RNA metabolism dysfunction and RNA-targeting therapeutics in neurodegeneration
The nervous system exhibits extremely complex RNA processing regulation. Dysfunction of RNA metabolism has emerged to play crucial roles in multiple neurological diseases. Mutations and pathologies of several RNA-binding proteins are found to be associated with neurodegeneration, including amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and Alzheimer’s disease (AD). Additionally, RNA-mediated toxicity can arise from microsatellite repeat instability in the human genome. Expanded repeat-containing RNAs could potentially induce neuronal toxicity by disrupting protein and RNA homeostasis through diverse mechanisms.
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The Sun lab is dedicated to exploring the dysfunction of RNA processing pathways to uncover the mechanisms of toxicity and the molecular basis of cell type-selective vulnerability. Another major focus of the group is identifying disease modifiers using advanced functional genomic screening platforms. We aim to translate the mechanistic findings at molecular level into therapeutic target development to advance treatment strategies for neurodegenerative diseases.
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