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The potential of utrophin modulators for the treatment of Duchenne muscular dystrophy

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Pages 179-192 | Received 15 Dec 2017, Accepted 06 Feb 2018, Published online: 15 Feb 2018

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Christine Péladeau & Bernard J. Jasmin. (2021) Targeting IRES-dependent translation as a novel approach for treating Duchenne muscular dystrophy. RNA Biology 18:9, pages 1238-1251.
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Simon Guiraud & Kay Davies. (2023) Utrophin correlates with disease severity in Duchenne muscular dystrophy. Med 4:4, pages 220-222.
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Maria Paz Ramirez, Sivaraman Rajaganapathy, Anthony R. Hagerty, Cailong Hua, Gloria C. Baxter, Joseph Vavra, Wendy R. Gordon, Joseph M. Muretta, Murti V. Salapaka & James M. Ervasti. (2023) Phosphorylation alters the mechanical stiffness of a model fragment of the dystrophin homologue utrophin. Journal of Biological Chemistry 299:2, pages 102847.
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Yu C. J. Chey, Jayshen Arudkumar, Annemieke Aartsma‐Rus, Fatwa Adikusuma & Paul Q. Thomas. (2022) CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies . WIREs Mechanisms of Disease 15:1.
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Maria Chatzopoulou, Daniel Conole, Enrico Emer, Jessica A. Rowley, Nicky J. Willis, Sarah E. Squire, Becky Gill, Steve Brough, Francis X. Wilson, Graham M. Wynne, Stephen G. Davies, Kay E. Davies & Angela J. Russell. (2022) Structure-activity relationships of 2-pyrimidinecarbohydrazides as utrophin modulators for the potential treatment of Duchenne muscular dystrophy. Bioorganic & Medicinal Chemistry 69, pages 116812.
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Theodora Markati, Maryam Oskoui, Michelle A Farrar, Tina Duong, Nathalie Goemans & Laurent Servais. (2022) Emerging therapies for Duchenne muscular dystrophy. The Lancet Neurology 21:9, pages 814-829.
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Tatiana V. Egorova, Ivan I. Galkin, Yulia V. Ivanova & Anna V. Polikarpova. 2022. Preclinical Animal Modeling in Medicine. Preclinical Animal Modeling in Medicine.
Angus Lindsay, Adam J. Trewin, Kate J. Sadler, Claire Laird, Paul A. Della Gatta & Aaron P. Russell. (2021) Sensitivity to behavioral stress impacts disease pathogenesis in dystrophin‐deficient mice. The FASEB Journal 35:12.
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Laura Salvadori, Sara Chiappalupi, Iva Arato, Francesca Mancuso, Mario Calvitti, Maria Cristina Marchetti, Francesca Riuzzi, Riccardo Calafiore, Giovanni Luca & Guglielmo Sorci. (2021) Sertoli Cells Improve Myogenic Differentiation, Reduce Fibrogenic Markers, and Induce Utrophin Expression in Human DMD Myoblasts. Biomolecules 11:10, pages 1504.
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Joanne Ban, Besa Beqaj & William D. Phillips. (2021) Vector‐mediated expression of muscle specific kinase restores specific force to muscles in the mdx mouse model of Duchenne muscular dystrophy . Experimental Physiology 106:8, pages 1794-1805.
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Aini Vuorinen, Isabel V.L. Wilkinson, Maria Chatzopoulou, Ben Edwards, Sarah E. Squire, Rebecca J. Fairclough, Noelia Araujo Bazan, Josh A. Milner, Daniel Conole, James R. Donald, Nandini Shah, Nicky J. Willis, R. Fernando Martínez, Francis X. Wilson, Graham M. Wynne, Stephen G. Davies, Kay E. Davies & Angela J. Russell. (2021) Discovery and mechanism of action studies of 4,6-diphenylpyrimidine-2-carbohydrazides as utrophin modulators for the treatment of Duchenne muscular dystrophy. European Journal of Medicinal Chemistry 220, pages 113431.
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Alicja Starosta & Patryk Konieczny. (2021) Therapeutic aspects of cell signaling and communication in Duchenne muscular dystrophy. Cellular and Molecular Life Sciences 78:11, pages 4867-4891.
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D’anna M. Nelson & James M. Ervasti. 2021. Encyclopedia of Biological Chemistry III. Encyclopedia of Biological Chemistry III 625 638 .
Maria Chatzopoulou, Enrico EmerCristina LecciJessica A. RowleyAnne-Sophie Casagrande, Lee MoirSarah E. SquireStephen G. Davies, Shawn HarrimanGraham M. WynneFrancis X. WilsonKay E. DaviesAngela J. Russell. (2020) Decreasing HepG2 Cytotoxicity by Lowering the Lipophilicity of Benzo[d]oxazolephosphinate Ester Utrophin Modulators. ACS Medicinal Chemistry Letters 11:12, pages 2421-2427.
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Cynthia Shu, Liubov Parfenova, Ekaterina Mokhonova, Judd R. Collado, Robert Damoiseaux, Jesus Campagna, Varghese John & Rachelle H. Crosbie. (2020) High-throughput screening identifies modulators of sarcospan that stabilize muscle cells and exhibit activity in the mouse model of Duchenne muscular dystrophy. Skeletal Muscle 10:1.
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Sean Y Ng & Vladimir Ljubicic. (2020) Recent insights into neuromuscular junction biology in Duchenne muscular dystrophy: Impacts, challenges, and opportunities. EBioMedicine 61, pages 103032.
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Kay E. Davies. (2020) The Long Journey from Diagnosis to Therapy. Annual Review of Genomics and Human Genetics 21:1, pages 1-13.
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Arran BabbsAdam BergMaria Chatzopoulou, Kay E. DaviesStephen G. Davies, Benjamin EdwardsDavid J. ElseyEnrico EmerSimon GuiraudShawn HarrimanCristina LecciLee MoirDavid PetersNeil RobinsonJessica A. RowleyAngela J. Russell, Sarah E. SquireJonathon M. TinsleyFrancis X. WilsonGraham M. Wynne. (2020) 2-Arylbenzo[ d ]oxazole Phosphinate Esters as Second-Generation Modulators of Utrophin for the Treatment of Duchenne Muscular Dystrophy . Journal of Medicinal Chemistry 63:14, pages 7880-7891.
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Arran BabbsMaria ChatzopoulouBen EdwardsSarah E. SquireIsabel V.L. WilkinsonGraham M. WynneAngela J. RussellKay E. Davies. (2020) From diagnosis to therapy in Duchenne muscular dystrophy. Biochemical Society Transactions 48:3, pages 813-821.
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Maria Chatzopoulou, Tim D. W. Claridge, Kay E. Davies, Stephen G. Davies, David J. Elsey, Enrico Emer, Ai M. Fletcher, Shawn Harriman, Neil Robinson, Jessica A. Rowley, Angela J. Russell, Jonathon M. Tinsley, Richard Weaver, Isabel V. L. Wilkinson, Nicky J. Willis, Francis X. Wilson & Graham M. Wynne. (2019) Isolation, Structural Identification, Synthesis, and Pharmacological Profiling of 1,2- trans -Dihydro-1,2-diol Metabolites of the Utrophin Modulator Ezutromid . Journal of Medicinal Chemistry 63:5, pages 2547-2556.
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Isabel V. L. Wilkinson, Kelly J. Perkins, Hannah Dugdale, Lee Moir, Aini Vuorinen, Maria Chatzopoulou, Sarah E. Squire, Sebastian Monecke, Alexander Lomow, Marcus Geese, Philip D. Charles, Peter Burch, Jonathan M. Tinsley, Graham M. Wynne, Stephen G. Davies, Francis X. Wilson, Fraydoon Rastinejad, Shabaz Mohammed, Kay E. Davies & Angela J. Russell. (2020) Chemical Proteomics and Phenotypic Profiling Identifies the Aryl Hydrocarbon Receptor as a Molecular Target of the Utrophin Modulator Ezutromid. Angewandte Chemie International Edition 59:6, pages 2420-2428.
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Isabel V. L. Wilkinson, Kelly J. Perkins, Hannah Dugdale, Lee Moir, Aini Vuorinen, Maria Chatzopoulou, Sarah E. Squire, Sebastian Monecke, Alexander Lomow, Marcus Geese, Philip D. Charles, Peter Burch, Jonathan M. Tinsley, Graham M. Wynne, Stephen G. Davies, Francis X. Wilson, Fraydoon Rastinejad, Shabaz Mohammed, Kay E. Davies & Angela J. Russell. (2020) Chemical Proteomics and Phenotypic Profiling Identifies the Aryl Hydrocarbon Receptor as a Molecular Target of the Utrophin Modulator Ezutromid. Angewandte Chemie 132:6, pages 2441-2449.
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Isabel V.L. Wilkinson, Jessica K. Reynolds, Sébastien R.G. Galan, Aini Vuorinen, Adam J. Sills, Elisabete Pires, Graham M. Wynne, Francis X. Wilson & Angela J. Russell. (2020) Characterisation of utrophin modulator SMT C1100 as a non-competitive inhibitor of firefly luciferase. Bioorganic Chemistry 94, pages 103395.
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Courtney S. Young, April D. Pyle & Melissa J. Spencer. (2019) CRISPR for Neuromuscular Disorders: Gene Editing and Beyond. Physiology 34:5, pages 341-353.
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S. Trajanovska, J. Ban, J. Huang, P. Gregorevic, M. Morsch, D. G. Allen & W. D. Phillips. (2019) Muscle specific kinase protects dystrophic mdx mouse muscles from eccentric contraction‐induced loss of force‐producing capacity . The Journal of Physiology 597:18, pages 4831-4850.
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Simon Guiraud, Benjamin Edwards, Arran Babbs, Sarah E Squire, Adam Berg, Lee Moir, Matthew J Wood & Kay E Davies. (2019) The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy. Human Molecular Genetics 28:13, pages 2189-2200.
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Sara Chiappalupi, Laura Salvadori, Giovanni Luca, Francesca Riuzzi, Riccardo Calafiore, Rosario Donato & Guglielmo Sorci. (2019) Do porcine Sertoli cells represent an opportunity for Duchenne muscular dystrophy?. Cell Proliferation 52:3.
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Simon Guiraud & KayE Davies. (2019) Regenerative biomarkers for Duchenne muscular dystrophy. Neural Regeneration Research 14:8, pages 1317.
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Simon Guiraud, Benjamin Edwards, Sarah E Squire, Lee Moir, Adam Berg, Arran Babbs, Nesrine Ramadan, Matthew J Wood & Kay E Davies. (2018) Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD. Human Molecular Genetics.
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