Výsledky vyhledávání - Virginia Arechavala‐Gomeza

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  1. 1
    Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges

    Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges Autor Virginia Arechavala‐Gomeza, Karen Anthony, Jennifer E. Morgan, Francesco Muntoni

    Vydáno 2012
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  2. 2
    Immunohistological intensity measurements as a tool to assess sarcolemma‐associated protein expression

    Immunohistological intensity measurements as a tool to assess sarcolemma‐associated protein expression Autor Virginia Arechavala‐Gomeza, Maria Kinali, L. Feng, S. Brown, Caroline A. Sewry, Jennifer E. Morgan, Francesco Muntoni

    Vydáno 2009
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  3. 3
    An Overview of Alternative Splicing Defects Implicated in Myotonic Dystrophy Type I

    An Overview of Alternative Splicing Defects Implicated in Myotonic Dystrophy Type I Autor Andrea López‐Martínez, Patricia Soblechero‐Martín, Laura de la Puente Ovejero, Gisela Nogales‐Gadea, Virginia Arechavala‐Gomeza

    Vydáno 2020
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  4. 4
    Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice

    Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice Autor Alberto Malerba, Paul Sharp, Ian R. Graham, Virginia Arechavala‐Gomeza, Keith Foster, Francesco Muntoni, Dominic J. Wells, George Dickson

    Vydáno 2010
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  5. 5
    Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

    Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy Autor Sebahattin Çirak, Lucy Feng, Karen Anthony, Virginia Arechavala‐Gomeza, Silvia Torelli, Caroline A. Sewry, Jennifer E. Morgan, Francesco Muntoni

    Vydáno 2011
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  6. 6
    Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides

    Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides Autor Aurélie Goyenvalle, C. Jimenez‐Mallebrera, Willeke M. C. van Roon‐Mom, Sabine Sewing, Arthur Μ. Krieg, Virginia Arechavala‐Gomeza, Patrik Andersson

    Vydáno 2022
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  7. 7
    A Duchenne Muscular Dystrophy Gene Hot Spot Mutation in Dystrophin-Deficient Cavalier King Charles Spaniels Is Amenable to Exon 51 Skipping

    A Duchenne Muscular Dystrophy Gene Hot Spot Mutation in Dystrophin-Deficient Cavalier King Charles Spaniels Is Amenable to Exon 51 Skipping Autor Gemma Walmsley, Virginia Arechavala‐Gomeza, Marta Fernández-Fuente, Margaret Burke, Nicole Nagel, Angela Holder, Rachael Stanley, Kate Chandler, Stanley L. Marks, Francesco Muntoni, G. Diane Shelton, Richard J. Piercy

    Vydáno 2010
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  8. 8
    Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials

    Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials Autor Virginia Arechavala‐Gomeza, Maria Kinali, Lucy Feng, Michela Guglieri, G. Edge, Marion Main, David M. Hunt, Jan Lehovsky, Volker Straub, Kate Bushby, Caroline A. Sewry, Jennifer E. Morgan, Francesco Muntoni

    Vydáno 2010
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  9. 9
    Comparative Analysis of Antisense Oligonucleotide Sequences for Targeted Skipping of Exon 51 During Dystrophin Pre-mRNA Splicing in Human Muscle

    Comparative Analysis of Antisense Oligonucleotide Sequences for Targeted Skipping of Exon 51 During Dystrophin Pre-mRNA Splicing in Human Muscle Autor Virginia Arechavala‐Gomeza, Ian R. Graham, Linda Popplewell, A.M. Adams, Annemieke Aartsma‐Rus, Maria Kinali, Jennifer E. Morgan, Judith C. van Deutekom, Steve D. Wilton, George Dickson, Francesco Muntoni

    Vydáno 2007
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  10. 10
    Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology

    Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology Autor Annemieke Aartsma‐Rus, Jennifer E. Morgan, Pallavi Lonkar, Hendrik Neubert, Jane Owens, Michael Binks, Marisol Montolio, Rahul Phadke, Nicole A. Datson, J. van Deutekom, Glenn E. Morris, V. Ashutosh Rao, Eric P. Hoffman, Francesco Muntoni, Virginia Arechavala‐Gomeza

    Vydáno 2019
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  11. 11
    Delivery is key: lessons learnt from developing splice‐switching antisense therapies

    Delivery is key: lessons learnt from developing splice‐switching antisense therapies Autor Caroline Godfrey, Lourdes R. Desviat, Bård Smedsrød, France Piétri‐Rouxel, Michela A. Denti, Petra Disterer, Stéphanie Lorain, Gisela Nogales‐Gadea, Valentina Sardone, Anwar Rayan, Samir EL Andaloussi, Taavi Lehto, Bernard Khoo, Camilla Brolin, Willeke M. C. van Roon‐Mom, Aurélie Goyenvalle, Annemieke Aartsma‐Rus, Virginia Arechavala‐Gomeza

    Vydáno 2017
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  12. 12
    Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

    Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials Autor Karen Anthony, Sebahattin Çirak, Silvia Torelli, Giorgio Tasca, Lucy Feng, Virginia Arechavala‐Gomeza, Annarita Armaroli, Michela Guglieri, C.S.M. Straathof, Jan J.G.M. Verschuuren, Annemieke Aartsma‐Rus, P. Helderman-van den Enden, K. Bushby, Volker Straub, Caroline A. Sewry, Alessandra Ferlini, Enzo Ricci, Jennifer E. Morgan, Francesco Muntoni

    Vydáno 2011
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  13. 13
    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

    Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-... Autor Sebahattin Çirak, Virginia Arechavala‐Gomeza, Michela Guglieri, Lucy Feng, Silvia Torelli, Karen Anthony, Stephen Abbs, Maria Elena Garralda, John Bourke, Dominic J. Wells, George Dickson, Matthew J. A. Wood, Steve D. Wilton, Volker Straub, Ryszard Kole, Stephen B. Shrewsbury, Caroline A. Sewry, Jennifer E. Morgan, Kate Bushby, Francesco Muntoni

    Vydáno 2011
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  14. 14
    Dystrophin quantification

    Dystrophin quantification Autor Karen Anthony, Virginia Arechavala‐Gomeza, Laura E. Taylor, Adeline Vulin, Yuuki Kaminoh, Silvia Torelli, Lucy Feng, Narinder Janghra, Gisèle Bonne, Maud Beuvin, Rita Barresi, Matt Henderson, Steven H. Laval, Afrodite Lourbakos, G. Campion, Volker Straub, Thomas Voït, Caroline A. Sewry, Jennifer E. Morgan, Kevin M. Flanigan, Francesco Muntoni

    Vydáno 2014
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  15. 15
    Muscle histology vs MRI in Duchenne muscular dystrophy

    Muscle histology vs MRI in Duchenne muscular dystrophy Autor Maria Kinali, Virginia Arechavala‐Gomeza, Sebahattin Çirak, Alan Glover, Michela Guglieri, L. Feng, Kieren G. Hollingsworth, David M. Hunt, Heinz Jungbluth, Helen Roper, Rosaline C. M. Quinlivan, Jayaprakash Gosalakkal, Sandeep Jayawant, Amélie Nadeau, L. Hughes-Carre, A. Manzur, Eugenio Mercuri, Jennifer E. Morgan, Volker Straub, K. Bushby, C. Sewry, Mary Rutherford, Francesco Muntoni

    Vydáno 2011
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  16. 16
    Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy

    Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy Autor Volker Straub, Pavel Balabanov, Kate Bushby, Monica Ensini, Nathalie Goemans, Annamaria De Luca, Alejandra Pereda, Robert Hemmings, G. Campion, Edward M. Kaye, Virginia Arechavala‐Gomeza, Aurélie Goyenvalle, Erik H. Niks, O. Veldhuizen, Pat Furlong, Violeta Stoyanova‐Beninska, Matthew J. A. Wood, Johnson Alex, Eugenio Mercuri, Francesco Muntoni, Bruno Sepodes, Manuel Haas, Elizabeth Vroom, Annemieke Aartsma‐Rus

    Vydáno 2016
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  17. 17
    Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

    Delivery of oligonucleotide‐based therapeutics: challenges and opportunities Autor Suzan M. Hammond, Annemieke Aartsma‐Rus, Sandra Alves, Sven Even Borgos, Ronald A.M. Buijsen, Rob W.J. Collin, Giuseppina Covello, Michela A. Denti, Lourdes R. Desviat, Lucía Echevarría, Camilla Foged, Gisela Găină, Alejandro Garanto, Aurélie Goyenvalle, Magdalena Guzowska, Irina Kholodnyuk, D. I. Jones, Sabine Krause, Taavi Lehto, Marisol Montolio, Willeke M. C. van Roon‐Mom, Virginia Arechavala‐Gomeza

    Vydáno 2021
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  18. 18
    Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

    Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-conce... Autor Maria Kinali, Virginia Arechavala‐Gomeza, Lucy Feng, Sebahattin Çirak, David M. Hunt, C. Adkin, Michela Guglieri, Emma Ashton, Stephen Abbs, Petros Nihoyannopoulos, Maria Elena Garralda, Mary Rutherford, Caroline McCulley, Linda Popplewell, Ian R. Graham, George Dickson, Matthew J. A. Wood, Dominic J. Wells, Steve D. Wilton, Ryszard Kole, Volker Straub, Kate Bushby, C. Sewry, Jennifer E. Morgan, Francesco Muntoni

    Vydáno 2009
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