Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics von McClorey, Graham, Banerjee, Subhashis

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An overview of the clinical application of antisense oligonucleotides for RNA-targeting therapies von McClorey, Graham, Wood, Matthew J

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Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment von Gait, Michael J., Arzumanov, Andrey A., McClorey, Graham, Godfrey, Caroline, Betts, Corinne, Hammond, Suzan, Wood, Matthew J.A.

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An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides von Baker, Ysobel R., Thorpe, Cameron, Chen, Jinfeng, Poller, Laura M., Cox, Lina, Kumar, Pawan, Lim, Wooi F., Lie, Lillian, McClorey, Graham, Epple, Sven, Singleton, Daniel, McDonough, Michael A., Hardwick, Jack S., Christensen, Kirsten E., Wood, Matthew J. A., Hall, James P., El-Sagheer, Afaf H., Brown, Tom

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Modulating the expression of disease genes with RNA-based therapy von Wood, Matthew, Yin, Haifang, McClorey, Graham

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Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies von Rouillon, Jérémy, Poupiot, Jérôme, Zocevic, Aleksandar, Amor, Fatima, Léger, Thibaut, Garcia, Camille, Camadro, Jean-Michel, Wong, Brenda, Pinilla, Robin, Cosette, Jérémie, Coenen-Stass, Anna M L, Mcclorey, Graham, Roberts, Thomas C, Wood, Matthew J A, Servais, Laurent, Udd, Bjarne, Voit, Thomas, Richard, Isabelle, Svinartchouk, Fedor

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Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration von Roberts, Thomas C, Johansson, Henrik J, McClorey, Graham, Godfrey, Caroline, Blomberg, K Emelie M, Coursindel, Thibault, Gait, Michael J, Smith, C I Edvard, Lehtiö, Janne, El Andaloussi, Samir, Wood, Matthew J A

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Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles von Peccate, Cécile, Mollard, Amédée, Le Hir, Maëva, Julien, Laura, McClorey, Graham, Jarmin, Susan, Le Heron, Anita, Dickson, George, Benkhelifa-Ziyyat, Sofia, Piétri-Rouxel, France, Wood, Matthew J, Voit, Thomas, Lorain, Stéphanie

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Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy von Blain, Alison M, Greally, Elizabeth, McClorey, Graham, Manzano, Raquel, Betts, Corinne A, Godfrey, Caroline, O'Donovan, Liz, Coursindel, Thibault, Gait, Mike J, Wood, Matthew J, MacGowan, Guy A, Straub, Volker W

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Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics von Coenen-Stass, Anna M. L., McClorey, Graham, Manzano, Raquel, Betts, Corinne A., Blain, Alison, Saleh, Amer F., Gait, Michael J., Lochmüller, Hanns, Wood, Matthew J. A., Roberts, Thomas C.

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Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice von Betts, Corinne A., Saleh, Amer F., Carr, Carolyn A., Hammond, Suzan M., Coenen-Stass, Anna M. L., Godfrey, Caroline, McClorey, Graham, Varela, Miguel A., Roberts, Thomas C., Clarke, Kieran, Gait, Michael J., Wood, Matthew J. A.

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Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides von Tone, Yuichiro, Mamchaoui, Kamel, Tsoumpra, Maria K, Hashimoto, Yasumasa, Terada, Reiko, Maruyama, Rika, Gait, Michael J, Arzumanov, Andrey A, McClorey, Graham, Imamura, Michihiro, Takeda, Shin'ichi, Yokota, Toshifumi, Wood, Matthew J A, Mouly, Vincent, Aoki, Yoshitsugu

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Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology von Westering, Tirsa L.E., Lomonosova, Yulia, Coenen‐Stass, Anna M.L., Betts, Corinne A., Bhomra, Amarjit, Hulsker, Margriet, Clark, Lucy E., McClorey, Graham, Aartsma‐Rus, Annemieke, Putten, Maaike, Wood, Matthew J.A., Roberts, Thomas C.

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Expression Analysis in Multiple Muscle Groups and Serum Reveals Complexity in the MicroRNA Transcriptome of the mdx Mouse with Implications for Therapy von Roberts, Thomas C, Blomberg, K Emelie M, McClorey, Graham, Andaloussi, Samir EL, Godfrey, Caroline, Betts, Corinne, Coursindel, Thibault, Gait, Michael J, Edvard Smith, CI, Wood, Matthew JA

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Splice-Switching Therapy for Spinal Muscular Atrophy von Meijboom, Katharina E, Wood, Matthew J A, McClorey, Graham

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Fine Tuning of Phosphorothioate Inclusion in 2′-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation von Aoki, Yoshitsugu, Rocha, Cristina S. J., Lehto, Taavi, Miyatake, Shouta, Johansson, Henrik, Hashimoto, Yasumasa, Nordin, Joel Z., Mager, Imre, Aoki, Misako, Graham, McClorey, Sathyaprakash, Chaitra, Roberts, Thomas C., Wood, Matthew J. A., Behlke, Mark A., Andaloussi, Samir El

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Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnover von Roberts, Thomas C., Godfrey, Caroline, McClorey, Graham, Vader, Pieter, Briggs, Deborah, Gardiner, Chris, Aoki, Yoshitsugu, Sargent, Ian, Morgan, Jennifer E., Wood, Matthew J.A.

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Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy von Betts, Corinne A., Saleh, Amer F., Carr, Carolyn A., Muses, Sofia, Wells, Kim E., Hammond, Suzan M., Godfrey, Caroline, McClorey, Graham, Woffindale, Caroline, Clarke, Kieran, Wells, Dominic J., Gait, Michael J., Wood, Matthew J. A.

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Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of D... von Malerba, Alberto, Kang, Jagjeet K, McClorey, Graham, Saleh, Amer F, Popplewell, Linda, Gait, Michael J, Wood, Matthew JA, Dickson, George

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Small RNA-Mediated Epigenetic Myostatin Silencing von Roberts, Thomas C, Andaloussi, Samir EL, Morris, Kevin V, McClorey, Graham, Wood, Matthew JA

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