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Neuroscience research

Designer genes target serious diseases

 

It's not surprising that neuroscientists in the School of Medicine are conducting research on epilepsy, Alzheimer's disease, neuropathic pain, cognition, mood disorders, and other nervous system-related conditions.

But it is extraordinary that all of them are using a similar gene therapy technique to blaze new trails of discovery. It's a technique made possible by a laboratory at the School of Medicine that engineers the key ingredients for gene therapy research used by neuroscientists around the world.

Marlene Wilson width=
Marlene Wilson

"We've had these core lab facilities for quite a while, and [lab director] Steve Wilson is recognized as one of the pioneers in developing this technology," said Marlene Wilson (no relation to Steve Wilson), a professor and chair of the Department of Pharmacology, Physiology, and Neuroscience.

Wilson and her colleagues in neuroscience all use common viruses that have been altered in special ways to deliver genetic instructions to a specific target -- either in the brain or the peripheral nervous system. The designer viruses are harmless but effective messengers of genetic material.

The gene transfer technique is allowing the neuroscientists to better understand the biochemical mechanisms associated with certain disorders and diseases. In some instances, they might be able to develop better targets for medications; in other cases, the gene therapy itself might introduce new ways of treating disease.

"There's a lot of effort going on now to make safe viral vectors for human delivery," Wilson said, "It's likely that gene transfer would initially be used for crippling disorders like degenerative diseases, chronic pain, and epilepsy. But it could be on the horizon to treat conditions such as severe anxiety and even depression disorders."

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