Abstract
Progressive Supranuclear palsy (PSP), a rare form of parkinsonism, is a sporadic and progressive neurodegenerative disease linked to the accumulation of Tau protein within neural cells (taupathies). No effective therapy is to date available for this no-option neurological disorder. Bone marrow (BM) mesenchymal stromal cells (MSC) have recently gained attention for therapeutic interventions due to their anti-inflammatory, anti-apoptotic and trophic properties, also for neurological disorders. In this context, our hospital-based GMP-approved facility provides autologous BMMSC manufactured as advanced therapy medicinal products (ATMPs) in a phase I clinical study (NCT01824121) with the aim to evaluate the safety of intra-arterial infusion of autologous BMMSC in patients with PSP. In connection with this project, we set up a parallel in vitro study with the aim to investigate MSC biology, and to disclose if any disease-linked defect occurs also in the non-neuronal mesenchymal compartment.
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