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Material Type: Artigo
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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 [Periódico revisado por pares]United States: Nature Publishing GroupTexto completo disponível |
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Material Type: Artigo
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LMNA-associated myopathies: The Italian experience in a large cohort of patientsMaggi, Lorenzo ; DʼAmico, Adele ; Pini, Antonella ; Sivo, Serena ; Pane, Marika ; Ricci, Giulia ; Vercelli, Liliana ; DʼAmbrosio, Paola ; Travaglini, Lorena ; Sala, Simone ; Brenna, Greta ; Kapetis, Dimos ; Scarlato, Marina ; Pegoraro, Elena ; Ferrari, Maurizio ; Toscano, Antonio ; Benedetti, Sara ; Bernasconi, Pia ; Colleoni, Lara ; Lattanzi, Giovanna ; Bertini, Enrico ; Mercuri, Eugenio ; Siciliano, Gabriele ; Rodolico, Carmelo ; Mongini, Tiziana ; Politano, Luisa ; Previtali, Stefano C ; Carboni, Nicola ; Mantegazza, Renato ; Morandi, LuciaNeurology, 2014-10, Vol.83 (18), p.1634-1644 [Periódico revisado por pares]Hagerstown, MD: American Academy of NeurologyTexto completo disponível |
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Material Type: Artigo
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Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophyDadgar, Sherry ; Wang, Zuyi ; Johnston, Helen ; Kesari, Akanchha ; Nagaraju, Kanneboyina ; Chen, Yi-Wen ; Hill, D Ashley ; Partridge, Terence A ; Giri, Mamta ; Freishtat, Robert J ; Nazarian, Javad ; Xuan, Jianhua ; Wang, Yue ; Hoffman, Eric PThe Journal of cell biology, 2014-10, Vol.207 (1), p.139-158 [Periódico revisado por pares]United States: Rockefeller University PressTexto completo disponível |
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Material Type: Artigo
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Evidence-based guideline summary: Diagnosis and treatment of limb-girdle and distal dystrophies: Report of the Guideline Development Subcommittee of the American Academy of Neurology and the Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic MedicineNarayanaswami, Pushpa ; Weiss, Michael ; Selcen, Duygu ; David, William ; Raynor, Elizabeth ; Carter, Gregory ; Wicklund, Matthew ; Barohn, Richard J ; Ensrud, Erik ; Griggs, Robert C ; Gronseth, Gary ; Amato, Anthony ANeurology, 2014-10, Vol.83 (16), p.1453-1463 [Periódico revisado por pares]United States: American Academy of NeurologyTexto completo disponível |
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Material Type: Artigo
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Importance and challenge of making an early diagnosis in LMNA-related muscular dystrophyMENEZES, M. P ; WADDELL, L. B ; HARBORD, M ; NORTH, K. N ; CLARKE, N. F ; EVESSON, F. J ; COOPER, S ; WEBSTER, R ; JONES, K ; MOWAT, D ; KIERNAN, M. C ; JOHNSTON, H. M ; CORBETT, ANeurology, 2012-04, Vol.78 (16), p.1258-1263 [Periódico revisado por pares]Hagerstown, MD: Lippincott Williams & WilkinsTexto completo disponível |
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Material Type: Artigo
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Natural disease history of the D2‐mdx mouse model for Duchenne muscular dystrophyPutten, Maaike ; Putker, Kayleigh ; Overzier, Maurice ; Adamzek, W. A. ; Pasteuning-Vuhman, Svetlana ; Plomp, Jaap J. ; Aartsma-Rus, AnnemiekeThe FASEB journal, 2019-07, Vol.33 (7), p.8110-8124 [Periódico revisado por pares]United States: Federation of American Societies for Experimental BiologyTexto completo disponível |
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Material Type: Artigo
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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 [Periódico revisado por pares]England: Oxford University PressTexto completo disponível |
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Material Type: Artigo
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Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophyEchigoya, Yusuke ; Nakamura, Akinori ; Nagata, Tetsuya ; Urasawa, Nobuyuki ; Lim, Kenji Rowel Q. ; Trieu, Nhu ; Panesar, Dharminder ; Kuraoka, Mutsuki ; Moulton, Hong M. ; Saito, Takashi ; Aoki, Yoshitsugu ; Iversen, Patrick ; Sazani, Peter ; Kole, Ryszard ; Maruyama, Rika ; Partridge, Terry ; Takeda, Shin’ichi ; Yokota, ToshifumiProceedings of the National Academy of Sciences - PNAS, 2017-04, Vol.114 (16), p.4213-4218 [Periódico revisado por pares]United States: National Academy of SciencesTexto completo disponível |
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Material Type: Artigo
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Degenerative and regenerative pathways underlying Duchenne muscular dystrophy revealed by single-nucleus RNA sequencingChemello, Francesco ; Wang, Zhaoning ; Li, Hui ; McAnally, John R. ; Liu, Ning ; Bassel-Duby, Rhonda ; Olson, Eric N.Proceedings of the National Academy of Sciences - PNAS, 2020-11, Vol.117 (47), p.29691-29701 [Periódico revisado por pares]United States: National Academy of SciencesTexto completo disponível |
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Material Type: Artigo
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TRIM32, but not its muscular dystrophy-associated mutant, positively regulates and is targeted to autophagic degradation by p62/SQSTM1Overå, Katrine Stange ; Garcia-Garcia, Juncal ; Bhujabal, Zambarlal ; Jain, Ashish ; Øvervatn, Aud ; Larsen, Kenneth Bowitz ; Deretic, Vojo ; Johansen, Terje ; Lamark, Trond ; Sjøttem, EvaJournal of cell science, 2019-12, Vol.132 (23) [Periódico revisado por pares]England: The Company of BiologistsTexto completo disponível |