Breakthrough gene therapy saved a child from “Bubble Boy Disease”
HT had a rare mutation in the DCLRE1C or Artemis gene, meaning he had Artemis-SCID – the most serious of primary immunodeficiencies.
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Joseph Shavit
- Sep 7, 2022
Single stem cell-gene therapy session can treat ALS for 3 years, study finds
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.
Joseph Shavit
- Mar 28, 2022
Opioid-free solution for chronic pain developed by scientists
A gene therapy for chronic pain, developed by researchers at UC San Diego, could offer a safer, non-addictive alternative to opioids.
Joseph Shavit
- Feb 24, 2022
'First-of-its-kind' gene therapy restores severe hearing loss
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.
Joseph Shavit
- Dec 16, 2021
Gene therapy advance shown to restore severe hearing loss
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.
Joseph Shavit
- Oct 19, 2021
Gene therapy can restore vision after stroke
Most strokes happen when an artery in the brain becomes blocked. Blood flow to the neural tissue stops, and those tissues typically die.
Joseph Shavit
- May 25, 2021
A new type of gene-based therapy partially restored a blind man’s vision
Light-activated proteins inserted in eye nerve cells and special goggles help him see objects
Joseph Shavit
- Mar 11, 2021
With gene therapy, scientists develop opioid-free solution for chronic pain
A gene therapy for chronic pain could offer a safer, non-addictive alternative to opioids. Researchers at the UCSD...
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