The hexosamine biosynthetic pathway rescues lysosomal dysfunction in Parkinson’s disease patient iPSC derived midbrain neurons von Wani, Willayat Y., Zunke, Friederike, Belur, Nandkishore R., Mazzulli, Joseph R.

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AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease von Chen, Xin, Dong, Thomas, Hu, Yuhui, Shaffo, Frances C, Belur, Nandkishore R, Mazzulli, Joseph R, Gray, Steven J

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Pregnancy-induced amelioration of muscular dystrophy phenotype in mdx mice via muscle membrane stabilization effect of glucocorticoid von Shimizu-Motohashi, Yuko, Asakura, Yoko, Motohashi, Norio, Belur, Nandkishore R, Baumrucker, Michael G, Asakura, Atsushi

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Reversible Conformational Conversion of α-Synuclein into Toxic Assemblies by Glucosylceramide von Zunke, Friederike, Moise, Alexandra C., Belur, Nandkishore R., Gelyana, Eilrayna, Stojkovska, Iva, Dzaferbegovic, Haris, Toker, Nicholas J., Jeon, Sohee, Fredriksen, Kristina, Mazzulli, Joseph R.

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Rescue of α-synuclein aggregation in Parkinson’s patient neurons by synergistic enhancement of ER proteostasis and protein trafficking von Stojkovska, Iva, Wani, Willayat Y., Zunke, Friederike, Belur, Nandkishore R., Pavlenko, Egor A., Mwenda, Nkatha, Sharma, Karan, Francelle, Laetitia, Mazzulli, Joseph R.

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Nuclear aggregates of NONO/SFPQ and A-to-I-edited RNA in Parkinson’s disease and dementia with Lewy bodies von Belur, Nandkishore R., Bustos, Bernabe I., Lubbe, Steven J., Mazzulli, Joseph R.

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Stress-Induced Cellular Clearance Is Mediated by the SNARE Protein ykt6 and Disrupted by α-Synuclein von Cuddy, Leah K., Wani, Willayat Y., Morella, Martino L., Pitcairn, Caleb, Tsutsumi, Kotaro, Fredriksen, Kristina, Justman, Craig J., Grammatopoulos, Tom N., Belur, Nandkishore R., Zunke, Friederike, Subramanian, Aarthi, Affaneh, Amira, Lansbury, Peter T., Mazzulli, Joseph R.

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Recombinant pro-CTSD (cathepsin D) enhances SNCA/α-Synuclein degradation in α-Synucleinopathy models von Prieto Huarcaya, Susy, Drobny, Alice, Marques, André R. A., Di Spiezio, Alessandro, Dobert, Jan Philipp, Balta, Denise, Werner, Christian, Rizo, Tania, Gallwitz, Lisa, Bub, Simon, Stojkovska, Iva, Belur, Nandkishore R., Fogh, Jens, Mazzulli, Joseph R, Xiang, Wei, Fulzele, Amitkumar, Dejung, Mario, Sauer, Markus, Winner, Beate, Rose-John, Stefan, Arnold, Philipp, Saftig, Paul, Zunke, Friederike

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Preclinical results in rodents strongly support clinical evaluation of scAAV9/MFSD8 as a potential gene therapy for CLN7 patients von Chen, Xin, Shaffo, Frances, Dong, Thomas, Hu, Yuhui, Belur, Nandkishore R., Mazzulli, Joseph R., Gray, Steven J.

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Preclinical results in rodents strongly support clinical evaluation of scAAV9/ as a potential gene therapy for CLN7 patients von Chen, Xin, Shaffo, Frances, Dong, Thomas, Hu, Yuhui, Belur, Nandkishore R., Mazzulli, Joseph R., Gray, Steven J.

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Preclinical efficacy and safety evaluation of scAAV9/CLN7 gene replacement therapy in rodents von Chen, Xin, Shaffo, Frances, Dong, Thomas, Hu, Yuhui, Belur, Nandkishore R., Mazzulli, Joseph R., Gray, Steven J.

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Stress-induced cellular clearance is mediated by SNARE protein ykt6 and disrupted by a-synuclein von Cuddy, Leah K., Wani, Willayat Y., Morella, Martino L., Pitcairn, Caleb, Tsutsumi, Kotaro, Fredriksen, Kristina, Justman, Craig J., Grammatopoulos, Tom N., Belur, Nandkishore R., Zunke, Friederike, Subramanian, Aarthi, Affaneh, Amira, Lansbury, Peter T., Mazzulli, Joseph R.

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Recombinant pro-CTSD (cathepsin D) enhances SNCA/α-Synuclein degradation in α-Synucleinopathy models von Huarcaya, Susy Prieto, Drobny, Alice, Marques, André R. A., Di Spiezio, Alessandro, Dobert, Jan Philipp, Balta, Denise, Werner, Christian, Rizo, Tania, Gallwitz, Lisa, Bub, Simon, Stojkovska, Iva, Belur, Nandkishore R., Fogh, Jens, Mazzulli, Joseph R, Xiang, Wei, Fulzele, Amitkumar, Dejung, Mario, Sauer, Markus, Winner, Beate, Rose-John, Stefan, Arnold, Philipp, Saftig, Paul, Zunke, Friederike

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