Gene Therapy Approach for Treating Neuropathy

Gene therapy strategies for treating peripheral neuropathy.

Peripheral neuropathy (PN) results in loss of innervation by the peripheral nerves in tissues such as skin, muscle, heart, and adipose tissue. It is a painful, debilitating disease, that is estimated to affect between 20 and 30 million Americans. The number of Americans affected by PN is likely much higher because many individuals with symptoms are not tested and there is a lack of accurate diagnostics, particularly for small fiber neuropathies. Small fiber are the top categories for PN, and encompass diabetes/obesity, aging, chemotherapy-induced, long COVID and more.

The Need

There are currently no therapies that can halt the neuropathy and stimulate regeneration of the peripheral axons. Under certain conditions like nerve crush injury, peripheral nervous system axons are more capable of regeneration than the nerves of the central nervous system. Therefore, new therapies capable of coaxing these neuropathic axons to recovery are needed. PN is the number one cause of neurodegeneration in humans.

The Technology

Researchers at The Ohio State University have developed a novel gene therapy strategy for treating peripheral neuropathy using adipocyte-targeting adeno-associated virus (AAV) to deliver neurotrophic factors (BDNF and NGF) directly to adipose tissue to encourage neurite outgrowth to those specific cells. This proof-of-concept approach showed promising results in improving tissue-specific PN in obese/diabetic mice. This approach could utilize other AAVs to target peripheral nerve subtypes or additional cells/tissues, reducing off-target effects from systemic treatments


  • Localized delivery to peripheral nervous tissue minimizes off-target effects.
  • Gene therapy approach offers a potentially long-term solution compared to traditional therapies.
  • Ability to provide combination therapy with multiple neurotrophic factors targeting different tissues/cells in a physiologically-relevant manner.
  • Addresses a critical gap in PN treatment, offering hope for millions suffering from metabolic conditions.

Patent application pending

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