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Elvira De Leonibus, PhD - "Targeting early disease’ mechanisms in neurodegenerative disorders: the special case of mucopolysaccharidosis type IIIA"

Head of Behavioral Core, TIGEM and PI Neuropsychopharmacology Lab, Institute of Cellular Biology and Neurobiology, National Research Council, Italy
When Apr 02, 2019
from 12:00 PM to 01:30 PM
Where Tigem, Vesuvius Auditorium
Contact Name
Contact Phone 081-19230659
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Short CV

Abstract
Neurodegenerative diseases are debilitating and largely untreatable conditions. Most of them, such as Parkinson’ and Alzheimer’ disease, are strongly linked with age; inherited neurodegenerative lysosomal storage disorders, such as mucopolysaccharidosis type III (MPS-IIIA), manifest during childhood.
One of the biggest challenges, to fight neurodegenerative disorders, is the identification of early signs of the pathology and associated disease’ mechanisms leading to neuronal loss to develop restorative or protective therapeutic approaches.
I will show how we address this challenge with a novel approach that uses early discrete behavioural dysfunctions to pinpoint synaptic and molecular disease’ mechanisms, while accounting for their intrinsic heterogeneity in the complex and evolving phenotypes of neurodegenerative disorders.
I will show how this approach allowed us to identify completely novel disease’ pathways in Parkinson’ disease and MPS-IIIA and how this approach permits the “repositioning” of treatments among different neurodegenerative and other chronic diseases.

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