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562 results for Dystrophin+-+metabolism

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ArticlesPeer ReviewedFull Text
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
Author / Creator:Amoasii, Leonela ; Hildyard, John C. W. ; Li, Hui ; Sanchez-Ortiz, Efrain ; Mireault, Alex ; Caballero, Daniel ; Harron, Rachel ; Stathopoulou, Thaleia-Rengina ; Massey, Claire ; Shelton, John M. ; Bassel-Duby, Rhonda ; Piercy, Richard J. ; Olson, Eric N.
Publisher:United States: The American Association for the Advancement of Science
Journal title:Science (American Association for the Advancement of Science), 2018-10, Vol.362 (6410), p.86-91
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Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice
Author / Creator:Kenjo, Eriya ; Hozumi, Hiroyuki ; Makita, Yukimasa ; Iwabuchi, Kumiko A. ; Fujimoto, Naoko ; Matsumoto, Satoru ; Kimura, Maya ; Amano, Yuichiro ; Ifuku, Masataka ; Naoe, Youichi ; Inukai, Naoto ; Hotta, Akitsu
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2021-12, Vol.12 (1), p.7101-13, Article 7101
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Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
Author / Creator:McGreevy, Joe W. ; Hakim, Chady H. ; McIntosh, Mark A. ; Duan, Dongsheng
Publisher:England: The Company of Biologists Ltd
Journal title:Disease models & mechanisms, 2015-03, Vol.8 (3), p.195-213
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Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division
Author / Creator:Dumont, Nicolas A ; Wang, Yu Xin ; von Maltzahn, Julia ; Pasut, Alessandra ; Bentzinger, C Florian ; Brun, Caroline E ; Rudnicki, Michael A
Publisher:London: Nature Publishing Group UK
Journal title:Nature medicine, 2015-12, Vol.21 (12), p.1455-1463
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Efficient precise in vivo base editing in adult dystrophic mice
Author / Creator:Xu, Li ; Zhang, Chen ; Li, Haiwen ; Wang, Peipei ; Gao, Yandi ; Mokadam, Nahush A. ; Ma, Jianjie ; Arnold, W. David ; Han, Renzhi
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2021-06, Vol.12 (1), p.3719-14, Article 3719
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Dystrophin Dp71 and the Neuropathophysiology of Duchenne Muscular Dystrophy
Author / Creator:Naidoo, Michael ; Anthony, Karen
Publisher:New York: Springer US
Journal title:Molecular neurobiology, 2020-03, Vol.57 (3), p.1748-1767
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Dystrophin–glycoprotein complex sequesters Yap to inhibit cardiomyocyte proliferation
Author / Creator:Morikawa, Yuka ; Heallen, Todd ; Leach, John ; Xiao, Yang ; Martin, James F.
Publisher:London: Nature Publishing Group UK
Journal title:Nature (London), 2017-07, Vol.547 (7662), p.227-231
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Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy
Author / Creator:Finkel, Richard S. ; Flanigan, Kevin M. ; Wong, Brenda ; Bönnemann, Carsten ; Sampson, Jacinda ; Sweeney, H. Lee ; Reha, Allen ; Northcutt, Valerie J. ; Elfring, Gary ; Barth, Jay ; Peltz, Stuart W.
Publisher:United States: Public Library of Science
Journal title:PloS one, 2013-12, Vol.8 (12), p.e81302-e81302
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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
Author / Creator:Cirak, Sebahattin, MD ; Arechavala-Gomeza, Virginia, PhD ; Guglieri, Michela, MD ; Feng, Lucy, PhD ; Torelli, Silvia, PhD ; Anthony, Karen, PhD ; Abbs, Stephen, PhD ; Garralda, Maria Elena, Prof ; Bourke, John, MD ; Wells, Dominic J, Prof ; Dickson, George, Prof ; Wood, Matthew JA, MD PhD ; Wilton, Steve D, Prof ; Straub, Volker, Prof ; Kole, Ryszard, Prof ; Shrewsbury, Stephen B, MD ; Sewry, Caroline, Prof ; Morgan, Jennifer E, PhD ; Bushby, Kate, Prof ; Muntoni, Francesco, Prof
Publisher:Kidlington: Elsevier Ltd
Journal title:The Lancet (British edition), 2011-08, Vol.378 (9791), p.595-605
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Progress toward Gene Therapy for Duchenne Muscular Dystrophy
Author / Creator:Chamberlain, Joel R. ; Chamberlain, Jeffrey S.
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2017-05, Vol.25 (5), p.1125-1131
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Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches
Author / Creator:Fairclough, Rebecca J. ; Wood, Matthew J. ; Davies, Kay E.
Publisher:London: Nature Publishing Group UK
Journal title:Nature reviews. Genetics, 2013-06, Vol.14 (6), p.373-378
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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
Author / Creator:Goemans, Nathalie M ; Tulinius, Mar ; van den Akker, Johanna T ; Burm, Brigitte E ; Ekhart, Peter F ; Heuvelmans, Niki ; Holling, Tjadine ; Janson, Anneke A ; Platenburg, Gerard J ; Sipkens, Jessica A ; Sitsen, J.M. Ad ; Aartsma-Rus, Annemieke ; van Ommen, Gert-Jan B ; Buyse, Gunnar ; Darin, Niklas ; Verschuuren, Jan J ; Campion, Giles V ; de Kimpe, Sjef J ; van Deutekom, Judith C
Publisher:Waltham, MA: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2011-04, Vol.364 (16), p.1513-1522
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Identification of Dp140 and α1-syntrophin as novel molecular interactors of the neuronal Ca V 2.1 channel
Author / Creator:Leyva-Leyva, Margarita ; Sandoval, Alejandro ; Morales-Lázaro, Sara Luz ; Corzo-López, Alejandra ; Felix, Ricardo ; González-Ramírez, Ricardo
Publisher:Germany
Journal title:Pflügers Archiv, 2023-05, Vol.475 (5), p.595
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ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs
Author / Creator:Hicks, Michael R. ; Hiserodt, Julia ; Paras, Katrina ; Fujiwara, Wakana ; Eskin, Ascia ; Jan, Majib ; Xi, Haibin ; Young, Courtney S. ; Evseenko, Denis ; Nelson, Stanley F. ; Spencer, Melissa J. ; Handel, Ben Van ; Pyle, April D.
Publisher:London: Nature Publishing Group UK
Journal title:Nature cell biology, 2018-01, Vol.20 (1), p.46-57
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Prednisolone rescues Duchenne muscular dystrophy phenotypes in human pluripotent stem cell–derived skeletal muscle in vitro
Author / Creator:Tanoury, Ziad Al ; Zimmerman, John F. ; Rao, Jyoti ; Sieiro, Daniel ; McNamara, Harold M. ; Cherrier, Thomas ; Rodríguez-delaRosa, Alejandra ; Hick-Colin, Aurore ; Bousson, Fanny ; Fugier-Schmucker, Charlotte ; Marchiano, Fabio ; Habermann, Bianca ; Chal, Jérome ; Nesmith, Alexander P. ; Gapon, Svetlana ; Wagner, Erica ; Gupta, Vandana A. ; Bassel-Duby, Rhonda ; Olson, Eric N. ; Cohen, Adam E. ; Parker, Kevin Kit ; Pourquié, Olivier
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2021-07, Vol.118 (28), p.1-12
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Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy
Author / Creator:Echigoya, Yusuke ; Nakamura, Akinori ; Nagata, Tetsuya ; Urasawa, Nobuyuki ; Lim, Kenji Rowel Q. ; Trieu, Nhu ; Panesar, Dharminder ; Kuraoka, Mutsuki ; Moulton, Hong M. ; Saito, Takashi ; Aoki, Yoshitsugu ; Iversen, Patrick ; Sazani, Peter ; Kole, Ryszard ; Maruyama, Rika ; Partridge, Terry ; Takeda, Shin’ichi ; Yokota, Toshifumi
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2017-04, Vol.114 (16), p.4213-4218
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Premature termination codons in the DMD gene cause reduced local mRNA synthesis
Author / Creator:García-Rodríguez, Raquel ; Hiller, Monika ; Jiménez-Gracia, Laura ; van der Pal, Zarah ; Balog, Judit ; Adamzek, Kevin ; Aartsma-Rus, Annemieke ; Spitali, Pietro
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2020-07, Vol.117 (28), p.16456-16464
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Cellular senescence-mediated exacerbation of Duchenne muscular dystrophy
Author / Creator:Sugihara, Hidetoshi ; Teramoto, Naomi ; Nakamura, Katsuyuki ; Shiga, Takanori ; Shirakawa, Taku ; Matsuo, Masafumi ; Ogasawara, Masashi ; Nishino, Ichizo ; Matsuwaki, Takashi ; Nishihara, Masugi ; Yamanouchi, Keitaro
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2020-10, Vol.10 (1), p.16385-16385, Article 16385
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DMD Mutations in 576 Dystrophinopathy Families: A Step Forward in Genotype-Phenotype Correlations
Author / Creator:Juan-Mateu, Jonas ; Gonzalez-Quereda, Lidia ; Rodriguez, Maria Jose ; Baena, Manel ; Verdura, Edgard ; Nascimento, Andres ; Ortez, Carlos ; Baiget, Montserrat ; Gallano, Pia
Publisher:United States: Public Library of Science
Journal title:PloS one, 2015-08, Vol.10 (8), p.e0135189-e0135189
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Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy
Author / Creator:Happi Mbakam, Cédric ; Rousseau, Joël ; Tremblay, Guillaume ; Yameogo, Pouiré ; Tremblay, Jacques P.
Publisher:Switzerland: MDPI AG
Journal title:International journal of molecular sciences, 2022-05, Vol.23 (11), p.6160
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Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

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Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice

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Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

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Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division

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Efficient precise in vivo base editing in adult dystrophic mice

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Dystrophin Dp71 and the Neuropathophysiology of Duchenne Muscular Dystrophy

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Dystrophin–glycoprotein complex sequesters Yap to inhibit cardiomyocyte proliferation

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Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

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Progress toward Gene Therapy for Duchenne Muscular Dystrophy

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Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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Identification of Dp140 and α1-syntrophin as novel molecular interactors of the neuronal Ca V 2.1 channel

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ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs

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Prednisolone rescues Duchenne muscular dystrophy phenotypes in human pluripotent stem cell–derived skeletal muscle in vitro

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Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy

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Premature termination codons in the DMD gene cause reduced local mRNA synthesis

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Cellular senescence-mediated exacerbation of Duchenne muscular dystrophy

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DMD Mutations in 576 Dystrophinopathy Families: A Step Forward in Genotype-Phenotype Correlations

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Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy

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