WP01 - In Vitro System Development

Objectives

The major goal of this work package is to construct for the first time cellular models of ASD with construct validity. The objectives of this workpackage are:

  • To derive and to bank validated iPSCs from ASD and neurotypical individuals
  • To utilize iPSCs and other cellular systems to define cellular phenotypes associated with ASD
  • To provide an aetiological link between the genetic and neurodevelopmental components of ASD

WP Leads

Academic Lead: Professor Dr. Jack Price, King's College London, United Kingdom

EFPIA Lead: Dr. Daniel Ursu, Eli Lilly and Company Ltd., United Kingdom

Workpackage partners

  • King's College London
  • Pfizer Limited
  • F. Hoffmann- La Roche
  • University Medical Centre
  • Biozentrum, University of Basel
  • Janssen Pharmaceutica
  • Eli Lilly and Company Ltd.
  • Institut de Recherches Servier

Workpackage outcomes

  • A bank of biospies of ASD will be established and ASD-specific iPS cell lines will be generated.
  • Appropriate electrical properties of iPSC-derived neurons shall be demonstrated.
  • We will achieve consensus across centres on utility of stem cell-based phenotypic assay for ASD.
  • Additionally, we will achieve differentiated, characterized neurons from iPSCs.
  • We will demonstrate ASD-relevant phenotype in a stem cell model and generate samples for biomarker analysis.
  • WP01 will provide evidence for ‘two-hit’ hypothesis in ASD and will provide a new cellular assay drug discovery screening for ASD.
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