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Gene Therapy May Be Future Prescription For Patients To Relieve Chronic Pain

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Gene Therapy May Be Future Prescription For Patients To Relieve

Chronic Pain

http://www.medicalnewstoday.com/articles/94869.php

Researchers in the Department of Medicine and Department of

Neurosciences at Mount Sinai School of Medicine have discovered that

chronic pain can be successfully treated with novel targeted gene

therapy. In an effort to find a more effective treatment for chronic

pain, researchers at Mount Sinai developed a gene therapy technique

that simulates the pain-killing effect of opiate drugs. In the new

study " Sensory neuron targeting by self-complementary AAV8 via

lumbar puncture for chronic pain " published in the January 22, 2008

issue of the Proceedings of the National Academy of Sciences (PNAS),

researchers suggest that gene therapy for pain might in the future

become a treatment alternative for patients with severe chronic

pain.

" Fifty million Americans suffer from chronic pain. Chronic pain

patients often do not experience satisfactory pain relief from

available treatments due to poor efficacy or intolerable side

effects like extreme sleepiness, mental clouding, and

hallucinations, " said Dr. s Beutler, MD, principal

investigator of the study and Assistant Professor of Medicine/

Hematology And Medical Oncology at Mount Sinai School of Medicine.

Mount Sinai researchers designed a viral vector to carry the prepro-

b-endorphin gene into primary sensory neurons in order to activate

opiate receptors selectively, in a rat model. The agents were

delivered directly into the spinal fluid of rats via a lumbar

puncture, or spinal tap with only one injection. Results showed that

the rats remained symptom-free for an extended period of time.

" Our research found that treating chronic pain with Adeno-Associated

Virus vector-based gene therapy allows for pain relief for more than

three months after a single injection, targeting selectively the

pain gate. The technique worked successfully with opioid- and non-

opioid therapeutic genes, " said Dr. Beutler. " Targeted gene therapy

will likely avoid the unwanted side effects associated with opioid

painkillers such as morphine. Based on our findings, this targeted

gene therapy via lumbar puncture appears to be a promising candidate

for bench-to-bedside research that might ultimately be tested in

patients with intractable chronic pain, e.g., to help patients

suffering from severe pain due to advanced cancer. "

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