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Material Type: Artigo
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Delivery of oligonucleotide‐based therapeutics: challenges and opportunitiesHammond, Suzan M ; Aartsma‐Rus, Annemieke ; Alves, Sandra ; Borgos, Sven E ; Buijsen, Ronald A M ; Collin, Rob W J ; Covello, Giuseppina ; Denti, Michela A ; Desviat, Lourdes R ; Echevarría, Lucía ; Foged, Camilla ; Gaina, Gisela ; Garanto, Alejandro ; Goyenvalle, Aurelie T ; Guzowska, Magdalena ; Holodnuka, Irina ; Jones, David R ; Krause, Sabine ; Lehto, Taavi ; Montolio, Marisol ; Van Roon‐Mom, Willeke ; Arechavala‐Gomeza, VirginiaEMBO molecular medicine, 2021-04, Vol.13 (4), p.e13243-n/a [Periódico revisado por pares]England: Wiley Subscription Services, IncTexto completo disponível |
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Material Type: Artigo
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Delivery is key: lessons learnt from developing splice‐switching antisense therapiesGodfrey, Caroline ; Desviat, Lourdes R ; Smedsrød, Bård ; Piétri‐Rouxel, France ; Denti, Michela A ; Disterer, Petra ; Lorain, Stéphanie ; Nogales‐Gadea, Gisela ; Sardone, Valentina ; Anwar, Rayan ; EL Andaloussi, Samir ; Lehto, Taavi ; Khoo, Bernard ; Brolin, Camilla ; Roon‐Mom, Willeke MC ; Goyenvalle, Aurélie ; Aartsma‐Rus, Annemieke ; Arechavala‐Gomeza, VirginiaEMBO molecular medicine, 2017-05, Vol.9 (5), p.545-557 [Periódico revisado por pares]England: EMBO PressTexto completo disponível |
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3 |
Material Type: Artigo
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Cognitive flexibility deficits in a mouse model for the absence of full‐length dystrophinRemmelink, E. ; Aartsma‐Rus, A. ; Smit, A. B. ; Verhage, M. ; Loos, M. ; van Putten, M.Genes, brain and behavior, 2016-07, Vol.15 (6), p.558-567 [Periódico revisado por pares]Oxford, UK: Blackwell Publishing LtdTexto completo disponível |
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4 |
Material Type: Artigo
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Percutaneous ultrasound-guided vacuum-assisted excision of benign breast lesions: A learning curve to assess outcomesSalazar, Juan Pablo ; Miranda, Ignacio ; de Torres, Juan ; Rus, María N ; Espinosa-Bravo, Martin ; Esgueva, Antonio ; Salvador, Rafael ; Rubio, Isabel TBritish journal of radiology, 2019-02, Vol.92 (1094), p.20180626-20180626 [Periódico revisado por pares]England: The British Institute of RadiologyTexto completo disponível |
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Material Type: Artigo
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Antisense-mediated modulation of splicing: Therapeutic implications for Duchenne muscular dystrophyAartsma-Rus, AnnemiekeRNA biology, 2010-07, Vol.7 (4), p.453-461 [Periódico revisado por pares]United States: Taylor & FrancisTexto completo disponível |
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6 |
Material Type: Artigo
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Ruscogenin attenuates particulate matter-induced acute lung injury in mice via protecting pulmonary endothelial barrier and inhibiting TLR4 signaling pathwayWang, Yu-Wei ; Wu, Yun-Hao ; Zhang, Jia-Zhi ; Tang, Jia-Hui ; Fan, Rui-Ping ; Li, Fang ; Yu, Bo-Yang ; Kou, Jun-Ping ; Zhang, Yuan-YuanActa pharmacologica Sinica, 2021-05, Vol.42 (5), p.726-734 [Periódico revisado por pares]United States: Nature Publishing GroupTexto completo disponível |
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Material Type: Artigo
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Affinity proteomics within rare diseases: a BIO‐NMD study for blood biomarkers of muscular dystrophiesAyoglu, Burcu ; Chaouch, Amina ; Lochmüller, Hanns ; Politano, Luisa ; Bertini, Enrico ; Spitali, Pietro ; Hiller, Monika ; Niks, Eric H ; Gualandi, Francesca ; Pontén, Fredrik ; Bushby, Kate ; Aartsma‐Rus, Annemieke ; Schwartz, Elena ; Le Priol, Yannick ; Straub, Volker ; Uhlén, Mathias ; Cirak, Sebahattin ; ‘t Hoen, Peter A C ; Muntoni, Francesco ; Ferlini, Alessandra ; Schwenk, Jochen M ; Nilsson, Peter ; Al‐Khalili Szigyarto, CristinaEMBO molecular medicine, 2014-07, Vol.6 (7), p.918-936 [Periódico revisado por pares]England: John Wiley & Sons, IncTexto completo disponível |
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Material Type: Artigo
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Cross‐sectional serum metabolomic study of multiple forms of muscular dystrophySpitali, Pietro ; Hettne, Kristina ; Tsonaka, Roula ; Sabir, Ekrem ; Seyer, Alexandre ; Hemerik, Jesse B.A. ; Goeman, Jelle J. ; Picillo, Esther ; Ergoli, Manuela ; Politano, Luisa ; Aartsma‐Rus, AnnemiekeJournal of cellular and molecular medicine, 2018-04, Vol.22 (4), p.2442-2448 [Periódico revisado por pares]England: John Wiley & Sons, IncTexto completo disponível |
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Material Type: Artigo
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The impact of Patient and Public Involvement in the SlowMo study: Reflections on peer innovationGreenwood, Kathryn ; Robertson, Sam ; Vogel, Evelin ; Vella, Claire ; Ward, Thomas ; McGourty, Alison ; Sacadura, Cat ; Hardy, Amy ; Rus‐Calafell, Mar ; Collett, Nicola ; Emsley, Richard ; Freeman, Daniel ; Fowler, David ; Kuipers, Elizabeth ; Bebbington, Paul ; Dunn, Graham ; Garety, PhilippaHealth expectations : an international journal of public participation in health care and health policy, 2022-02, Vol.25 (1), p.191-202 [Periódico revisado por pares]England: John Wiley & Sons, IncTexto completo disponível |
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10 |
Material Type: Artigo
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Antisense‐mediated exon skipping: a therapeutic strategy for titin‐based dilated cardiomyopathyGramlich, Michael ; Pane, Luna Simona ; Zhou, Qifeng ; Chen, Zhifen ; Murgia, Marta ; Schötterl, Sonja ; Goedel, Alexander ; Metzger, Katja ; Brade, Thomas ; Parrotta, Elvira ; Schaller, Martin ; Gerull, Brenda ; Thierfelder, Ludwig ; Aartsma‐Rus, Annemieke ; Labeit, Siegfried ; Atherton, John J ; McGaughran, Julie ; Harvey, Richard P ; Sinnecker, Daniel ; Mann, Matthias ; Laugwitz, Karl‐Ludwig ; Gawaz, Meinrad Paul ; Moretti, AlessandraEMBO molecular medicine, 2015-05, Vol.7 (5), p.562-576 [Periódico revisado por pares]England: John Wiley & Sons, IncTexto completo disponível |