For instance, CMT type 4c is an autosomal recessive condition caused by a defective SH3TC2 gene.
Gene replacement: This approach is suitable when the IPN is caused by a single defective gene.
#DOITNOW GENE TRIAL#
A Phase I/IIa trial is currently evaluating the effect of the NT-3 gene in patients with Charcot-Marie-Tooth Neuropathy (CMT) type 1A ( Identifier: NCT03520751). NT-3 has been shown to induce axonal regeneration in mouse models.
Gene addition: The Neurotrophin-3 gene is known to influence proliferation and migration of Schwann cells, and release of neurotrophic factors, all of which aid in axonal growth. “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. “There is no clear mode of action,” explained Dr Young Bin Hong, Associate Professor of Biochemistry at Dong-A University, Korea. Today, treatment of IPN is based on modifying the cellular response to degeneration, rather than addressing the cause. In this regard, treatment of inherited peripheral neuropathy (IPN) poses a challenge as the cause is genetic. Palliative treatment may include the use of specific medication for chronic neuropathic pain or mechanical aids for motor weakness.Īddressing 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. Currently, management of neuropathy has two arms – palliative control of symptoms and addressing the root cause of the disease to prevent progression. 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. Limitations of currently available treatments
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