Scientists find drug that helps Huntington's disease-afflicted mice — and their offspring

Posted: December 23, 2014 at 5:48 am

IMAGE:Authors of the new study include (left to right) Elizabeth Thomas, Ph.D., associate professor at the Scripps Research Institute, and Haiqun Jia, first author and professional scientific collaborator. view more

Credit: Photo courtesy of The Scripps Research Institute.

LA JOLLA, CA--December 22, 2014--Famine, drug abuse and even stress can "silence" certain genes, causing health problems in generations to come. Now scientists are wondering--could therapies that change gene expression in parents help their children?

A new study from scientists at The Scripps Research Institute (TSRI) suggests this is possible. The research showed that the offspring of mice treated with a drug also had delayed onset and reduced symptoms of Huntington's disease, an inherited, degenerative disease that causes a loss of motor skills, cognitive impairment and death.

This was the first time scientists have shown that drug compounds that benefit parents can also cause changes in genetic expression that benefit offspring--in this case, improved memory and motor skills.

"One exciting aspect of our study is that the parental drug treatment made the offspring better, not worse, like other compounds known to cause transgenerational effects," said Elizabeth Thomas, associate professor at TSRI who led the new study.

Thomas and her colleagues report their findings online ahead of print in this week's Early Edition of the journal Proceedings of the National Academy of Sciences.

Compound Shows Potential

The Huntington's Disease Society of America estimates that more than a quarter of a million Americans have the disease or are at risk of inheriting it from a parent. Thomas began studying Huntington's disease 15 years ago, when she found out that a close friend's mother had the disease.

"If your mom or dad carries the mutation, you have a 50-50 chance of inheriting the disease," said Thomas. Although there is a test to see if a person will develop Huntington's, Thomas said many people don't get tested because there are no good treatments to prevent or reduce symptoms.

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Scientists find drug that helps Huntington's disease-afflicted mice -- and their offspring

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