Glucocerebrosidase pharmacological chaperones attenuate α-synuclein-induced neurotoxicity in chronic cortico-striatal slices
Antonino Iurato La Rocca, Elisabetta Gerace, Miriam Cerullo, Giuseppe Ranieri, Lorenzo Curti, Valentina Ferrara, Francesca Clemente, Camilla Matassini, Francesca Cardona, Andrea Goti, Rodolfo Tonin, Amelia Morrone, Guido Mannaioni, Alessio Masi · FENS Forum 2024
Background: Parkinson's disease (PD) is characterized by the formation of proteinaceous aggregates of α-synuclein (α-syn) in cell bodies and neurites of Substantia Nigra pars compacta (SNpc) dopaminergic neurons, eventually causing cell death. Interestingly, defective activity or mutations in GBA1, the gene encoding the lysosomal enzyme Glucocerebrosidase (GCase), are the strongest risk factor to develop PD. Based on this evidence, reverting the cytological and molecular abnormalities caused by GCase mutations or enhancing the activity of the wild type enzyme may represent a feasible therapeut
ePoster · Neuroscience