The field of interest applies to the study of the molecular basis of neurodegenerative diseases and to the exploitation of novel RNA-based therapies. In particular, this project aims to discover new mechanisms of gene expression regulation controlled by non coding RNAs (ncRNAs) and how they impact in neuronal differentiation and in neurodegenerative disorders, such as Amyotrophic lateral sclerosis (ALS). This is one of the most common motor neuron disease whose pathogenic relevance is linked to altered RNA binding proteins (RBP), such as FUS and TDP-43, and defective RNA metabolism. Mutations in these proteins induce the formation of insoluble cytoplasmic aggregates, feature which is common to many neurodegenerative diseases.
Recent works indicate that also RNA and aberrant ribonucleoprotein (RNP) formation has an important pathogenic relevance in such diseases. How defective RNPs form, what are their integral components and which events trigger their appearance late in life are still unsolved issues.
This project aims to combine innovative computational approaches, imaging methodologies and powerful genetic tools to characterize RNP complexes and how they are modified in disease conditions. Moreover, we plan to develop new RNA molecules (aptamers) able to counteract aggregate formation and to impede pathological cascades driven by RNP assemblies.
This research holds promise for a significant increase in our understanding of basic molecular processes controlled by ncRNAs and should also constitute a largely unexplored territory for the development of novel therapeutics and diagnostics.
