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New Drug Targets for Brain Diseases


Identifying new molecular targets driving brain disease is essential to developing biomarkers and precision therapeutics.


FutureNeuro researchers discovered a new microRNA linked to epilepsy (miR-335) which targets voltage-gated sodium channels. This microRNA normally functions to restrain brain excitability and the study showed that a gene therapy (AAV9) carrying miR-335 delivered to the hippocampus of mice protected against seizures in a preclinical model. The study also discovered that cannabidiol (CBD), a new treatment for Dravet syndrome, alters expression of microRNAs in the mouse brain and may contribute to the drug’s mechanism of action. The study also included analysis of which gene transcripts are targeted by microRNAs in the hippocampus of TLE patients, detecting over 7,000 transcripts controlled by over 200 different microRNAs.

Potential impact

The discovery has led to a patent application on the targeting of this molecule as a potential treatment for epilepsies. By working with business development and technology transfer experts in the Centre, we aim to establish a potential spin-out company that will develop a microRNA-based gene therapy for epilepsy.

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