues, eluding the body’s typically efficient DNA repair machinery and potentially affecting our health ...

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

For decades, the dream of fixing harmful mutations in mitochondrial DNA felt out of reach. Scientists have long known these mutations cause serious diseases that pass down only through mothers. They ...

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

March 24 (Reuters) - Revealing the limitations of cloning, researchers who repeatedly cloned mice for two decades have ...