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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.

Collaborators

Picture Name Organization
Peter Szatmari, MD, MSc, FRCPC Picture 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 Picture Evdokia Anagnostou, MD Senior Clinician Scientist, Bloorview Research Institute
Stephen Scherer, PhD, FRSC Picture 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 Picture Mick Bhatia, PhD Director and Senior Scientist, Stem Cell and Cancer Research Institute (SCC-RI)

Funders

Impacts

Karun Singh

Karun Singh

Principal Investigator