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.