A team of researchers from the University of California, San Diego, showed that gene therapy, which blocks the signaling of targeted neurons, was effective in reducing neuropathic pain without detectable side effects in mice with spinal cord or peripheral nerve injuries. Of course, studies on mice are not the same as human studies, but the results are really promising.
It’s an entirely new way to treat a condition that affects more than half of patients with spinal cord injuries. It is noteworthy that neuropathy is a really broad term – disorders of this type include dysfunction related to one nerve, and even several. The same applies to the causes of neuropathy, which can also be different. Neuropathy is damage or dysfunction of nerves elsewhere in the body, usually causing chronic or debilitating numbness, tingling, muscle weakness, and pain.
Unfortunately, there are no 100% effective treatments for neuropathy. Pharmacological agents often require complex and continuous drug administration and are associated with unpleasant side effects. Opioids can be effective, although you can get addicted quite easily.
But because doctors are able to pinpoint the exact location of a spinal cord injury and the source of nerve pain, it’s possible to use a method that selectively targets damaged nerve cells. Gene therapy is such a method. Scientists from the University of California injected mice with impulse nerve damage and neuropathic pain with a harmless adenovirus that contains a pair of transgenes encoding GABA. It is a neurotransmitter that blocks impulses between nerve cells (specifically here, pain signals).
The GAD65 and VGAT genes were delivered to the sciatic nerve lesion in mice, so there were no detectable side effects such as impaired movement or loss of normal sensation. Production of GABA by the transgene resulted in a measurable inhibition of pain signaling to neurons in mice that persisted for at least 2.5 months after treatment.
A prerequisite for clinically acceptable anti-panic (preventing) treatment is the reduction or absence of side effects, such as muscle weakness, general anesthesia, or the development of treatment tolerance. The invention in the form of a single remedy, which provides a long-term therapeutic effect, is also very desirable. These findings point to a way forward in both areas, said Dr. Martin Marsala, a professor in the department of anesthesiology at the University of California, San Diego School of Medicine.
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