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GSK3β is a new therapeutic target for myotonic dystrophy type 1

Christina Wei Department of Molecular Physiology and Biophysics; Baylor College of Medicine; Houston, TX USA

Karlie Jones Department of Molecular Physiology and Biophysics; Baylor College of Medicine; Houston, TX USA

Nikolai A Timchenko Pathology and Immunology; Huffington Center on Aging; Baylor College of Medicine; Houston, TX USA

Lubov Timchenko Department of Molecular Physiology and Biophysics; Baylor College of Medicine; Houston, TX USACorrespondence[email protected]

Article: e26555 | Received 31 Jul 2013, Accepted 20 Sep 2013, Published online: 26 Sep 2013

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https://doi.org/10.4161/rdis.26555

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Remco T. P. van Cruchten, Daniël van As, Jeffrey C. Glennon, Baziel G. M. van Engelen, Peter A. C. ‘t Hoen, K Okkersen, C Jimenez-Moreno, S Wenninger, F Daidj, S Cumming, R Littleford, D G Monckton, H Lochmüller, M Catt, C G Faber, A Hapca, P T Donnan, G Gorman, G Bassez, B Schoser, H Knoop, S Treweek, Derick G. Wansink, Francis Impens, Ralf Gabriels, Tine Claeys, Aymeric Ravel-Chapuis, Bernard J. Jasmin, Niamh Mahon, Sylvia Nieuwenhuis, Lennart Martens, Petr Novak, Denis Furling, Arie Baak, Genevieve Gourdon, Alex MacKenzie, Cecile Martinat, Nafisa Neault, Andreas Roos, Elise Duchesne, Renee Salz, Rachel Thompson, Sandrine Baghdoyan, Anu Mary Varghese, Paul Blom, Sally Spendiff & Alexander Manta. (2022) Clinical improvement of DM1 patients reflected by reversal of disease-induced gene expression in blood. BMC Medicine 20:1.
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Stojan Peric, Jovan Pesovic, Dusanka Savic-Pavicevic, Vidosava Rakocevic Stojanovic & Giovanni Meola. (2021) Molecular and Clinical Implications of Variant Repeats in Myotonic Dystrophy Type 1. International Journal of Molecular Sciences 23:1, pages 354.
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Xiaomeng Xing, Anjani Kumari, Jake Brown & John David Brook. (2021) Disrupting the Molecular Pathway in Myotonic Dystrophy. International Journal of Molecular Sciences 22:24, pages 13225.
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Estefanía Cerro-Herreros, Irene González-Martínez, Nerea Moreno, Jorge Espinosa-Espinosa, Juan M. Fernández-Costa, Anna Colom-Rodrigo, Sarah J. Overby, David Seoane-Miraz, Javier Poyatos-García, Juan J. Vilchez, Adolfo López de Munain, Miguel A. Varela, Matthew J. Wood, Manuel Pérez-Alonso, Beatriz Llamusí & Rubén Artero. (2021) Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy. Molecular Therapy - Nucleic Acids 26, pages 174-191.
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Valentina Grande, Denisa Hathazi, Emily O’Connor, Theo Marteau, Ulrike Schara-Schmidt, Andreas Hentschel, Genevieve Gourdon, Nikoletta Nikolenko, Hanns Lochmüller & Andreas Roos. (2021) Dysregulation of GSK3β-Target Proteins in Skin Fibroblasts of Myotonic Dystrophy Type 1 (DM1) Patients. Journal of Neuromuscular Diseases 8:4, pages 603-619.
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Marta Pascual-Gilabert, Arturo López-Castel & Ruben Artero. (2021) Myotonic dystrophy type 1 drug development: A pipeline toward the market. Drug Discovery Today 26:7, pages 1765-1772.
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Neil Datta & Partha S. Ghosh. (2020) Update on Muscular Dystrophies with Focus on Novel Treatments and Biomarkers. Current Neurology and Neuroscience Reports 20:6.
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Samantha LoRusso, Benjamin Weiner & W. David Arnold. (2018) Myotonic Dystrophies: Targeting Therapies for Multisystem Disease. Neurotherapeutics 15:4, pages 872-884.
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James D. Thomas, Ruan Oliveira, Łukasz J. Sznajder & Maurice S. Swanson. 2011. Comprehensive Physiology. Comprehensive Physiology 509 553 .

Valle Palomo, Daniel I. Perez, Carlos Roca, Cara Anderson, Natalia Rodríguez-Muela, Concepción Perez, Jose A. Morales-Garcia, Julio A. Reyes, Nuria E. Campillo, Ana M. Perez-Castillo, Lee L. Rubin, Lubov Timchenko, Carmen Gil & Ana Martinez. (2017) Subtly Modulating Glycogen Synthase Kinase 3 β: Allosteric Inhibitor Development and Their Potential for the Treatment of Chronic Diseases. Journal of Medicinal Chemistry 60:12, pages 4983-5001.
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Ellen L. van Agtmaal, Laurène M. André, Marieke Willemse, Sarah A. Cumming, Ingeborg D.G. van Kessel, Walther J.A.A. van den Broek, Geneviève Gourdon, Denis Furling, Vincent Mouly, Darren G. Monckton, Derick G. Wansink & Bé Wieringa. (2017) CRISPR/Cas9-Induced (CTG⋅CAG) n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing. Molecular Therapy 25:1, pages 24-43.
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Angela Sch?ller, Stephan Wenninger, Federica Montagnese & Benedikt Schoser. (2016) Multimodales Monitoring und Therapie der Myotonen Dystrophien. DNP - Der Neurologe und Psychiater 17:1, pages 42-50.
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Olof Joakim Pettersson, Lars Aagaard, Thomas Gryesten Jensen & Christian Kroun Damgaard. (2015) Molecular mechanisms in DM1 — a focus on foci. Nucleic Acids Research 43:4, pages 2433-2441.
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GSK3β is a new therapeutic target for myotonic dystrophy type 1

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GSK3β is a new therapeutic target for myotonic dystrophy type 1

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