Autism Spectrum Disorder Drug Screening
To develop the first Canadian high-throughput drug screening platform using autism spectrum disorder patient-derived neurons to ultimately move drugs to clinical trial testing.
Using novel SCC-RI developed technology, we are working to convert autism spectrum disorder (ASD) patient blood to neurons. The focus is on patient samples with mutations in high-risk ASD genes.
The goal of this project is to phenotype ASD human neurons using high-throughput techniques (e.g. electrophysiology and imaging) to determine the underlying pathophysiology of this disorder. Additionally, we are using patient-specific neurons to develop high-throughput drug screening approaches that will aid in identifying drugs that will address ASD-specific neuronal defects (e.g. synapse deficits). As current ASD treatments are focused on treating symptoms, shifting our focus to addressing the underlying cellular mechanisms represents an innovative step.
|Peter Szatmari, MD, MSc, FRCPC||Chief, Child and Youth Mental Health Collaborative, Centre for Addiction and Mental Health and The Hospital for Sick Children; Director, Division of Child and Youth Mental Health, University of Toronto|
|Evdokia Anagnostou, MD||Senior Clinician Scientist, Bloorview Research Institute|
|Stephen Scherer, PhD, FRSC||Director, The Centre for Applied Genomics, The Hospital for Sick Children; Director, The McLaughlin Centre for Molecular Medicine, University of Toronto|
|Mick Bhatia, PhD||Director and Senior Scientist, Stem Cell and Cancer Research Institute (SCC-RI)|