Gene therapy of neuropathy
Gene Therapy May Relieve Pain from Spinal Cord Injury
Gene therapy for spinal cord injury can open a new page in the possibilities of modern medicine. It shows promising results in mice, the researchers report.
About half of patients with spinal cord injuries suffer from neuropathy — chronic or debilitating pain, tingling, numbness or muscle weakness caused by nerve damage or malfunction.
The treatment of neuropathy can be difficult. For example, medications often require complex and continuous administration and can cause side effects such as sedation and motor weakness. Prescription opioid painkillers may be effective, but may lead to increased tolerance and risk of abuse, the researchers explained.
It is possible to accurately determine the location of spinal cord injury and the origin of neuropathic pain, so scientists are trying to develop treatment methods aimed at damaged or malfunctioning neurons in the affected areas of the spine.
Gene therapy for spinal cord injury involves the injection of a harmless virus carrying a pair of transgenes — GAD65 and VGAT, which encode gamma-aminobutyric acid (GABA), a neurotransmitter that blocks pain signals between nerve cells.
Gene therapy for spinal cord injury: what experiments have shown
The therapy was tested on mice with sciatic nerve injuries that caused severe neuropathic pain, and caused noticeable suppression of neurons transmitting pain signals. The effect persisted for at least two and a half months after treatment.
According to the authors, since the therapy was aimed at a specific site of sciatic nerve damage in animals, no side effects were found in them.
"One of the necessary conditions for clinically acceptable antinociceptive (pain—blocking) therapy is a minimal amount or absence of side effects, such as muscle weakness, general sedation or the development of tolerance to treatment," said senior study author Dr. Martin Marsala, professor of the Department of Anesthesiology at the University of California, San Diego School of Medicine.
"It is also very desirable to have a single invention that provides a long-term therapeutic effect. These results make it possible to advance in both directions," Marsala added in a university press release.
It is noted, however, that animal studies do not always produce the same effect in humans.
The study was recently published online in the journal Molecular Therapy (Tadokoro et al., Precision spinal gene delivery-induced functional switch in nociceptive neurons reverses neuropathic pain).
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