Understanding the molecular mechanisms of ALS and FTD to inspire targeted therapies
We are a diverse team of neuroscientists, molecular biologists, biochemists, RNA biologists and bioinformaticians, joined by our common interest in the molecular mechanisms of neurodegeneration. At the PolyLab, we study amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which are heterogenous and fatal neurodegenerative diseases. ALS and FTD are characterized by the accumulation of RNA-binding proteins, such as TDP-43 and FUS, as well as non-canonically translated dipeptide repeat proteins. The molecular mechanisms that trigger aggregation of ALS/FTD-linked proteins, the basis of disease heterogeneity and the mechanisms of neurotoxicity remain elusive. The aim of our research is to address these important questions using a multidisciplinary approach, working at the molecular, cellular and organismal level. Our vision is to gain deep mechanistic understanding of ALS and FTD to inspire rational design of target-based therapies for these devastating diseases.