Huntington's Disease Fact Sheet

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Huntington's Disease

Medium spiny neurons (Jan Nolta
at the University of California, Davis)

CIRM funds several research projects that have the aim of developing stem cell-based therapies for neurological diseases including Huntington's disease. One of these is an Early Translational Award that is making the first steps in translating basic discoveries into therapies.

If you want to learn more about CIRM funding decisions or make a comment directly to our board, join us at a public meeting. You can find agendas for upcoming public meetings on our meetings page.

Learn more about stem cell research:
Stem Cell Basics Primer | Stem Cell Videos | What We Fund

Find clinical trials:
CIRM does not track stem cell clinical trials. If you or a family member is interested in participating in a clinical trial, please see the national trial database to find a trial near you: clinicaltrials.gov

Description

[From the National Institute of Neurological Disorders and Stroke] Huntington's disease (HD) results from genetically programmed degeneration of brain cells, called neurons, in certain areas of the brain. This degeneration causes uncontrolled movements, loss of intellectual faculties, and emotional disturbance.

HD is a familial disease, passed from parent to child through a mutation in the normal gene. Each child of an HD parent has a 50-50 chance of inheriting the HD gene. If a child does not inherit the HD gene, he or she will not develop the disease and cannot pass it to subsequent generations. A person who inherits the HD gene will sooner or later develop the disease. Whether one child inherits the gene has no bearing on whether others will or will not inherit the gene.

Some early symptoms of HD are mood swings, depression, irritability or trouble driving, learning new things, remembering a fact, or making a decision. As the disease progresses, concentration on intellectual tasks becomes increasingly difficult and the patient may have difficulty feeding himself or herself and swallowing. The rate of disease progression and the age of onset vary from person to person. A genetic test, coupled with a complete medical history and neurological and laboratory tests, helps physicians diagnose HD. Presymptomic testing is available for individuals who are at risk for carrying the HD gene. In 1 to 3 percent of individuals with HD, no family history of HD can be found.

CIRM Grants Targeting Huntington's Disease

Title Total Funds
MSC engineered to produce BDNF for the treatment of Huntington's disease $99,248
Triplet Repeat Instability in Human iPSCs $1,755,861
A hESc-based Development Candidate for Huntington's Disease $3,799,817
Sustained siRNA production from human MSC to treat Huntingtons Disease and other neurodegenerative disorders $2,753,559
New Cell Lines for Huntington's Disease $1,369,800

CIRM Huntington's Disease Videos

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