CIRM Awards UC Irvine Neuroscientist Another $2 Million for Huntington's Disease Research

Goal is to determine why the fatal condition destroys some brain cells but spares others

Irvine, Calif., Oct. 8, 2025 — A UC Irvine neuroscientist has been awarded more than $2 million by California’s stem cell agency to probe one of medicine’s most baffling mysteries: why Huntington’s disease devastates some brain cells while sparing others.

Leslie M. Thompson, the Donald Bren Professor of Psychiatry & Human Behavior and Neurobiology & Behavior, will receive $2,056,195 from the California Institute for Regenerative Medicine. Her team plans to use stem cell-based models to map how the defective Huntington’s protein interacts with other proteins and RNA across different cell types. This approach takes advantage of the unique ability of stem cells to differentiate into multiple cell types.

The hope, Thompson says, is to explain the selective vulnerability of neurons to the disorder and, ultimately, to identify new therapeutic targets.

Huntington’s disease is a fatal, inherited condition marked by progressive loss of motor control, psychiatric symptoms and cognitive decline. It stems from a single gene mutation discovered in 1993 – a project in which Thompson played a key role – but the disorder’s biology remains poorly understood.

“The disability, loss of independence and medical costs associated with Huntington’s disease and other neurodegenerative disorders have a profound impact on patients, families and society,” Thompson said. “There have been exciting new advances in the treatment of HD showing that it’s feasible to modify the course of the disease, and this project has the potential to better understand the disease and provide a new approach that can also lead to insights into other brain diseases.”

Read the full press release in UC Irvine News.