Scientists Use Stem Cells to Mimic Huntington’s Disease

FRIDAY, July 6 (HealthDay News) — Scientists have found a way to
use stem cell technology to study Huntington’s disease, a progressive
inherited brain disorder that causes lack of muscle control, psychiatric
disorders, dementia and ultimately death. The disease is currently
incurable.

Working with an international consortium, researchers from Johns
Hopkins in Baltimore cultured skin cells from a young person with a
severe, early onset form of the disease. They used these cells to create
stem cells, which were turned into neurons that degenerated rapidly, just
as they do in people with Huntington‘s.

“These [Huntington’s disease] cells acted just as we were hoping,” one
of the study’s lead researchers, Dr. Christopher Ross, director of the
Baltimore Huntington’s Disease Center, said in a Johns Hopkins news
release.

“A lot of people said, ‘You’ll never be able to get a model in a dish
of a human neurodegenerative disease like this,'” he said. “Now we have
them where we can really study and manipulate them, and try to cure them
of this horrible disease. The fact that we are able to do this at all
still amazes us.”

By creating “Huntington’s disease in a dish,” the researchers said,
they have made strides in understanding what disables and kills the cells
in people with the disease. The findings also will help test the effects
of possible treatments on cells that were previously found only deep
inside the brain.

“Having these cells will allow us to screen for therapeutics in a way
we haven’t been able to before in Huntington’s disease,” said Ross, who
also is a professor of psychiatry and behavioral sciences, neurology,
pharmacology and neuroscience at the Johns Hopkins University School of
Medicine. “For the first time, we will be able to study how drugs work on
human [Huntington’s disease] neurons and hopefully take those findings
directly to the clinic.”

The study authors added that their findings could have implications for
similar research on other degenerative brain diseases, such as Alzheimer’s
and Parkinson’s disease.

The study was published online in the journal Cell Stem
Cell
.

More information

The U.S. National Library of Medicine has more about Huntington’s disease.

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