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CGT-102 is a preclinical-stage RNA interference (RNAi) gene therapy developed by CavGene Inc. in collaboration with N-Vector and Iron Horse Diagnostics. It is designed to treat levodopa-induced dyskinesia (LID), a common motor complication in patients with Parkinson's disease. The therapy specifically targets and knocks down the expression of the Cav1.3 voltage-gated L-type calcium channel (encoded by the CACNA1D gene) in the striatum. Research indicates that the upregulation of Cav1.3 channels contributes to the maladaptive synaptic plasticity responsible for dyskinesia. By utilizing a viral vector to deliver RNAi triggers, CGT-102 aims to restore normal calcium signaling and alleviate dyskinetic movements without compromising the motor benefits of levodopa therapy.
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