Nanostructured Lipid Carriers for the delivery of Idebenone in Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay

The aim of this work was to evaluate the antioxidant activity of Idebenone-loaded Nanostructured Lipid Carriers (IDE-NLCs) in the treatment of Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS), a neurodegenerative disease characterized by autosomal recessive mutations in sacsin gene (SACS), encoding a protein localized at the mitochondrial surface and involved in mitochondrial dynamics. The first part of the work was focused on the synthesis and characterization of IDE-NLCs, on the evaluation of their loading efficiency and antioxidant activity and on cellular internalization analysis, performed in parallel on healthy and ARSACS derived primary skin fibroblasts. The ability of IDE-NLCs to cross the blood brain barrier (BBB) was evaluated in vitro. Finally, the work was focused on the development of technologies for targeted delivery of drugs to the mitochondria, for this purpose a peptide for mitochondrial targeting was exploited