HT had a rare mutation in the DCLRE1C or Artemis gene, meaning he had Artemis-SCID – the most serious of primary immunodeficiencies.

Using stem cells is a powerful way to deliver important proteins to the brain or spinal cord that can’t get through the blood-brain barrier.

A gene therapy for chronic pain, developed by researchers at UC San Diego, could offer a safer, non-addictive alternative to opioids.

Hearing loss has been linked to mutations in at least 100 different genes, but up to 16 percent can be traced to just one gene, STRC.

Hearing loss has been linked to mutations in ~100 different genes, but up to 16 percent of genetic hearing loss can be traced to STRC gene.

Most strokes happen when an artery in the brain becomes blocked. Blood flow to the neural tissue stops, and those tissues typically die.

Light-activated proteins inserted in eye nerve cells and special goggles help him see objects

A gene therapy for chronic pain could offer a safer, non-addictive alternative to opioids. Researchers at the UCSD...