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Proceedings of the National Academy of Sciences of the United States of America|February 23, 2017
Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy modelsS Pablo Sardi, Catherine Viel, Jennifer Clarke, et al.
Proceedings of the National Academy of Sciences of the United States of America|January 9, 2013
Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathiesS Pablo Sardi, Jennifer Clarke, Catherine Viel, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 12, 2025
Artificial enforcement of the unfolded protein response reduces disease features in multiple preclinical models of ALS/FTDVicente Valenzuela, Daniela Becerra, José I Astorga, et al.
Science Translational Medicine|March 4, 2011
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophyMarco A Passini, Jie Bu, Amy M Richards, et al.
Neurobiology of Aging|March 30, 2021
LRRK2 p.M1646T is associated with glucocerebrosidase activity and with Parkinson's diseaseYuri L Sosero, Eric Yu, Lynne Krohn, et al.
The Journal of Pharmacology and Experimental Therapeutics|September 7, 2023
Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe MiceAllyson Anding, Sofia Kinton, Kaitlyn Baranowski, et al.
Human Molecular Genetics|July 25, 2014
SMN regulates axonal local translation via miR-183/mTOR pathwayMin Jeong Kye, Emily D Niederst, Mary H Wertz, et al.
Annals of Neurology|April 8, 2011
Acid β-glucosidase mutants linked to Gaucher disease, Parkinson disease, and Lewy body dementia alter α-synuclein processingValerie Cullen, S Pablo Sardi, Juliana Ng, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 11, 2025
AAV gene therapy for GBA1-related diseasesSwathi Ayloo, Jae Cheon Ryu, Shih-Ching Chou, et al.
Nature Communications|June 7, 2014
iPSC-derived neurons from GBA1-associated Parkinson's disease patients show autophagic defects and impaired calcium homeostasisDavid C Schöndorf, Massimo Aureli, Fiona E McAllister, et al.
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