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Gene Therapy Protects Against Diabetic Nerve Damage

Gene Therapy Protects Against Diabetic Nerve Damage

MANCHESTER, England, May 31 ? An investigational gene therapy offers promise for diabetic neuropathy, investigators here reported. It would prod the body into ramping up growth factor production.

"Our approach to gene therapy is quite different to previous attempts at treatment," said David Tomlinson, Ph.D., of the University of Manchester here. "We use a DNA-binding protein called zinc finger protein transcription factor (ZFP-TF) to poke life into the patient's own genes and produce a growth factor that has a role in nerve protection and regeneration."

In preclinical studies, a single injection of the DNA-binding protein protected nerve function, stimulated nerve growth, and prevented tissue neuropathy related tissue damage, Dr. Tomlinson and colleagues reported in the June issue of Diabetes.

Sangamo BioSciences of Richmond, Calif., reported that the compound has been tested in 12 diabetic patients, with mild injection-site reactions in four. The clinical data were not discussed in the Diabetes paper.

"We look forward to the next stage of development of this novel therapeutic when phase-two clinical trials start later this year," said Edward Lanphier, the company's president and CEO.

The therapeutic compound is designed to stimulate or up-regulate the expression of the gene encoding for vascular endothelial growth factor (VEGF-A), which has been shown to have direct neurotrophic and neuroprotective properties.

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