Search Results - "Duchenne muscular dystrophy"

CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy by Pei Zhu, Furen Wu, Jeffrey Mosenson, Hongmei Zhang, Tong-Chuan He, Wen-Shu Wu

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Early myocardial damage assessment in dystrophinopathies using 99Tcm-MIBI gated myocardial perfusion imaging by Zhang L, Liu Z, Hu KY, Tian QB, Wei LG, Zhao Z, Shen HR, Hu J

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Assessment of Weighted Gene Co-Expression Network Analysis to Explore Key Pathways and Novel Biomarkers in Muscular Dystrophy by Xu X, Hao Y, Wu J, Zhao J, Xiong S

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Orofacial Manifestations Associated with Muscular Dystrophies: A Review by Petros Papaefthymiou, Kyriaki Kekou, Fulya Özdemir

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Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies by Ilaria Andreana, Mathieu Repellin, Flavia Carton, David Kryza, Stéphanie Briançon, Bénédicte Chazaud, Rémi Mounier, Silvia Arpicco, Manuela Malatesta, Barbara Stella, Giovanna Lollo

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Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene by Cedric Happi Mbakam, Joel Rousseau, Yaoyao Lu, Anne Bigot, Kamel Mamchaoui, Vincent Mouly, Jacques P. Tremblay

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Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology by Man Amanat, Christina L. Nemeth, Amena Smith Fine, Doris G. Leung, Ali Fatemi

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Proteomic profiling of the interface between the stomach wall and the pancreas in dystrophinopathy by Paul Dowling, Stephen Gargan, Margit Zweyer, Hemmen Sabir, Michael Henry, Paula Meleady, Dieter Swandulla, Kay Ohlendieck

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Satellite Cells: Regenerative Mechanisms and Applicability in Muscular Dystrophy by Gustavo Torres de Souza, Rafaella de Souza Salomão Zanette, Danielle Luciana Aurora Soares do Amaral, Francisco Carlos da Guia, Claudinéia Pereira Maranduba, Camila Maurmann de Souza, Ernesto da Silveira Goulart Guimarães, João Vitor Paes Rettore, Natana Chaves Rabelo, Antônio Márcio Resende do Carmo, Fernando de Sá Silva, Carlos Magno da Costa Maranduba

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Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models by Christa L Tanganyika-de Winter, Hans Heemskerk, Tatyana G Karnaoukh, Maaike van Putten, Sjef J de Kimpe, Judith van Deutekom, Annemieke Aartsma-Rus

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Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor by Dwi U Kemaladewi, Svitlana Pasteuning, Joke W van der Meulen, Sandra H van Heiningen, Gert-Jan van Ommen, Peter ten Dijke, Annemieke Aartsma-Rus, Peter AC 't Hoen, Willem M Hoogaars

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Oxidised Albumin Levels in Plasma and Skeletal Muscle as Biomarkers of Disease Progression and Treatment Efficacy in Dystrophic <i>mdx</i> Mice by Jessica R. Terrill, Angelo Patrick R. Bautista, Irene Tsioutsias, Miranda D. Grounds, Peter G. Arthur

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Beneficial Role of Exercise in the Modulation of <em>mdx </em>Muscle Plastic Remodeling and Oxidative Stress by Monica Frinchi, Giuseppe Morici, Giuseppa Mudó, Maria R. Bonsignore, Valentina Di Liberto

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Multi-Laboratory Evaluation of Prototype Dried Blood Spot Quality Control Materials for Creatine Kinase-MM Newborn Screening Assays by Paul Dantonio, Norma P. Tavakoli, Brooke Migliore, Elizabeth McCown, Timothy Lim, Sunju Park, Michele Caggana, Katerina S. Kucera, Han Phan, Natalie Street, Konstantinos Petritis, Robert F. Vogt

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Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein by Dunhui Li, Abbie M. Adams, Russell D. Johnsen, Susan Fletcher, Steve D. Wilton

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Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice by Flavien Bizot, Remko Goossens, Thomas Tensorer, Sergei Dmitriev, Luis Garcia, Annemieke Aartsma-Rus, Pietro Spitali, Aurélie Goyenvalle

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Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method by Jean-Paul, Dominique L Ouellet, Xavier Barbeau, Joël Rousseau, Pierre Chapdelaine, Patrick Lagüe, Jacques P Tremblay

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MicroRNA-206 Downregulation Improves Therapeutic Gene Expression and Motor Function in mdx Mice by Karen Bulaklak, Bin Xiao, Chunping Qiao, Jianbin Li, Tejash Patel, Quan Jin, Juan Li, Xiao Xiao

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Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice by Limin Cao, Gang Han, Caorui Lin, Ben Gu, Xianjun Gao, Hong M Moulton, Yiqi Seow, HaiFang Yin

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Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice by Ngoc Lu-Nguyen, Alberto Malerba, Linda Popplewell, Fred Schnell, Gunnar Hanson, George Dickson

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