Elucidation of pathogenesis and monitoring of treatment
- Establish standardized disease-based cell models (hiPSCs and others)
- Correlate omics profiling of hiPSC derived cells and in vivo animal models/patient samples
- Identify, prioritize and test disease modifiers in cell assays and animal models
- Develop molecular therapies for plausible targets in cell and animal models
- Facilitate translation of preclinical approaches to clinical trial stage
