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Searching for causes, biomarkers and therapies for motor neuronopathies/axonopathies

Abstract

Motor neuronopathies and axonopathies represent a large and heterogeneous group of neurological disorders characterized by primary damage either to the cell bodies or to the axonal extensions of Motor Neurons (MN). They may affect upper or lower MN. On clinical grounds, these conditions may affect children and adults, but in general follow a chronic course and deeply impact morbi-mortality. The major conditions included under this umbrella are: Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophies (SMAs) and Hereditary Spastic Paraplegias (HSPs). Despite their obvious clinical relevance, there are still many unanswered questions relative to the causes, mechanisms and treatment of these diseases. This is the main motivation for the current research application. Genetic abnormalities play a major role here. However, the genetic bases of 30-40% of HSPs and >50% of distal SMAs are still unclear. Likewise, biomarkers able to track progression are still lacking for ALS and HSP, which turns the design of clinical trials into a challenge. Advanced neuroimaging, particularly focusing in the spinal cord and using state-of-the-art acquisitions, emerge as promising candidates. Preliminary data - essentially in ALS - indicate that serum microRNAs are another putative biomarker, but so far, no consistent data have been published in HSP and SMA. Treatment for ALS, distal SMA and HSP remains as an unmet medical need. So, we intend to explore 2 therapeutic strategies: in a clinical setting, to investigate the effects of non-invasive neuromodulation for diseases with prominent spasticity (ie, HSPs) and in an experimental setting, to assess the potential benefits of mesenchymal stem cell-derived exosomes in ALS cell models. Our large patient database and established national as well as international collaborations set place for the successful completion of this audacious application. For the neuromuscular group at UNICAMP, this project will expand the research landscape with the inclusion of new tools, such as neuromodulation and cell-based experiments. (AU)

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