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Pathogenic mechanisms of C9ORF72 repeat expansion in ALS and development of therapeutics

Principal Investigator:
  • Kessen Patten, Centre INRS-Institut Armand Frappier
  • ALS Society of Canada

Project Overview

Dr. Patten started his research career as a PhD student at the University of Alberta under the supervision of Dr. Declan Ali in 2004. There he trained in electrophysiology, cell biology and imaging using zebrafish as a model to study neurodevelopment. After publishing several manuscripts on his discoveries and receiving multiple awards, including national recognition for the outstanding quality of his PhD thesis, Dr. Patten pursued a postdoctoral fellowship in Montreal with Drs. Florina Moldovan and Pierre Drapeau. During that time, among other achievements, he developed zebrafish models of human disease including ALS, and used those models to develop a high-throughput method for drug discovery. This procedure was then used by Dr. Patten in the identification of pimozide as a lead compound in a translational pipeline that has led to a multi-centre Canadian clinical trial to start in 2017. The trial is being supported by the first ALS Canada-Brain Canada Arthur J. Hudson Translational Team Grant that was awarded in 2014.

In the initial years of his independence as an Assistant Professor, Dr. Patten will pursue the development and use of zebrafish models of the most common genetic cause of ALS, C9ORF72, as well as use of the high-throughput screening method to examine more promising compounds for further examination. As a key addition to his work, he has formed strong collaborations with international ALS experts with proficiency in developing motor neurons from induced pluripotent stem cells (iPSCs) that will undoubtedly strengthen the ability to translate zebrafish discoveries to the clinic via the use of human cells.