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Microglia Induce Motor Neuron Death via the Classical NF-κB Pathway in Amyotrophic Lateral Sclerosis
Author / Creator:Frakes, Ashley E. ; Ferraiuolo, Laura ; Haidet-Phillips, Amanda M. ; Schmelzer, Leah ; Braun, Lyndsey ; Miranda, Carlos J. ; Ladner, Katherine J. ; Bevan, Adam K. ; Foust, Kevin D. ; Godbout, Jonathan P. ; Popovich, Phillip G. ; Guttridge, Denis C. ; Kaspar, Brian K.
Publisher:United States: Elsevier Inc
Journal title:Neuron (Cambridge, Mass.), 2014-03, Vol.81 (5), p.1009-1023
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Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Author / Creator:Meyer, Kathrin ; Ferraiuolo, Laura ; Schmelzer, Leah ; Braun, Lyndsey ; McGovern, Vicki ; Likhite, Shibi ; Michels, Olivia ; Govoni, Alessandra ; Fitzgerald, Julie ; Morales, Pablo ; Foust, Kevin D ; Mendell, Jerry R ; Burghes, Arthur H M ; Kaspar, Brian K
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2015-03, Vol.23 (3), p.477-487
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Translational profiling identifies a cascade of damage initiated in motor neurons and spreading to glia in mutant SOD1-mediated ALS
Author / Creator:Sun, Shuying ; Sun, Ying ; Ling, Shuo-Chien ; Ferraiuolo, Laura ; McAlonis-Downes, Melissa ; Zou, Yiyang ; Drenner, Kevin ; Wang, Yin ; Ditsworth, Dara ; Tokunaga, Seiya ; Kopelevich, Alex ; Kaspar, Brian K. ; Lagier-Tourenne, Clotilde ; Cleveland, Don W.
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2015-12, Vol.112 (50), p.E6993-E7002
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Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS
Author / Creator:Meyer, Kathrin ; Ferraiuolo, Laura ; Miranda, Carlos J. ; Likhite, Shibi ; McElroy, Sohyun ; Renusch, Samantha ; Ditsworth, Dara ; Lagier-Tourenne, Clotilde ; Smith, Richard A. ; Ravits, John ; Burghes, Arthur H. ; Shaw, Pamela J. ; Cleveland, Don W. ; Kolb, Stephen J. ; Kaspar, Brian K.
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2014-01, Vol.111 (2), p.829-832
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Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism
Author / Creator:Ferraiuolo, Laura ; Meyer, Kathrin ; Sherwood, Thomas W. ; Vick, Jonathan ; Likhite, Shibi ; Frakes, Ashley ; Miranda, Carlos J. ; Braun, Lyndsey ; Heath, Paul R. ; Pineda, Ricardo ; Beattie, Christine E. ; Shaw, Pamela J. ; Askwith, Candice C. ; McTigue, Dana ; Kaspar, Brian K.
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2016-10, Vol.113 (42), p.E6496-E6505
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A role for glia in the progression of Rett’s syndrome
Author / Creator:Lioy, Daniel T. ; Garg, Saurabh K. ; Monaghan, Caitlin E. ; Raber, Jacob ; Foust, Kevin D. ; Kaspar, Brian K. ; Hirrlinger, Petra G. ; Kirchhoff, Frank ; Bissonnette, John M. ; Ballas, Nurit ; Mandel, Gail
Publisher:London: Nature Publishing Group UK
Journal title:Nature (London), 2011-07, Vol.475 (7357), p.497-500
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Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS
Author / Creator:Bravo-Hernandez, Mariana ; Tadokoro, Takahiro ; Navarro, Michael R. ; Platoshyn, Oleksandr ; Kobayashi, Yoshiomi ; Marsala, Silvia ; Miyanohara, Atsushi ; Juhas, Stefan ; Juhasova, Jana ; Skalnikova, Helena ; Tomori, Zoltan ; Vanicky, Ivo ; Studenovska, Hana ; Proks, Vladimir ; Chen, PeiXi ; Govea-Perez, Noe ; Ditsworth, Dara ; Ciacci, Joseph D. ; Gao, Shang ; Zhu, Wenlian ; Ahrens, Eric T. ; Driscoll, Shawn P. ; Glenn, Thomas D. ; McAlonis-Downes, Melissa ; Da Cruz, Sandrine ; Pfaff, Samuel L. ; Kaspar, Brian K. ; Cleveland, Don W. ; Marsala, Martin
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2020-01, Vol.26 (1), p.118-130
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Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis
Author / Creator:Song, SungWon ; Miranda, Carlos J ; Braun, Lyndsey ; Meyer, Kathrin ; Frakes, Ashley E ; Ferraiuolo, Laura ; Likhite, Shibi ; Bevan, Adam K ; Foust, Kevin D ; McConnell, Michael J ; Walker, Christopher M ; Kaspar, Brian K
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2016-04, Vol.22 (4), p.397-403
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ArticlesPeer ReviewedFull Text
Astrocytes from familial and sporadic ALS patients are toxic to motor neurons
Author / Creator:Haidet-Phillips, Amanda M ; Hester, Mark E ; Miranda, Carlos J ; Meyer, Kathrin ; Braun, Lyndsey ; Frakes, Ashley ; Song, SungWon ; Likhite, Shibi ; Murtha, Matthew J ; Foust, Kevin D ; Rao, Meghan ; Eagle, Amy ; Kammesheidt, Anja ; Christensen, Ashley ; Mendell, Jerry R ; Burghes, Arthur H M ; Kaspar, Brian K
Publisher:New York: Nature Publishing Group US
Journal title:Nature biotechnology, 2011-09, Vol.29 (9), p.824-828
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Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS
Author / Creator:Foust, Kevin D ; Salazar, Desirée L ; Likhite, Shibi ; Ferraiuolo, Laura ; Ditsworth, Dara ; Ilieva, Hristelina ; Meyer, Kathrin ; Schmelzer, Leah ; Braun, Lyndsey ; Cleveland, Don W ; Kaspar, Brian K
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2013-12, Vol.21 (12), p.2148-2159
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A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
Author / Creator:Mendell, Jerry R ; Sahenk, Zarife ; Malik, Vinod ; Gomez, Ana M ; Flanigan, Kevin M ; Lowes, Linda P ; Alfano, Lindsay N ; Berry, Katherine ; Meadows, Eric ; Lewis, Sarah ; Braun, Lyndsey ; Shontz, Kim ; Rouhana, Maria ; Clark, Kelly Reed ; Rosales, Xiomara Q ; Al-Zaidy, Samiah ; Govoni, Alessandra ; Rodino-Klapac, Louise R ; Hogan, Mark J ; Kaspar, Brian K
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2015-01, Vol.23 (1), p.192-201
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Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
Author / Creator:Mendell, Jerry R. ; Sahenk, Zarife ; Al-Zaidy, Samiah ; Rodino-Klapac, Louise R. ; Lowes, Linda P. ; Alfano, Lindsay N. ; Berry, Katherine ; Miller, Natalie ; Yalvac, Mehmet ; Dvorchik, Igor ; Moore-Clingenpeel, Melissa ; Flanigan, Kevin M. ; Church, Kathleen ; Shontz, Kim ; Curry, Choumpree ; Lewis, Sarah ; McColly, Markus ; Hogan, Mark J. ; Kaspar, Brian K.
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2017-04, Vol.25 (4), p.870-879
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ArticlesPeer ReviewedFull Text
SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits
Author / Creator:Hautbergue, Guillaume M. ; Castelli, Lydia M. ; Ferraiuolo, Laura ; Sanchez-Martinez, Alvaro ; Cooper-Knock, Johnathan ; Higginbottom, Adrian ; Lin, Ya-Hui ; Bauer, Claudia S. ; Dodd, Jennifer E. ; Myszczynska, Monika A. ; Alam, Sarah M. ; Garneret, Pierre ; Chandran, Jayanth S. ; Karyka, Evangelia ; Stopford, Matthew J. ; Smith, Emma F. ; Kirby, Janine ; Meyer, Kathrin ; Kaspar, Brian K. ; Isaacs, Adrian M. ; El-Khamisy, Sherif F. ; De Vos, Kurt J. ; Ning, Ke ; Azzouz, Mimoun ; Whitworth, Alexander J. ; Shaw, Pamela J.
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2017-07, Vol.8 (1), p.16063-18, Article 16063
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Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors
Author / Creator:Haidet, Amanda M ; Rizo, Liza ; Handy, Chalonda ; Umapathi, Priya ; Eagle, Amy ; Shilling, Chris ; Boue, Daniel ; Martin, Paul T ; Sahenk, Zarife ; Mendell, Jerry R ; Kaspar, Brian K
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2008-03, Vol.105 (11), p.4318-4322
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Rapid and Efficient Generation of Functional Motor Neurons From Human Pluripotent Stem Cells Using Gene Delivered Transcription Factor Codes
Author / Creator:Hester, Mark E ; Murtha, Matthew J ; Song, SungWon ; Rao, Meghan ; Miranda, Carlos J ; Meyer, Kathrin ; Tian, Jinbin ; Boulting, Gabriella ; Schaffer, David V ; Zhu, Michael X ; Pfaff, Samuel L ; Gage, Fred H ; Kaspar, Brian K
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2011-10, Vol.19 (10), p.1905-1912
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ArticlesPeer ReviewedFull Text
Retrograde Viral Delivery of IGF-1 Prolongs Survival in a Mouse ALS Model
Author / Creator:Kaspar, Brian K. ; Lladó, Jerònia ; Sherkat, Nushin ; Rothstein, Jeffrey D. ; Gage, Fred H.
Publisher:Washington, DC: American Association for the Advancement of Science
Journal title:Science (American Association for the Advancement of Science), 2003-08, Vol.301 (5634), p.839-842
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Directed evolution of adeno-associated virus to an infectious respiratory virus
Author / Creator:Excoffon, Katherine J.D.A ; Koerber, James T ; Dickey, David D ; Murtha, Matthew ; Keshavjee, Shaf ; Kaspar, Brian K ; Zabner, Joseph ; Schaffer, David V
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2009-03, Vol.106 (10), p.3865-3870
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AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice
Author / Creator:Dodge, James C ; Treleaven, Christopher M ; Fidler, Jonathan A ; Hester, Mark ; Haidet, Amanda ; Handy, Chalonda ; Rao, Meghan ; Eagle, Amy ; Matthews, Jennifer C ; Taksir, Tatyana V ; Cheng, Seng H ; Shihabuddin, Lamya S ; Kaspar, Brian K
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2010-12, Vol.18 (12), p.2075-2084
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Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes
Author / Creator:Gomes, Cátia ; Sequeira, Catarina ; Likhite, Shibi ; Dennys, Cassandra N ; Kolb, Stephen J ; Shaw, Pamela J ; Vaz, Ana R ; Kaspar, Brian K ; Meyer, Kathrin ; Brites, Dora
Publisher:Switzerland: MDPI AG
Journal title:Cells (Basel, Switzerland), 2022-04, Vol.11 (7), p.1186
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Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS
Author / Creator:Iannitti, Tommaso ; Scarrott, Joseph M. ; Likhite, Shibi ; Coldicott, Ian R.P. ; Lewis, Katherine E. ; Heath, Paul R. ; Higginbottom, Adrian ; Myszczynska, Monika A. ; Milo, Marta ; Hautbergue, Guillaume M. ; Meyer, Kathrin ; Kaspar, Brian K. ; Ferraiuolo, Laura ; Shaw, Pamela J. ; Azzouz, Mimoun
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy. Nucleic acids, 2018-09, Vol.12, p.75-88
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Showing 1 to 20 of 66 results

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Microglia Induce Motor Neuron Death via the Classical NF-κB Pathway in Amyotrophic Lateral Sclerosis

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Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

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Translational profiling identifies a cascade of damage initiated in motor neurons and spreading to glia in mutant SOD1-mediated ALS

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Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS

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Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism

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A role for glia in the progression of Rett’s syndrome

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Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS

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Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis

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Astrocytes from familial and sporadic ALS patients are toxic to motor neurons

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Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS

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A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

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Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes

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SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits

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Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors

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Rapid and Efficient Generation of Functional Motor Neurons From Human Pluripotent Stem Cells Using Gene Delivered Transcription Factor Codes

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Retrograde Viral Delivery of IGF-1 Prolongs Survival in a Mouse ALS Model

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Directed evolution of adeno-associated virus to an infectious respiratory virus

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AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice

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Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes

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Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS

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