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Brain Channelopathies – Target Validation and Novel Therapeutic Strategies

  • Project Leaders:
  • Institutions:
  • Program/Competition: Partner Programs
  • Fiscal Year: 2013
  • Status: Closed

This project expected to provide new insights into how particular genetic alterations affect calcium channel properties resulting in the disruption of normal brain functions, and causing serious diseases of the nervous system. The team made a number of important discoveries that provide advancements to several fields. These include: 1) the first design, development and successful implementation of lipid nanoparticles as a vehicle towards the selective knock-down of disease-related targets in vivo. The work resulted in a number of high-impact publications and a PCT patent mapping crucial structural regions implicated in aberrant firing, and focusing on novel methods for treating brain edema; 2) the diffusion MRI technology developed represents a novel methodology for tracking the progression of spreading depression (SD) in live animals, and provided direct evidence to suggest the use of pregabalin as a potential therapeutics for patients with migraine; 3) the team developed the state-of-the-art 3rd generation MinION sequencing platform, with Oxford Nanopore Technologies. This platform has enormous implications for numerous fields including real time examination of direct RNA expression and epigenetic modifications in the CNS.