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
Academic Lead: Professor Dr. Jack Price, King's College London, United Kingdom
EFPIA Lead: Dr. Daniel Ursu, Eli Lilly and Company Ltd., United Kingdom
- 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
- 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.