WP01 - In Vitro System Development


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.