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1 |
Material Type: Artículo
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Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal modelsSong, Yafeng ; Morales, Leon ; Malik, Alock S ; Mead, Andrew F ; Greer, Christopher D ; Mitchell, Marilyn A ; Petrov, Mihail T ; Su, Leonard T ; Choi, Margaret E ; Rosenblum, Shira T ; Lu, Xiangping ; VanBelzen, Daniel J ; Krishnankutty, Ranjith K ; Balzer, Frederick J ; Loro, Emanuele ; French, Robert ; Propert, Kathleen J ; Zhou, Shangzhen ; Kozyak, Benjamin W ; Nghiem, Peter P ; Khurana, Tejvir S ; Kornegay, Joe N ; Stedman, Hansell HNature medicine, 2019-10, Vol.25 (10), p.1505-1511 [Revista revisada por pares]United States: Nature Publishing GroupTexto completo disponible |
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2 |
Material Type: Artículo
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Tempol treatment shows phenotype improvement in mdx miceHermes, Túlio de Almeida ; Mâncio, Rafael Dias ; Macedo, Aline Barbosa ; Mizobuti, Daniela Sayuri ; Rocha, Guilherme Luiz da ; Cagnon, Valéria Helena Alves ; Minatel, Elaine Ervasti, James M.PloS one, 2019-04, Vol.14 (4), p.e0215590-e0215590 [Revista revisada por pares]United States: Public Library of ScienceTexto completo disponible |
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3 |
Material Type: Artículo
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Identification of muscle-specific microRNAs in serum of muscular dystrophy animal models: promising novel blood-based markers for muscular dystrophyMizuno, Hideya ; Nakamura, Akinori ; Aoki, Yoshitsugu ; Ito, Naoki ; Kishi, Soichiro ; Yamamoto, Kazuhiro ; Sekiguchi, Masayuki ; Takeda, Shin'ichi ; Hashido, Kazuo Pfeffer, SebastienPloS one, 2011-03, Vol.6 (3), p.e18388-e18388 [Revista revisada por pares]United States: Public Library of ScienceTexto completo disponible |
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4 |
Material Type: Artículo
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Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy PatientsDuan, DongshengHuman gene therapy, 2018-07, Vol.29 (7), p.733-736 [Revista revisada por pares]United States: Mary Ann Liebert, IncTexto completo disponible |
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5 |
Material Type: Artículo
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Broad phenotypic spectrum and genotype-phenotype correlations in GMPPB-related dystroglycanopathies: an Italian cross-sectional studyAstrea, Guja ; Romano, Alessandro ; Angelini, Corrado ; Antozzi, Carlo Giuseppe ; Barresi, Rita ; Battini, Roberta ; Battisti, Carla ; Bertini, Enrico ; Bruno, Claudio ; Cassandrini, Denise ; Fanin, Marina ; Fattori, Fabiana ; Fiorillo, Chiara ; Guerrini, Renzo ; Maggi, Lorenzo ; Mercuri, Eugenio ; Morani, Federica ; Mora, Marina ; Moro, Francesca ; Pezzini, Ilaria ; Picillo, Esther ; Pinelli, Michele ; Politano, Luisa ; Rubegni, Anna ; Sanseverino, Walter ; Savarese, Marco ; Striano, Pasquale ; Torella, Annalaura ; Trevisan, Carlo Pietro ; Trovato, Rosanna ; Zaraieva, Irina ; Muntoni, Francesco ; Nigro, Vincenzo ; D'Amico, Adele ; Santorelli, Filippo MOrphanet journal of rare diseases, 2018-09, Vol.13 (1), p.170-170, Article 170 [Revista revisada por pares]England: BioMed Central LtdTexto completo disponible |
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6 |
Material Type: Artículo
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MicroRNA-486-dependent modulation of DOCK3/PTEN/AKT signaling pathways improves muscular dystrophy-associated symptomsAlexander, Matthew S ; Casar, Juan Carlos ; Motohashi, Norio ; Vieira, Natássia M ; Eisenberg, Iris ; Marshall, Jamie L ; Gasperini, Molly J ; Lek, Angela ; Myers, Jennifer A ; Estrella, Elicia A ; Kang, Peter B ; Shapiro, Frederic ; Rahimov, Fedik ; Kawahara, Genri ; Widrick, Jeffrey J ; Kunkel, Louis MThe Journal of clinical investigation, 2014-06, Vol.124 (6), p.2651-2667 [Revista revisada por pares]United States: American Society for Clinical InvestigationTexto completo disponible |
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7 |
Material Type: Artículo
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Histone deacetylase inhibitors in the treatment of muscular dystrophies: epigenetic drugs for genetic diseasesConsalvi, Silvia ; Saccone, Valentina ; Giordani, Lorenzo ; Minetti, Giulia ; Mozzetta, Chiara ; Puri, Pier LorenzoMolecular medicine (Cambridge, Mass.), 2011-05, Vol.17 (5-6), p.457-465 [Revista revisada por pares]England: ScholarOneTexto completo disponible |
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8 |
Material Type: Artículo
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LARGE can functionally bypass α-dystroglycan glycosylation defects in distinct congenital muscular dystrophiesCampbell, Kevin P ; Barresi, Rita ; Michele, Daniel E ; Kanagawa, Motoi ; Harper, Hollie A ; Dovico, Sherri A ; Satz, Jakob S ; Moore, Steven A ; Zhang, Wenli ; Schachter, Harry ; Dumanski, Jan P ; Cohn, Ronald D ; Nishino, IchizoNature medicine, 2004-07, Vol.10 (7), p.696-703 [Revista revisada por pares]United StatesTexto completo disponible |
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9 |
Material Type: Artículo
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The beneficial effect of chronic muscular exercise on muscle fragility is increased by Prox1 gene transfer in dystrophic mdx muscleMonceau, Alexandra ; Delacroix, Clément ; Lemaitre, Mégane ; Revet, Gaelle ; Furling, Denis ; Agbulut, Onnik ; Klein, Arnaud ; Ferry, Arnaud Asakura, AtsushiPloS one, 2022-04, Vol.17 (4), p.e0254274-e0254274 [Revista revisada por pares]United States: Public Library of ScienceTexto completo disponible |
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10 |
Material Type: Artículo
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Dysfunctional polycomb transcriptional repression contributes to lamin A/C-dependent muscular dystrophyBianchi, Andrea ; Mozzetta, Chiara ; Pegoli, Gloria ; Lucini, Federica ; Valsoni, Sara ; Rosti, Valentina ; Petrini, Cristiano ; Cortesi, Alice ; Gregoretti, Francesco ; Antonelli, Laura ; Oliva, Gennaro ; De Bardi, Marco ; Rizzi, Roberto ; Bodega, Beatrice ; Pasini, Diego ; Ferrari, Francesco ; Bearzi, Claudia ; Lanzuolo, ChiaraThe Journal of clinical investigation, 2020-05, Vol.130 (5), p.2408-2421 [Revista revisada por pares]United States: American Society for Clinical InvestigationTexto completo disponible |