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Gene therapy for muscular dystrophy: current progress and future prospects

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Pages 849-866 | Published online: 15 Jun 2009

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Elisa Negroni, Denis Vallese, Jean-Thomas Vilquin, Gillian Butler-Browne, Vincent Mouly & Capucine Trollet. (2011) Current advances in cell therapy strategies for muscular dystrophies. Expert Opinion on Biological Therapy 11:2, pages 157-176.
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Ying Tang, James Cummins, Johnny Huard & Bing Wang. (2010) AAV-directed muscular dystrophy gene therapy. Expert Opinion on Biological Therapy 10:3, pages 395-408.
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Articles from other publishers (37)

Alexandra Monceau, Dylan Moutachi, Mégane Lemaitre, Luis Garcia, Capucine Trollet, Denis Furling, Arnaud Klein & Arnaud Ferry. (2022) Dystrophin Restoration after Adeno-Associated Virus U7–Mediated Dmd Exon Skipping Is Modulated by Muscular Exercise in the Severe D2-Mdx Duchenne Muscular Dystrophy Murine Model. The American Journal of Pathology 192:11, pages 1604-1618.
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Peng Zhang, Jian He, Fei Wang, Jing Gong, Lu Wang, Qian Wu, Wenjiong Li, Hongju Liu, Jing Wang, Kunshan Zhang, Mao Li, Xusheng Huang, Chuanqiang Pu, Ying Li, Fengjie Jiang, Fudi Wang, Junxia Min & Xiaoping Chen. (2019) Hemojuvelin is a novel suppressor for Duchenne muscular dystrophy and age‐related muscle wasting. Journal of Cachexia, Sarcopenia and Muscle 10:3, pages 557-573.
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Isabella Gazzoli & Annemieke Aartsma-Rus. 2018. Oligonucleotide-Based Drugs and Therapeutics. Oligonucleotide-Based Drugs and Therapeutics 445 489 .
Ngoc Lu-Nguyen, George Dickson & Alberto Malerba. 2018. Exon Skipping and Inclusion Therapies. Exon Skipping and Inclusion Therapies 343 354 .
Charles H. Vannoy, Haowen Zhou, Chunping Qiao, Xiao Xiao, Anne G. Bang & Qi L. Lu. (2017) Adeno-Associated Virus–Mediated Mini-Agrin Delivery Is Unable to Rescue Disease Phenotype in a Mouse Model of Limb Girdle Muscular Dystrophy Type 2I. The American Journal of Pathology 187:2, pages 431-440.
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A. Arvanitidis, K. Henriksen, M.A. Karsdal & A. Nedergaard. (2016) Neo-epitope Peptides as Biomarkers of Disease Progression for Muscular Dystrophies and Other Myopathies. Journal of Neuromuscular Diseases 3:3, pages 333-346.
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Lorenzo Ferri, Giuseppina Covello, Anna Caciotti, Renzo Guerrini, Michela Alessandra Denti & Amelia Morrone. (2016) Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations. Molecular Therapy - Nucleic Acids 5, pages e380.
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Fernanda Pinto-Mariz, Luciana Rodrigues Carvalho, Alexandra Prufer De Queiroz Campos Araujo, Wallace De Mello, Márcia Gonçalves Ribeiro, Maria Do Carmo Soares Alves Cunha, Pedro Hernan Cabello, Ingo Riederer, Elisa Negroni, Isabelle Desguerre, Mariana Veras, Erica Yada, Yves Allenbach, Olivier Benveniste, Thomas Voit, Vincent Mouly, Suse Dayse Silva-Barbosa, Gillian Butler-Browne & Wilson Savino. (2015) CD49d is a disease progression biomarker and a potential target for immunotherapy in Duchenne muscular dystrophy. Skeletal Muscle 5:1.
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Laurent Servais, Marie Montus, Caroline Le Guiner, Rabah Ben Yaou, Mélanie Annoussamy, Amélie Moraux, Jean-Yves Hogrel, Andreea M. Seferian, Karima Zehrouni, Anne-Gaëlle Le Moing, Teresa Gidaro, Catherine Vanhulle, Vincent Laugel, Nina Butoianu, Jean-Marie Cuisset, Pascal Sabouraud, Claude Cances, Andrea Klein, France Leturcq, Philippe Moullier & Thomas Voit. (2015) Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype. Journal of Neuromuscular Diseases 2:3, pages 269-279.
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Cheng-Cao Sun, Shu-Jun Li, Cui-Li Yang, Rui-Lin Xue, Yong-Yong Xi, Liang Wang, Qian-Long Zhao & De-Jia Li. (2015) Sulforaphane Attenuates Muscle Inflammation in Dystrophin-deficient mdx Mice via NF-E2-related Factor 2 (Nrf2)-mediated Inhibition of NF-κB Signaling Pathway. Journal of Biological Chemistry 290:29, pages 17784-17795.
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Elisa Negroni, Teresa Gidaro, Anne Bigot, Gillian S. Butler‐Browne, Vincent Mouly & Capucine Trollet. (2015) Invited review: Stem cells and muscle diseases: advances in cell therapy strategies. Neuropathology and Applied Neurobiology 41:3, pages 270-287.
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Denis Vallese, Elisa Negroni, Stéphanie Duguez, Arnaud Ferry, Capucine Trollet, Ahmed Aamiri, Christian AJ Vosshenrich, Ernst-Martin Füchtbauer, James P Di Santo, Libero Vitiello, Gillian Butler-Browne & Vincent Mouly. (2013) The Rag2–Il2rb–Dmd– Mouse: a Novel Dystrophic and Immunodeficient Model to Assess Innovating Therapeutic Strategies for Muscular Dystrophies. Molecular Therapy 21:10, pages 1950-1957.
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Flavie Sicard, Cedric Sapet, Nicolas Laurent, Elodie Bertosio, Melanie Bertuzzi & Olivier Zelphati. 2013. Minicircle and Miniplasmid DNA Vectors. Minicircle and Miniplasmid DNA Vectors 165 176 .
Kewal K. JainKewal K. Jain. 2013. Applications of Biotechnology in Neurology. Applications of Biotechnology in Neurology 383 476 .
Filippo Serra, Marco Quarta, Marta Canato, Luana Toniolo, Valeria De Arcangelis, Attilio Trotta, Lucia Spath, Lucia Monaco, Carlo Reggiani & Fabio Naro. (2012) Inflammation in muscular dystrophy and the beneficial effects of non-steroidal anti-inflammatory drugs. Muscle & Nerve 46:5, pages 773-784.
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Aur?lie Goyenvalle, Arran Babbs, Jordan Wright, Vivienne Wilkins, Dave Powell, Luis Garcia & Kay E. Davies. (2012) Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping. Human Molecular Genetics 21:11, pages 2559-2571.
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Denis Vallese, Erica Yada, Gillian Butler-Browne & Vincent Mouly. 2012. Muscle. Muscle 1053 1063 .
Linda J. Popplewell, Alberto Malerba & George Dickson. 2012. Exon Skipping. Exon Skipping 143 167 .
Kamel Mamchaoui, Capucine Trollet, Anne Bigot, Elisa Negroni, Soraya Chaouch, Annie Wolff, Prashanth K Kandalla, Solenne Marie, James Di Santo, Jean Lacau St Guily, Francesco Muntoni, Jihee Kim, Susanne Philippi, Simone Spuler, Nicolas Levy, Sergiu C Blumen, Thomas Voit, Woodring E Wright, Ahmed Aamiri, Gillian Butler-Browne & Vincent Mouly. (2011) Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders. Skeletal Muscle 1:1.
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Dwi U Kemaladewi, Willem MH Hoogaars, Sandra H van Heiningen, Samuel Terlouw, David JJ de Gorter, Johan T den Dunnen, Gert Jan B van Ommen, Annemieke Aartsma-Rus, Peter ten Dijke & Peter AC 't Hoen. (2011) Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy. BMC Medical Genomics 4:1.
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Gemma L. Mendel, Brian Paszkiet & Rafal P. Witek. 2011. Primary and Stem Cells. Primary and Stem Cells 163 197 .
Gerald Both, Ian Alexander, Sue Fletcher, Tamara J. Nicolson, John E.J. Rasko, Steve D. Wilton & Geoff Symonds. (2011) Gene therapy: therapeutic applications and relevance to pathology. Pathology 43:6, pages 642-656.
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Brian Bostick, Jin-Hong Shin, Yongping Yue & Dongsheng Duan. (2011) AAV-microdystrophin Therapy Improves Cardiac Performance in Aged Female mdx Mice. Molecular Therapy 19:10, pages 1826-1832.
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Alberto MalerbaLuisa BoldrinGeorge Dickson. (2011) Long-Term Systemic Administration of Unconjugated Morpholino Oligomers for Therapeutic Expression of Dystrophin by Exon Skipping in Skeletal Muscle: Implications for Cardiac Muscle Integrity. Nucleic Acid Therapeutics 21:4, pages 293-298.
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Manuel AFV Gonçalves, Josephine M Janssen, Quynh G Nguyen, Takis Athanasopoulos, Stephen D Hauschka, George Dickson & Antoine AF de Vries. (2011) Transcription Factor Rational Design Improves Directed Differentiation of Human Mesenchymal Stem Cells Into Skeletal Myocytes. Molecular Therapy 19:7, pages 1331-1341.
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Ken Smith. (2011) Feline muscular dystrophy: parallels between cats and people. Veterinary Record 168:19, pages 507-508.
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Ma?t? Carre-Pierrat, Aude Lafoux, Guillaume Tanniou, Lucie Chambonnier, Alexandra Divet, Francoise Fougerousse, Corinne Huchet-Cadiou & Laurent S?galat. (2011) Pre-clinical study of 21 approved drugs in the mdx mouse. Neuromuscular Disorders 21:5, pages 313-327.
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Nicolas Laurent, Cédric Sapet, Loic Le Gourrierec, Elodie Bertosio & Olivier Zelphati. (2011) Nucleic acid delivery using magnetic nanoparticles: the Magnetofection™ technology. Therapeutic Delivery 2:4, pages 471-482.
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M G Toscano, Z Romero, P Muñoz, M Cobo, K Benabdellah & F Martin. (2010) Physiological and tissue-specific vectors for treatment of inherited diseases. Gene Therapy 18:2, pages 117-127.
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Linda J. Popplewell, Ian R. Graham, Alberto Malerba & George Dickson. 2011. Muscle Gene Therapy. Muscle Gene Therapy 153 178 .
Cem Şöllü, Kaweh Pars, Tatjana I. Cornu, Stacey Thibodeau-Beganny, Morgan L. Maeder, J. Keith Joung, Regine Heilbronn & Toni Cathomen. (2010) Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion. Nucleic Acids Research 38:22, pages 8269-8276.
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Yuko Miyagoe-Suzuki & Shin'ichi Takeda. (2010) Gene therapy for muscle disease. Experimental Cell Research 316:18, pages 3087-3092.
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Eyayu Belay, Janka Mátrai, Abel Acosta-Sanchez, Ling Ma, Mattia Quattrocelli, Lajos Mátés, Pau Sancho-Bru, Martine Geraerts, Bing Yan, Joris Vermeesch, Melvin Yesid Rincón, Ermira Samara-Kuko, Zoltán Ivics, Catherine Verfaillie, Maurilio Sampaolesi, Zsuzsanna Izsvák, Thierry VandenDriessche & Marinee K. L. Chuah. (2010) Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation  . Stem Cells 28:10, pages 1760-1771.
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Tania Incitti, Fernanda G De Angelis, Valentina Cazzella, Olga Sthandier, Chiara Pinnar?Ivano Legnini & Irene Bozzoni. (2010) Exon Skipping and Duchenne Muscular Dystrophy Therapy: Selection of the Most Active U1 snRNA Antisense Able to Induce Dystrophin Exon 51 Skipping. Molecular Therapy 18:9, pages 1675-1682.
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Bing Wang. (2010) Gene Therapy and Muscles: The Use of Adeno-associated Virus—Where are We Today?. Operative Techniques in Orthopaedics 20:2, pages 136-143.
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Hideo SUGITA & Shin’ichi TAKEDA. (2010) Progress in muscular dystrophy research with special emphasis on gene therapy. Proceedings of the Japan Academy, Series B 86:7, pages 748-756.
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Julie Dumonceaux & Helge Amthor. (2009) Current advances in the development of therapies for neuromuscular disorders based on myostatin signalling, 3rd International Institute of Myology Workshop, Paris, September 12th, 2008. Neuromuscular Disorders 19:11, pages 797-799.
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