UCD stem cell research battles Huntington's disease

A team of researchers at UC Davis has pioneered a technique to
use stem cells to smother the genetic problem that causes
Huntington's disease.

The findings, due in the journal Molecular and Cellular
Neuroscience, could pave the way for a treatment that stops the
disease's devastating progression.

Huntington's is an inherited disease in which the body produces
a mutant version of a protein, huntingtin, that destroys nerve
cells in the brain.

It causes uncontrolled movements and difficulty walking, plus
dementia that grows progressively worse until the disease
ultimately results in death. It strikes about one in every
10,000 people in this country, according to the Huntington's
Disease Society of America.

There is no known cure. Treatment aims to slow down the
worsening of symptoms and keep the patient comfortable.

Researchers at the UC Davis Institute for Regenerative Cures,
led by Jan A. Nolta, attacked abnormal huntingtin with a
technique called RNA interference.

This is how it works: RNA is a molecule similar to DNA that
occurs naturally in the body and which cells use to produce
proteins.

If a strand of RNA is producing a bad protein, like the mutant
huntingtin, researchers can create another strand that's
essentially an inverted version of the bad one. Inject that new
molecule into a cell, and it locks onto the bad RNA like an
opposite puzzle piece, blocking it from making any protein.

For the first time, Nolta and colleagues were able to generate
huntingtin-blocking RNA in stem cells and inject them straight
into nerve cells – a treatment that significantly reduced the
amount of the mutant protein produced.

The scientists used stem cells derived from the bone marrow of
healthy human donors.

The California Institute for Regenerative Medicine and Team KJ
funded the research.

Nolta said the findings could lead to treatments for genetic
disorders such as ALS (Lou Gehrig's disease) and Parkinson's,
as well.

Now, she said, "Our challenge with RNA interference technology
is to figure out how to deliver it into the human brain in a
sustained, safe and effective manner. We're exploring how to
use human stem cells to create RNAi production factories within
the brain."

Nolta's lab recently received funding from the California
Institute for Regenerative Medicine to develop an RNAi delivery
system for Huntington's disease.

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Call The Bee's Grace Rubenstein, (916) 321-1270.

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UCD stem cell research battles Huntington's disease