Science

L-type calcium channels (LTCCs) have distinct physiological functions within the brain that include the regulation of neuronal excitability and synaptic plasticity and the modulation of intracellular Ca^2+-dependent 2nd messenger signaling pathways affecting gene-transcription. LTCCs have been implicated in the pathophysiology of multiple neurological disorders such as Parkinson’s disease, epilepsy, migraine, ataxia, drug addiction, and Alzheimer’s disease. Consequently, the selective modulation of LTCCs in the brain presents an attractive approach for powerful long-term treatment of some of the most devastating neurodegenerative disorders. In addition, the company’s viral vector based RNA interference (RNAi) gene therapy approach makes it possible to address these otherwise undruggable targets with low risk for off-target adverse effects. Our silencing (si)RNA therapeutic is precisely delivered to targeted neurons in the specific brain region of interest by MRI guided stereotactic injection.