This technology identifies and functionally targets cerebellar circuit defects, specifically climbing fiber-Purkinje cell (CF-PC) synapses, to restore cerebellar functions and alleviate ataxia symptoms.
Cerebellar degeneration leads to severe ataxia symptoms such as imbalance, frequent falls, and loss of motor coordination. Current treatments manage symptoms but fail to address the root cause of the disease. In patients, climbing fiber-Purkinje cells (CF-PC) undergo early synaptic pruning followed by neuronal loss at later stages. However, the causal link between synaptic circuit changes and clinical symptoms remains unclear, limiting the development of effective therapies.
The technology demonstrates that loss of climbing fiber-Purkinje cell (CF-PC) synapses is a key driver of disrupted cerebellar function and ataxia symptoms. It identifies cerebellar alpha rhythm as a biomarker of circuit integrity and shows that restoring CF-PC synaptic activity normalizes cerebellar alpha rhythm and alleviates ataxia symptoms. This technology opens the door to therapies for cerebellar degeneration and other neurological disorders involving synaptic loss.
This technology has been validated in mouse models.
IR CU26133
Licensing Contact: Jerry Kokoshka
