Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic von Sardi, S. Pablo, Cedarbaum, Jesse M., Brundin, Patrik

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Cerebrospinal fluid proteomics implicates the granin family in Parkinson’s disease von Rotunno, Melissa S., Lane, Monica, Zhang, Wenfei, Wolf, Pavlina, Oliva, Petra, Viel, Catherine, Wills, Anne-Marie, Alcalay, Roy N., Scherzer, Clemens R., Shihabuddin, Lamya S., Zhang, Kate, Sardi, S. Pablo

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iPSC-derived neurons from GBA1-associated Parkinson’s disease patients show autophagic defects and impaired calcium homeostasis von Schöndorf, David C., Aureli, Massimo, McAllister, Fiona E., Hindley, Christopher J., Mayer, Florian, Schmid, Benjamin, Sardi, S. Pablo, Valsecchi, Manuela, Hoffmann, Susanna, Schwarz, Lukas Kristoffer, Hedrich, Ulrike, Berg, Daniela, Shihabuddin, Lamya S., Hu, Jing, Pruszak, Jan, Gygi, Steven P., Sonnino, Sandro, Gasser, Thomas, Deleidi, Michela

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Sterol auto-oxidation adversely affects human motor neuron viability and is a neuropathological feature of amyotrophic lateral sclerosis von Dodge, James C., Yu, Jinlong, Sardi, S. Pablo, Shihabuddin, Lamya S.

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ER Stress and Autophagic Perturbations Lead to Elevated Extracellular α-Synuclein in GBA-N370S Parkinson's iPSC-Derived Dopamine Neurons von Fernandes, Hugo J.R., Hartfield, Elizabeth M., Christian, Helen C., Emmanoulidou, Evangelia, Zheng, Ying, Booth, Heather, Bogetofte, Helle, Lang, Charmaine, Ryan, Brent J., Sardi, S. Pablo, Badger, Jennifer, Vowles, Jane, Evetts, Samuel, Tofaris, George K., Vekrellis, Kostas, Talbot, Kevin, Hu, Michele T., James, William, Cowley, Sally A., Wade-Martins, Richard

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Neutral Lipid Cacostasis Contributes to Disease Pathogenesis in Amyotrophic Lateral Sclerosis von Dodge, James C., Jensen, Elizabeth H., Yu, Jinlong, Sardi, S. Pablo, Bialas, Allison R., Taksir, Tatyana, Bangari, Dinesh S., Shihabuddin, Lamya S.

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Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models von Sardi, S. Pablo, Viel, Catherine, Clarke, Jennifer, Treleaven, Christopher M., Richards, Amy M., Park, Hyejung, Olszewski, Maureen A., Dodge, James C., Marshall, John, Makino, Elina, Wang, Bing, Sidman, Richard L., Cheng, Seng H., Shihabuddin, Lamya S.

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Acid β-glucosidase mutants linked to gaucher disease, parkinson disease, and lewy body dementia alter α-synuclein processing von Cullen, Valerie, Sardi, S. Pablo, Ng, Juliana, Xu, You-Hai, Sun, Ying, Tomlinson, Julianna J., Kolodziej, Piotr, Kahn, Ilana, Saftig, Paul, Woulfe, John, Rochet, Jean-Christophe, Glicksman, Marcie A., Cheng, Seng H., Grabowski, Gregory A., Shihabuddin, Lamya S., Schlossmacher, Michael G.

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New Era in disease modification in Parkinson's disease: Review of genetically targeted therapeutics von Sardi, S. Pablo, Simuni, Tanya

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Modulation of β-glucocerebrosidase increases α-synuclein secretion and exosome release in mouse models of Parkinson's disease von Papadopoulos, Vassilis E, Nikolopoulou, Georgia, Antoniadou, Ivi, Karachaliou, Antonia, Arianoglou, Giovanna, Emmanouilidou, Evangelia, Sardi, S Pablo, Stefanis, Leonidas, Vekrellis, Kostas

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Open science in precision medicine for neurodegenerative diseases von Leonard, Hampton L., Nalls, Mike A., Day-Williams, Aaron, Esmaeeli, Sahar, Jarreau, Paige, Bandres-Ciga, Sara, Heutink, Peter, Sardi, S. Pablo, Singleton, Andrew B.

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Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model von Viel, Catherine, Clarke, Jennifer, Kayatekin, Can, Richards, Amy M., Chiang, Ming Sum R., Park, Hyejung, Wang, Bing, Shihabuddin, Lamya S., Sardi, S. Pablo

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Safety and efficacy of venglustat in GBA1-associated Parkinson's disease: an international, multicentre, double-blind, randomised, placebo-controlled, phase 2 trial von Giladi, Nir, Alcalay, Roy N, Cutter, Gary, Gasser, Thomas, Gurevich, Tanya, Höglinger, Günter U, Marek, Kenneth, Pacchetti, Claudio, Schapira, Anthony H V, Scherzer, Clemens R, Simuni, Tanya, Minini, Pascal, Sardi, S Pablo, Peterschmitt, M Judith

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Gaucher-related synucleinopathies: the examination of sporadic neurodegeneration from a rare (disease) angle von Sardi, S Pablo, Cheng, Seng H, Shihabuddin, Lamya S

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CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy von Sardi, S. Pablo, Clarke, Jennifer, Kinnecom, Cathrine, Tamsett, Thomas J., Li, Lingyun, Stanek, Lisa M., Passini, Marco A., Grabowski, Gregory A., Schlossmacher, Michael G., Sidman, Richard L., Cheng, Seng H., Shihabuddin, Lamya S.

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Dysregulation of Mdm2 and Mdm4 alternative splicing underlies motor neuron death in spinal muscular atrophy von Van Alstyne, Meaghan, Simon, Christian M, Sardi, S Pablo, Shihabuddin, Lamya S, Mentis, George Z, Pellizzoni, Livio

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Presenilin-Dependent ErbB4 Nuclear Signaling Regulates the Timing of Astrogenesis in the Developing Brain von Sardi, S. Pablo, Murtie, Joshua, Koirala, Samir, Patten, Brooke A., Corfas, Gabriel

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Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies von Sardi, S. Pablo, Clarke, Jennifer, Viel, Catherine, Chan, Monyrath, Tamsett, Thomas J., Treleaven, Christopher M., Bu, Jie, Sweet, Lindsay, Passini, Marco A., Dodge, James C., Yu, W. Haung, Sidman, Richard L., Cheng, Seng H., Shihabuddin, Lamya S.

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The Mutation Matters: CSF Profiles of GCase, Sphingolipids, α‐Synuclein in PDGBA von Lerche, Stefanie, Schulte, Claudia, Wurster, Isabel, Machetanz, Gerrit, Roeben, Benjamin, Zimmermann, Milan, Deuschle, Christian, Hauser, Ann‐Kathrin, Böhringer, Judith, Krägeloh‐Mann, Ingeborg, Waniek, Katharina, Lachmann, Ingolf, Petterson, Xuan‐Mai T., Chiang, Ruby, Park, Hyejung, Wang, Bing, Liepelt‐Scarfone, Inga, Maetzler, Walter, Galasko, Douglas, Scherzer, Clemens R., Gasser, Thomas, Mielke, Michelle M., Hutten, Samantha J., Mollenhauer, Brit, Sardi, S. Pablo, Berg, Daniela, Brockmann, Kathrin

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Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis von Dodge, James C, Christopher M. Treleaven, Joshua Pacheco, Samantha Cooper, Channa Bao, Marissa Abraham, Mandy Cromwell, S. Pablo Sardi, Wei-Lien Chuang, Richard L. Sidman, Seng H. Cheng, Lamya S. Shihabuddin

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