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High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources
Author / Creator:Haraszti, Reka A. ; Didiot, Marie-Cecile ; Sapp, Ellen ; Leszyk, John ; Shaffer, Scott A. ; Rockwell, Hannah E. ; Gao, Fei ; Narain, Niven R. ; DiFiglia, Marian ; Kiebish, Michael A. ; Aronin, Neil ; Khvorova, Anastasia
Publisher:Sweden: Taylor & Francis
Journal title:Journal of extracellular vesicles, 2016-01, Vol.5 (1), p.32570-n/a
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Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models
Author / Creator:Conroy, Faith ; Miller, Rachael ; Alterman, Julia F. ; Hassler, Matthew R. ; Echeverria, Dimas ; Godinho, Bruno M. D. C. ; Knox, Emily G. ; Sapp, Ellen ; Sousa, Jaquelyn ; Yamada, Ken ; Mahmood, Farah ; Boudi, Adel ; Kegel-Gleason, Kimberly ; DiFiglia, Marian ; Aronin, Neil ; Khvorova, Anastasia ; Pfister, Edith L.
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2022-10, Vol.13 (1), p.5802-5802, Article 5802
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Huntingtin associates with the actin cytoskeleton and α-actinin isoforms to influence stimulus dependent morphology changes
Author / Creator:Tousley, Adelaide ; Iuliano, Maria ; Weisman, Elizabeth ; Sapp, Ellen ; Richardson, Heather ; Vodicka, Petr ; Alexander, Jonathan ; Aronin, Neil ; DiFiglia, Marian ; Kegel-Gleason, Kimberly B.
Publisher:United States: Public Library of Science
Journal title:PloS one, 2019-02, Vol.14 (2), p.e0212337-e0212337
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Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain
Author / Creator:Alterman, Julia F ; Hall, Lauren M ; Coles, Andrew H ; Hassler, Matthew R ; Didiot, Marie-Cecile ; Chase, Kathryn ; Abraham, Jasmin ; Sottosanti, Emily ; Johnson, Emily ; Sapp, Ellen ; Osborn, Maire F ; Difiglia, Marian ; Aronin, Neil ; Khvorova, Anastasia
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy. Nucleic acids, 2015-12, Vol.4 (12), p.e266-e266, Article e266
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Kalirin Interacts with TRAPP and Regulates Rab11 and Endosomal Recycling
Author / Creator:Wang, Xiaolong ; Weng, Meiqian ; Ke, Yuting ; Sapp, Ellen ; DiFiglia, Marian ; Li, Xueyi
Publisher:Switzerland: MDPI AG
Journal title:Cells (Basel, Switzerland), 2020-05, Vol.9 (5), p.1132
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Aggregation of Huntingtin in Neuronal Intranuclear Inclusions and Dystrophic Neurites in Brain
Author / Creator:DiFiglia, Marian ; Sapp, Ellen ; Chase, Kathryn O. ; Davies, Stephen W. ; Bates, Gillian P. ; Vonsattel, J. P. ; Aronin, Neil
Publisher:Washington, DC: American Society for the Advancement of Science
Journal title:Science (American Association for the Advancement of Science), 1997-09, Vol.277 (5334), p.1990-1993
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Caspase 3-Cleaved N-Terminal Fragments of Wild-Type and Mutant Huntingtin are Present in Normal and Huntington's Disease Brains, Associate with Membranes, and Undergo Calpain-Dependent Proteolysis
Author / Creator:Kim, Yun J. ; Yi, Yong ; Sapp, Ellen ; Wang, Yumei ; Cuiffo, Ben ; Kegel, Kimberly B. ; Qin, Zheng-Hong ; Aronin, Neil ; DiFiglia, Marian
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2001-10, Vol.98 (22), p.12784-12789
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Behavioral deficits, early gliosis, dysmyelination and synaptic dysfunction in a mouse model of mucolipidosis IV
Author / Creator:Grishchuk, Yulia ; Sri, Sarmi ; Rudinskiy, Nikita ; Ma, Weiyuan ; Stember, Katherine G. ; Cottle, Matthew W. ; Sapp, Ellen ; Difiglia, Marian ; Muzikansky, Alona ; Betensky, Rebecca A. ; Wong, Andrew M. S. ; Bacskai, Brian J. ; Hyman, Bradley T. ; Kelleher, Raymond J. ; Cooper, Jonathan D. ; Slaugenhaupt, Susan A.
Publisher:England: BioMed Central Ltd
Journal title:Acta neuropathologica communications, 2014-09, Vol.2 (1), p.133-133, Article 133
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Axonal Transport of N-terminal Huntingtin Suggests Early Pathology of Corticostriatal Projections in Huntington Disease
Author / Creator:Sapp, Ellen ; Penney, John ; Young, Anne ; Aronin, Neil ; Vonsattel, Jean-Paul ; DiFiglia, Marian
Publisher:Hagerstown, MD: American Association of Neuropathologists, Inc
Journal title:Journal of neuropathology and experimental neurology, 1999-02, Vol.58 (2), p.165-173
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Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors
Author / Creator:Kegel, Kimberly B ; Sapp, Ellen ; Alexander, Jonathan ; Reeves, Patrick ; Bleckmann, Dorothee ; Sobin, Linsday ; Masso, Nicholas ; Valencia, Antonio ; Jeong, Hyunkyung ; Krainc, Dimitri ; Palacino, James ; Curtis, Daniel ; Kuhn, Rainer ; Betschart, Claudia ; Sena-Esteves, Miguel ; Aronin, Neil ; Paganetti, Paolo ; DiFiglia, Marian
Publisher:England: BioMed Central Ltd
Journal title:Molecular neurodegeneration, 2010-12, Vol.5 (1), p.58-58
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Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing
Author / Creator:Didiot, Marie-Cécile ; Hall, Lauren M ; Coles, Andrew H ; Haraszti, Reka A ; Godinho, Bruno MDC ; Chase, Kathryn ; Sapp, Ellen ; Ly, Socheata ; Alterman, Julia F ; Hassler, Matthew R ; Echeverria, Dimas ; Raj, Lakshmi ; Morrissey, David V ; DiFiglia, Marian ; Aronin, Neil ; Khvorova, Anastasia
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2016-10, Vol.24 (10), p.1836-1847
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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
Author / Creator:Alterman, Julia F. ; Godinho, Bruno M. D. C. ; Hassler, Matthew R. ; Ferguson, Chantal M. ; Echeverria, Dimas ; Sapp, Ellen ; Haraszti, Reka A. ; Coles, Andrew H. ; Conroy, Faith ; Miller, Rachael ; Roux, Loic ; Yan, Paul ; Knox, Emily G. ; Turanov, Anton A. ; King, Robert M. ; Gernoux, Gwladys ; Mueller, Christian ; Gray-Edwards, Heather L. ; Moser, Richard P. ; Bishop, Nina C. ; Jaber, Samer M. ; Gounis, Matthew J. ; Sena-Esteves, Miguel ; Pai, Athma A. ; DiFiglia, Marian ; Aronin, Neil ; Khvorova, Anastasia
Publisher:New York: Nature Publishing Group US
Journal title:Nature biotechnology, 2019-08, Vol.37 (8), p.884-894
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Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector
Author / Creator:Choudhury, Sourav R ; Harris, Anne F ; Cabral, Damien J ; Keeler, Allison M ; Sapp, Ellen ; Ferreira, Jennifer S ; Gray-Edwards, Heather L ; Johnson, Jacob A ; Johnson, Aime K ; Su, Qin ; Stoica, Lorelei ; DiFiglia, Marian ; Aronin, Neil ; Martin, Douglas R ; Gao, Guangping ; Sena-Esteves, Miguel
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2016-04, Vol.24 (4), p.726-735
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Assessing average somatic CAG repeat instability at the protein level
Author / Creator:Aviolat, Hubert ; Pinto, Ricardo Mouro ; Godschall, Elizabeth ; Murtha, Ryan ; Richey, Hannah E. ; Sapp, Ellen ; Vodicka, Petr ; Wheeler, Vanessa C. ; Kegel-Gleason, Kimberly B. ; DiFiglia, Marian
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2019-12, Vol.9 (1), p.19152-14, Article 19152
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Targeting Tau Mitigates Mitochondrial Fragmentation and Oxidative Stress in Amyotrophic Lateral Sclerosis
Author / Creator:Petrozziello, Tiziana ; Bordt, Evan A. ; Mills, Alexandra N. ; Kim, Spencer E. ; Sapp, Ellen ; Devlin, Benjamin A. ; Obeng-Marnu, Abigail A. ; Farhan, Sali M. K. ; Amaral, Ana C. ; Dujardin, Simon ; Dooley, Patrick M. ; Henstridge, Christopher ; Oakley, Derek H. ; Neueder, Andreas ; Hyman, Bradley T. ; Spires-Jones, Tara L. ; Bilbo, Staci D. ; Vakili, Khashayar ; Cudkowicz, Merit E. ; Berry, James D. ; DiFiglia, Marian ; Silva, M. Catarina ; Haggarty, Stephen J. ; Sadri-Vakili, Ghazaleh
Publisher:New York: Springer US
Journal title:Molecular neurobiology, 2022-01, Vol.59 (1), p.683-702
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Huntingtin associates with the actin cytoskeleton and [alpha]-actinin isoforms to influence stimulus dependent morphology changes
Author / Creator:Tousley, Adelaide ; Iuliano, Maria ; Weisman, Elizabeth ; Sapp, Ellen ; Richardson, Heather ; Vodicka, Petr ; Alexander, Jonathan ; Aronin, Neil ; DiFiglia, Marian ; Kegel-Gleason, Kimberly B
Publisher:Public Library of Science
Journal title:PloS one, 2019-02, Vol.14 (2), p.e0212337
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Glucose transporter 3 is a rab11-dependent trafficking cargo and its transport to the cell surface is reduced in neurons of CAG140 Huntington’s disease mice
Author / Creator:McClory, Hollis ; Williams, Dana ; Sapp, Ellen ; Gatune, Leah W ; Wang, Ping ; DiFiglia, Marian ; Li, Xueyi
Publisher:England: BioMed Central Ltd
Journal title:Acta neuropathologica communications, 2014-12, Vol.2 (1), p.179-179, Article 179
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The COOH-terminal domain of huntingtin interacts with RhoGEF kalirin and modulates cell survival
Author / Creator:McClory, Hollis ; Wang, Xiaolong ; Sapp, Ellen ; Gatune, Leah W. ; Iuliano, Maria ; Wu, Chiu-Yi ; Nathwani, Gina ; Kegel-Gleason, Kimberly B. ; DiFiglia, Marian ; Li, Xueyi
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2018-05, Vol.8 (1), p.8000-10, Article 8000
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Detection of HTTex1p by western blot and immunostaining of HD human and mouse brain using neo-epitope antibody P90 highlights impact of CAG repeat expansion on its size, solubility, and response to MS...
Author / Creator:Sapp, Ellen ; Boudi, Adel ; Iwanowicz, Andrew ; Belgrad, Jillian ; Miller, Rachael ; O'reilly, Daniel ; Yamada, Ken ; Deng, Yunping ; Marion, Joni ; Li, Xueyi ; Kegel-Gleason, Kimberly ; Khvorova, Anastasia ; Reiner, Anton ; Aronin, Neil ; Difiglia, Marian
Publisher:Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Journal title:bioRxiv, 2025-01
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Early whole-body mutant huntingtin lowering preserves proteins and lipids important for synapse function and white matter maintenance in the LacQ140 mouse model
Author / Creator:Sapp, Ellen ; Shing, Kai ; Boudi, Adel ; Liu, Sophia ; Seeley, Connor ; Marchionini, Deanna ; Difiglia, Marian ; Kegel-Gleason, Kimberly B
Publisher:Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Journal title:bioRxiv, 2023-01
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High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources

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Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models

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Huntingtin associates with the actin cytoskeleton and α-actinin isoforms to influence stimulus dependent morphology changes

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Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

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Kalirin Interacts with TRAPP and Regulates Rab11 and Endosomal Recycling

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Aggregation of Huntingtin in Neuronal Intranuclear Inclusions and Dystrophic Neurites in Brain

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Caspase 3-Cleaved N-Terminal Fragments of Wild-Type and Mutant Huntingtin are Present in Normal and Huntington's Disease Brains, Associate with Membranes, and Undergo Calpain-Dependent Proteolysis

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Behavioral deficits, early gliosis, dysmyelination and synaptic dysfunction in a mouse model of mucolipidosis IV

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Axonal Transport of N-terminal Huntingtin Suggests Early Pathology of Corticostriatal Projections in Huntington Disease

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Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors

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Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing

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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

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Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector

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Assessing average somatic CAG repeat instability at the protein level

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Targeting Tau Mitigates Mitochondrial Fragmentation and Oxidative Stress in Amyotrophic Lateral Sclerosis

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Huntingtin associates with the actin cytoskeleton and [alpha]-actinin isoforms to influence stimulus dependent morphology changes

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Glucose transporter 3 is a rab11-dependent trafficking cargo and its transport to the cell surface is reduced in neurons of CAG140 Huntington’s disease mice

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The COOH-terminal domain of huntingtin interacts with RhoGEF kalirin and modulates cell survival

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Detection of HTTex1p by western blot and immunostaining of HD human and mouse brain using neo-epitope antibody P90 highlights impact of CAG repeat expansion on its size, solubility, and response to MS...

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Early whole-body mutant huntingtin lowering preserves proteins and lipids important for synapse function and white matter maintenance in the LacQ140 mouse model

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