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Co-clinical trials of autism in mice and humans

Principal Investigator:
  • Zsuzsa Lindenmaier,

    University of Toronto

  • Hospital for Sick Children

Project Overview

Autism is a neuro-developmental disorder characterized by social/communication deficits and repetitive behaviours. It is very heterogeneous, with over 250 genes implicated, but is highly heritable. The current therapeutic approach is frequently ineffective partly because often a single treatment is given to a heterogeneous patient population. This project will address the lack of individualized treatment in autism through a parallel co-clinical trial of promising autism treatments in mice and humans. Treatments will be oxytocin, a drug associated with increased social behaviours, and Tideglusib, a drug that acts on a protein downstream of the most common single gene cause of an autism-related disorder. Both humans (phase II clinical trial) and mice will be treated with promising drugs, and analyzed and compared on behavioural, genomic, and advanced, high-resolution imaging measures. Ms. Lindenmaier hypothesizes that this will allow the identification of both genetic and phenotypic information that can be used to stratify individuals with autism into responders and non-responders for specific treatment options. Ultimately, she hopes to make the first steps towards a personalized medicine approach in autism in which there are defined effective therapies for subpopulations of the disorder.