In Trieste the Molecular Pathology Group (Buratti) investigates aberrant pre-mRNA processing defects that lead to neurodegeneration. In particular, this Group studies the biological properties of TDP43, a nuclear factor involved in amyotrophic lateral sclerosis and frontotemporal lobar degeneration. The Neurobiology Group (Feiguin) uses the fruitfly, Drosophila melanogaster, as a model organism having remarkable genetic conservation with humans, to study some of the most common neurological disorders, including Alzheimer’s and motor neuron diseases.
A long-term collaboration between the Molecular Pathology and Neurobiology Groups in Trieste continues to make excellent progress in our understanding of the role of TDP43 in neurodegeneration. In their latest study they showed that a specific group of hnRNP proteins have great potential for alleviating the deleterious effects of TDP43 loss-of-function, and most importantly this is conserved between both human and fly model systems (Appocher et al., 2017, Nuc. Acids Res., 45, 8026). This suggests that modulating specific levels of hnRNP expression might have potential therapeutic value for treatment of TDP43-associated neurological diseases.