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Material Type: Artículo
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Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy miceAoki, Yoshitsugu ; Nagata, Tetsuya ; Yokota, Toshifumi ; Nakamura, Akinori ; Wood, Matthew J A ; Partridge, Terence ; Takeda, Shin'ichiHuman molecular genetics, 2013-12, Vol.22 (24), p.4914-4928 [Revista revisada por pares]England: Oxford University PressTexto completo disponible |
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Material Type: Artículo
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Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patientsHathout, Yetrib ; Marathi, Ramya L ; Rayavarapu, Sree ; Zhang, Aiping ; Brown, Kristy J ; Seol, Haeri ; Gordish-Dressman, Heather ; Cirak, Sebahattin ; Bello, Luca ; Nagaraju, Kanneboyina ; Partridge, Terry ; Hoffman, Eric P ; Takeda, Shin'ichi ; Mah, Jean K ; Henricson, Erik ; McDonald, CraigHuman molecular genetics, 2014-12, Vol.23 (24), p.6458-6469 [Revista revisada por pares]England: Oxford University PressTexto completo disponible |
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Material Type: Artículo
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How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouseGodfrey, Caroline ; Muses, Sofia ; McClorey, Graham ; Wells, Kim E ; Coursindel, Thibault ; Terry, Rebecca L ; Betts, Corinne ; Hammond, Suzan ; O'Donovan, Liz ; Hildyard, John ; El Andaloussi, Samir ; Gait, Michael J ; Wood, Matthew J ; Wells, Dominic JHuman molecular genetics, 2015-08, Vol.24 (15), p.4225-4237 [Revista revisada por pares]England: Oxford University PressTexto completo disponible |
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Material Type: Artículo
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AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expressionGicquel, Evelyne ; Maizonnier, Natacha ; Foltz, Steven J ; Martin, William J ; Bourg, Nathalie ; Svinartchouk, Fedor ; Charton, Karine ; Beedle, Aaron M ; Richard, IsabelleHuman molecular genetics, 2017-05, Vol.26 (10), p.1952-1965 [Revista revisada por pares]England: Oxford University Press (OUP)Texto completo disponible |
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Material Type: Artículo
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Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscleKlymiuk, Nikolai ; Blutke, Andreas ; Graf, Alexander ; Krause, Sabine ; Burkhardt, Katinka ; Wuensch, Annegret ; Krebs, Stefan ; Kessler, Barbara ; Zakhartchenko, Valeri ; Kurome, Mayuko ; Kemter, Elisabeth ; Nagashima, Hiroshi ; Schoser, Benedikt ; Herbach, Nadja ; Blum, Helmut ; Wanke, Rüdiger ; Aartsma-Rus, Annemieke ; Thirion, Christian ; Lochmüller, Hanns ; Walter, Maggie C ; Wolf, EckhardHuman molecular genetics, 2013-11, Vol.22 (21), p.4368-4382 [Revista revisada por pares]EnglandTexto completo disponible |
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Material Type: Artículo
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Shifts in macrophage phenotypes and macrophage competition for arginine metabolism affect the severity of muscle pathology in muscular dystrophyVillalta, S. Armando ; Nguyen, Hal X. ; Deng, Bo ; Gotoh, Tomomi ; Tidball, James G.Human molecular genetics, 2009-02, Vol.18 (3), p.482-496 [Revista revisada por pares]Oxford: Oxford University PressTexto completo disponible |
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Material Type: Artículo
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Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virusYue, Yongping ; Pan, Xiufang ; Hakim, Chady H ; Kodippili, Kasun ; Zhang, Keqing ; Shin, Jin-Hong ; Yang, Hsiao T ; McDonald, Thomas ; Duan, DongshengHuman molecular genetics, 2015-10, Vol.24 (20), p.5880-5890 [Revista revisada por pares]England: Oxford University PressTexto completo disponible |
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Material Type: Artículo
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Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophyZhang, Yadong ; Yue, Yongping ; Li, Liang ; Hakim, Chady H ; Zhang, Keqing ; Thomas, Gail D ; Duan, DongshengHuman molecular genetics, 2013-09, Vol.22 (18), p.3720-3729 [Revista revisada por pares]England: Oxford University PressTexto completo disponible |
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Material Type: Artículo
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Macrophages escape Klotho gene silencing in the mdx mouse model of Duchenne muscular dystrophy and promote muscle growth and increase satellite cell numbers through a Klotho-mediated pathwayWehling-Henricks, Michelle ; Welc, Steven S ; Samengo, Guiseppina ; Rinaldi, Chiara ; Lindsey, Catherine ; Wang, Ying ; Lee, Jeongyoon ; Kuro-O, Makoto ; Tidball, James GHuman molecular genetics, 2018-01, Vol.27 (1), p.14-29 [Revista revisada por pares]England: Oxford University PressTexto completo disponible |
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10 |
Material Type: Artículo
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Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restorationRoberts, 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 AHuman molecular genetics, 2015-12, Vol.24 (23), p.6756-6768 [Revista revisada por pares]England: Oxford University PressTexto completo disponible |