Gene therapy for pain could be the safer, non-addictive optionAlternative to opioidsrepresent. Researchers developed the new therapeutic approach by temporarily suppressing a gene that is involved in the sensation of pain. This increased pain tolerance in mice, reducing their pain sensitivity and providing months of pain relief without causing numbness.
Promising gene therapy for pain
Gene therapy could be used to treat a wide range of chronic pain conditions. Such pain ranges from the lower back to rare neuropathic pain disorders. The latter are conditions for which painkillers are the current standard of care. Opioids, on the other hand, can make people more sensitive to pain over time. This results in them having to rely on ever higher doses. A genetic mutation studied by the researchers inactivates a protein in neurons in the spinal cord. This is called NaV1.7. Accordingly, in people without functional NaV1.7, sensations such as touching something hot or sharp are not registered as pain. The team also developed a CRISPR/dead Cas9 system to target and suppress the gene encoding NaV1.7. They administered it to mice with inflammatory and chemotherapy-induced pain. The experimental animals showed correspondingly higher pain thresholds than mice that did not receive gene therapy for pain.
To validate their results, the researchers then ran the same tests using a different tool. This involved an older technology than CRISPR, which does the same job. Here, the researchers designed zinc fingers that similarly bind to the gene target and block the expression of NaV1.7. Spinal injections of the zinc fingers in mice produced the same results as the CRISPR-dead Cas9 system. The scientists claim that this solution could work for a wide range of chronic pain conditions. This includes diabetic polyneuropathy, erythromelalgia, sciatica and osteoarthritis. The treatment could also provide relief for patients undergoing chemotherapy. Next, researchers will work to optimize both approaches for targeting the human gene. They will conduct experiments on non-human primates to test efficacy and toxicity. The authors go beyond thatthis studyexpect to begin human clinical trials in a few years.
