Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
Hua, Yimin, Sahashi, Kentaro, Hung, Gene, Rigo, Frank, Passini, Marco A, Bennett, C Frank, Krainer, Adrian R
Published in Genes & development (01.08.2010)
Published in Genes & development (01.08.2010)
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Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
Passini, Marco A, Bu, Jie, Richards, Amy M, Kinnecom, Cathrine, Sardi, S Pablo, Stanek, Lisa M, Hua, Yimin, Rigo, Frank, Matson, John, Hung, Gene, Kaye, Edward M, Shihabuddin, Lamya S, Krainer, Adrian R, Bennett, C Frank, Cheng, Seng H
Published in Science translational medicine (02.03.2011)
Published in Science translational medicine (02.03.2011)
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Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies
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.
Published in Proceedings of the National Academy of Sciences - PNAS (26.02.2013)
Published in Proceedings of the National Academy of Sciences - PNAS (26.02.2013)
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CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy
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.
Published in Proceedings of the National Academy of Sciences - PNAS (19.07.2011)
Published in Proceedings of the National Academy of Sciences - PNAS (19.07.2011)
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SMN regulates axonal local translation via miR-183/mTOR pathway
Kye, Min Jeong, Niederst, Emily D, Wertz, Mary H, Gonçalves, Inês do Carmo G, Akten, Bikem, Dover, Katarzyna Z, Peters, Miriam, Riessland, Markus, Neveu, Pierre, Wirth, Brunhilde, Kosik, Kenneth S, Sardi, S Pablo, Monani, Umrao R, Passini, Marco A, Sahin, Mustafa
Published in Human molecular genetics (01.12.2014)
Published in Human molecular genetics (01.12.2014)
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A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice
Gan, Li, Wu, Leslie C.L., Wood, Jenna A., Yao, Monica, Treleaven, Chris M., Estrella, Nelsa L., Wentworth, Bruce M., Hanson, Gunnar J., Passini, Marco A.
Published in Molecular therapy. Nucleic acids (13.12.2022)
Published in Molecular therapy. Nucleic acids (13.12.2022)
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CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy
Passini, Marco A, Bu, Jie, Roskelley, Eric M, Richards, Amy M, Sardi, S Pablo, O'Riordan, Catherine R, Klinger, Katherine W, Shihabuddin, Lamya S, Cheng, Seng H
Published in The Journal of clinical investigation (01.04.2010)
Published in The Journal of clinical investigation (01.04.2010)
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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy
Passini, Marco A, Bu, Jie, Richards, Amy M, Treleaven, Christopher M, Sullivan, Jennifer A, O'Riordan, Catherine R, Scaria, Abraham, Kells, Adrian P, Samaranch, Lluis, San Sebastian, Waldy, Federici, Thais, Fiandaca, Massimo S, Boulis, Nicholas M, Bankiewicz, Krystof S, Shihabuddin, Lamya S, Cheng, Seng H
Published in Human gene therapy (01.07.2014)
Published in Human gene therapy (01.07.2014)
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Development of a single vector system that enhances trans-splicing of SMN2 transcripts
Coady, Tristan H, Baughan, Travis D, Shababi, Monir, Passini, Marco A, Lorson, Christian L
Published in PloS one (22.10.2008)
Published in PloS one (22.10.2008)
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Intraventricular Enzyme Replacement Improves Disease Phenotypes in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis
Chang, Michael, Cooper, Jonathan D, Sleat, David E, Cheng, Seng H, Dodge, James C, Passini, Marco A, Lobel, Peter, Davidson, Beverly L
Published in Molecular therapy (01.04.2008)
Published in Molecular therapy (01.04.2008)
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Systemic Administration of a Recombinant AAV1 Vector Encoding IGF-1 Improves Disease Manifestations in SMA Mice
Tsai, Li-Kai, Chen, Chien-Lin, Ting, Chen-Hung, Lin-Chao, Sue, Hwu, Wuh-Liang, Dodge, James C, Passini, Marco A, Cheng, Seng H
Published in Molecular therapy (01.08.2014)
Published in Molecular therapy (01.08.2014)
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Delivery of AAV-IGF-1 to the CNS Extends Survival in ALS Mice Through Modification of Aberrant Glial Cell Activity
Dodge, James C, Haidet, Amanda M, Yang, Wendy, Passini, Marco A, Hester, Mark, Clarke, Jennifer, Roskelley, Eric M, Treleaven, Christopher M, Rizo, Liza, Martin, Heather, Kim, Soo H, Kaspar, Rita, Taksir, Tatyana V, Griffiths, Denise A, Cheng, Seng H, Shihabuddin, Lamya S, Kaspar, Brian K
Published in Molecular therapy (01.06.2008)
Published in Molecular therapy (01.06.2008)
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IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice
Tsai, Li-Kai, Chen, Yi-Chun, Cheng, Wei-Cheng, Ting, Chen-Hung, Dodge, James C, Hwu, Wuh-Liang, Cheng, Seng H, Passini, Marco A
Published in Neurobiology of disease (01.01.2012)
Published in Neurobiology of disease (01.01.2012)
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Intraventricular Brain Injection of Adeno-Associated Virus Type 1 (AAV1) in Neonatal Mice Results in Complementary Patterns of Neuronal Transduction to AAV2 and Total Long-Term Correction of Storage Lesions in the Brains of β-Glucuronidase-Deficient Mice
Passini, Marco A, Watson, Deborah J, Vite, Charles H, Landsburg, Daniel J, Feigenbaum, Alyson L, Wolfe, John H
Published in Journal of Virology (01.06.2003)
Published in Journal of Virology (01.06.2003)
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Effective gene therapy for an inherited CNS disease in a large animal model
Vite, Charles H., McGowan, Joseph C., Niogi, Sumit N., Passini, Marco A., Drobatz, Kenneth J., Haskins, Mark E., Wolfe, John H.
Published in Annals of neurology (01.03.2005)
Published in Annals of neurology (01.03.2005)
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Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A
Bu, Jie, Ashe, Karen M, Bringas, John, Marshall, John, Dodge, James C, Cabrera-Salazar, Mario A, Forsayeth, John, Schuchman, Edward H, Bankiewicz, Krystof S, Cheng, Seng H, Shihabuddin, Lamya S., Passini, Marco A
Published in Molecular therapy (01.10.2012)
Published in Molecular therapy (01.10.2012)
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Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann–Pick A disease
Dodge, James C., Clarke, Jennifer, Treleaven, Christopher M., Taksir, Tatyana V., Griffiths, Denise A., Yang, Wendy, Fidler, Jonathan A., Passini, Marco A., Karey, Kenneth P., Schuchman, Edward H., Cheng, Seng H., Shihabuddin, Lamya S.
Published in Experimental neurology (01.02.2009)
Published in Experimental neurology (01.02.2009)
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Gene Transfer to the CNS Is Efficacious in Immune-primed Mice Harboring Physiologically Relevant Titers of Anti-AAV Antibodies
Treleaven, Christopher M, Tamsett, Thomas J, Bu, Jie, Fidler, Jonathan A, Sardi, S Pablo, Hurlbut, Gregory D, Woodworth, Lisa A, Cheng, Seng H, Passini, Marco A, Shihabuddin, Lamya S, Dodge, James C
Published in Molecular therapy (01.09.2012)
Published in Molecular therapy (01.09.2012)
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A Mouse Model of Classical Late-Infantile Neuronal Ceroid Lipofuscinosis Based on Targeted Disruption of the CLN2 Gene Results in a Loss of Tripeptidyl-Peptidase I Activity and Progressive Neurodegeneration
Sleat, David E, Wiseman, Jennifer A, El-Banna, Mukarram, Kim, Kwi-Hye, Mao, Qinwen, Price, Sandy, Macauley, Shannon L, Sidman, Richard L, Shen, Michael M, Zhao, Qi, Passini, Marco A, Davidson, Beverly L, Stewart, Gregory R, Lobel, Peter
Published in The Journal of neuroscience (13.10.2004)
Published in The Journal of neuroscience (13.10.2004)
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