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'Forged' brain cells offers hope for Huntington's disease treatment

March 16th, 2012 3:51 pm

Washington, Mar 16 (ANI): A special type of brain cell forged from stem cells could help restore the muscle coordination deficits that cause the uncontrollable spasms characteristic of Huntington's disease, a new study has suggested.

Huntington's disease, the debilitating congenital neurological disorder that progressively robs patients of muscle coordination and cognitive ability, is a condition without effective treatment, a slow death sentence.

"This is really something unexpected," said Su-Chun Zhang, a University of Wisconsin-Madison neuroscientist and the senior author of the new study, which showed that locomotion could be restored in mice with a Huntington's-like condition.

Zhang is an expert at making different types of brain cells from human embryonic or induced pluripotent stem cells.

In the new study, his group focused on what are known as GABA neurons, cells whose degradation is responsible for disruption of a key neural circuit and loss of motor function in Huntington's patients.

GABA neurons, Zhang explained, produce a key neurotransmitter, a chemical that helps underpin the communication network in the brain that coordinates movement.

In the laboratory, Zhang and his colleagues at the UW-Madison Waisman Center have learned how to make large amounts of GABA neurons from human embryonic stem cells, which they sought to test in a mouse model of Huntington's disease.

The goal of the study, Zhang noted, was simply to see if the cells would safely integrate into the mouse brain.

To their astonishment, the cells not only integrated but also project to the right target and effectively re-established the broken communication network, restoring motor function.

The results of the study were surprising, Zhang explained, because GABA neurons reside in one part of the brain, the basal ganglia, which plays a key role in voluntary motor coordination.

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'Forged' brain cells offers hope for Huntington's disease treatment

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