Discovery of Early Neuron Dysfunction Could Lead to Spinocerebellar Ataxia Therapy

A neuronal abnormality discovered in mice suffering from spinocerebellar ataxia type 3 could help develop therapies for people with the same condition.

Researchers have found an abnormality in the neurons of mice suffering from spinocerebellar ataxia type 3 (SCA3) that could help develop therapies for people with the same condition. The research, published this week in the Journal of Neuroscience, found that the mice’s Purkinje neurons fire in an aberrational manner long before they begin to die, which is a typical consequence of the condition.

Up to 20,000 people in the US have SCA3, which is also known as Machado-Joseph disease. Symptoms include slowly increasing clumsiness and difficulty walking and maintaining balance as well as difficulty with swallowing and aspiration. It is currently untreatable with medication or other therapies.

The researchers found that activation of a specific type of potassium channel helped reverse motor dysfunction in the afflicted mice and could be helpful to people suffering from the disorder.

Sources

Abnormality Discovered in Patients With Specific Ataxia That Could Be Target for Treatment [Press Release]

Early Changes in Cerebellar Physiology Accompany Motor Dysfunction in the Polyglutamine Disease Spinocerebellar Ataxia Type 3 (abstract) [Journal of Neuroscience]