Chronic pain patients primary source of relief is typically a pain-killing opiate. Unfortunately, opiate drugs are often unsatisfactory because of poor efficacy or intolerable side effects like extreme sleepiness, mental clouding, and hallucinations.

Dr. Anreas Beutler, MD, Assistant Professor of Medicine/ Hematology and Medical Oncology, Mount Sinai School of Medicine, has developed a novel approach using gene therapy with the help of her research team.

The researchers designed a viral vector that was used to carry the gene prepro-b-endorphin to neurons that would selectively activate opiate receptors.

Using rats, researchers found that the subjects could be injected once with Adeno-Associated Virus vector based gene therapy and experience pain relief for more than 3 months. The technique worked successfully with opioid and non-opioid therapeutic genes. Using targeted gene therapy is likely to avoid the unwanted side effects associated with opioid painkillers–like morphine.