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In peripheral neuropathies, therapeutic genes operate at the cellular level to suppress factors causing degeneration and to induce an environment that is conducive to axonal growth and regeneration. Credit: Getty Images
Peripheral neuropathy is an umbrella term for a number of conditions in which there is degeneration of peripheral nerves. There are over 150 different causes of peripheral neuropathy, including heredity, diabetes, autoimmune disorders, and chemotherapy. The leading cause is diabetes, with about 60-70% of people with diabetes developing the condition. Overall, it is estimated that almost 20 million people in the United States currently live with some form of peripheral neuropathy.1
Limitations of currently available treatments
While not always life-threatening, peripheral neuropathies damage both sensory and motor nerves, leading to a complex array of symptoms that can significantly impact quality of life. Currently, management of neuropathy has two arms – palliative control of symptoms and addressing the root cause of the disease to prevent progression. Palliative treatment may include the use of specific medication for chronic neuropathic pain or mechanical aids for motor weakness.
Addressing the root cause of the disease depends on the disease itself, and may involve strict glucose control for diabetes or immunosuppressive medication for autoimmune diseases. In this regard, treatment of inherited peripheral neuropathy (IPN) poses a challenge as the cause is genetic. Today, treatment of IPN is based on modifying the cellular response to degeneration, rather than addressing the cause. “There is no clear mode of action,” explained Dr Young Bin Hong, Associate Professor of Biochemistry at Dong-A University, Korea. “While such drugs, especially small molecules, have advantage in the proof of safety, the disease-modifying efficacy is still questionable.” There is, therefore, a need to find drugs that address the cause of nerve degeneration and halt the process.
How gene therapy can transform management of peripheral neuropathy
Gene therapy introduces selected sections of DNA into the human body to dictate cellular functioning. The main feature of peripheral neuropathy is axonal degeneration. Therapeutic genes operate at the cellular level to suppress factors causing such degeneration and to induce an environment that is conducive to axonal growth and regeneration.
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