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Gene therapy approach reverses diabetic neuropathy in animal model, Pitt study finds

SAN DIEGO, Oct. 26 Researchers from the University of Pittsburgh School of Medicine have demonstrated for the first time that gene therapy can reverse diabetic neuropathy. While their studies have so far only involved mice, the results are significant because they provide the earliest evidence that such an approach might some day help people with diabetes, in whom neuropathy is a common complication that causes irreversible nerve damage. Details of the research were presented today at the 34th Annual Meeting of the Society for Neuroscience, being held Oct. 23 27 in San Diego.

More than two thirds of patients with diabetes develop neuropathy, which generally affects the sensory neurons within the peripheral nervous system and is characterized by such symptoms as numbness, tingling, pins and needles or loss of sensation, most often to the legs and feet. As a result, patients may be unaware of the presence of ulcers or infections or that they've experienced injury. No treatments exist that can stop progression of neuropathy, let alone reverse any damage to nerves.

Five weeks after a one-time inoculation, diabetic animals receiving gene therapy had complete reversal of established peripheral neuropathy and restoration of lost nerve endings to their feet, reported James R. Goss, Ph.D., research assistant professor of molecular genetics and biochemistry at the University of Pittsburgh School of Medicine. He and colleagues had previously found the same gene therapy approach could prevent the development of neuropathy in diabetic animals.

While the exact cause of diabetic neuropathy is unknown, there is evidence to suggest it is associated with a deficiency or dysfunction of certain neurotrophic factors, which are proteins essential for the survival and proper function of neurons. Therefore, the Pitt researchers sought to develop a therapy that would shuttle the genes responsible for their production directly into affected neurons. To gain entry
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