Human iPSC modeling and screening to investigate pathogenesis and disease modifiers of ALS

ALS and FTD are highly heterogenous diseases without effective treatment. This is largely due to a shortage of reliable preclinical models that can serve as the foundation for studying pathogenesis and exploring new therapeutic targets.

To close this research gap, we will use multi-dimensional approaches that build on sophisticated human iPSC-derived preclinical models with special focus on axonal degeneration, metabolic alteration, epigenetic modifications, and glial cell toxicity. We will explore the interactions and cross talks among these key pathological changes, and  eventually set up trustworthy genetic and phenotype large scale screen to identify new disease modifiers and therapeutic targets for ALS and FTD.  We aim to create a translational circle from clinical patients to lab investigations based on human centralized research.