We investigate the molecular mechanisms underlying neurodegeneration. We are particularly interested in alterations in non-coding genetics and RNA metabolism of neurodegeneration. We study RNA-related neuro-protective functions and insufficiency in neuro-diseases, including amyotrophic lateral sclerosis (ALS) and FTD.
We use a multidisciplinary approaches, including computational data science, mouse genetics and iPS cell-derived human neurons. We also translate the molecular mechanisms we have discovered, that underlie neurodegeneration into potential therapies. We develop cell-free biomarkers and test a small molecule therapy based on our findings in a clinical trial.