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Material Type: Artigo
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Quantum rods as nanocarriers of gene therapyAalinkeel, Ravikumar ; Nair, Bindukumar ; Reynolds, Jessica L. ; Sykes, Donald E. ; Law, Wing-Cheung ; Mahajan, Supriya D. ; Prasad, Paras N. ; Schwartz, Stanley A.Drug delivery, 2012-05, Vol.19 (4), p.220-231 [Periódico revisado por pares]England: Informa HealthcareTexto 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, A.RNA Biology, 2010-08, Vol.7 (4), p.453-461Sem texto completo |
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3 |
Material Type: Artigo
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Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cellsAARTSMA-RUS, A ; KAMAN, W. E ; BREMMER-BOUT, M ; JANSON, A. A. M ; DEN DUNNEN, J. T ; VAN OMMEN, G-Jb ; VAN DEUTEKOM, J. C. TGene therapy, 2004-09, Vol.11 (18), p.1391-1398 [Periódico revisado por pares]Basingstoke: Nature Publishing GroupTexto completo disponível |
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Material Type: Artigo
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Therapeutic exon skipping for dysferlinopathies?Aartsma-Rus, A. ; Singh, K.H.K. ; Fokkema, I.F.A.C. ; Ginjaar, I.B. ; Ommen, G.J. van ; Dunnen, J.T. den ; Maarel, S.M. van derEuropean Journal of Human Genetics, 2010-08, Vol.18 (8), p.889-894Sem texto completo |
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Material Type: Artigo
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Antisense-Mediated Exon Skipping: Networking to Meet Opportunities and to Overcome ChallengesAartsma-Rus, AnnemiekeNucleic acid therapeutics, 2014-02, Vol.24 (1), p.1-3 [Periódico revisado por pares]United States: Mary Ann Liebert, IncTexto completo disponível |
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Material Type: Artigo
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Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophyAartsma-Rus, Annemieke ; Bremmer-Bout, Mattie ; Janson, Anneke A.M ; den Dunnen, Johan T ; van Ommen, Gert-Jan B ; van Deutekom, Judith C.TNeuromuscular disorders : NMD, 2002-10, Vol.12, p.S71-S77 [Periódico revisado por pares]England: Elsevier B.VTexto completo disponível |
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Material Type: Artigo
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The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European ExperienceAartsma-Rus, Annemieke ; De Waele, Liesbeth ; Houwen-Opstal, Saskia ; Kirschner, Janbernd ; Krom, Yvonne D ; Mercuri, Eugenio ; Niks, Erik H ; Straub, Volker ; van Duyvenvoorde, Hermine A ; Vroom, ElizabethJournal of neuromuscular diseases, 2023-01, Vol.10 (3), p.315-325 [Periódico revisado por pares]NetherlandsSem texto completo |
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Material Type: Artigo
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Therapeutic exon skipping for dysferlinopathies?AARTSMA-RUS, Annemieke ; SINGH, Kavita Hk ; FOKKEMA, Ivo Fac ; GINJAAR, Ieke B ; VAN OMMEN, Gert-Jan ; DEN DUNNEN, Johan T ; VAN DER MAAREL, Silvère MEuropean journal of human genetics : EJHG, 2010-08, Vol.18 (8), p.889-894 [Periódico revisado por pares]Basingstoke: Nature Publishing GroupTexto completo disponível |
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Material Type: Artigo
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Spinal muscular atrophy: Molecular mechanism of pathogenesis, diagnosis, therapeutics, and clinical trials in the Indian contextAasdev, Ashutosh ; Sreelekshmi, R S ; Iyer, V Rajesh ; Moharir, Shivranjani CJournal of biosciences, 2024-03, Vol.49 (1), p.36 [Periódico revisado por pares]New Delhi: Springer IndiaTexto completo disponível |
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10 |
Material Type: Artigo
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Novel Exon-Skipping Therapeutic Approach for the DMD Gene Based on Asymptomatic Deletions of Exon 49Abaji, Mario ; Gorokhova, Svetlana ; Da Silva, Nathalie ; Busa, Tiffany ; Grelet, Maude ; Missirian, Chantal ; Sigaudy, Sabine ; Philip, Nicole ; Leturcq, France ; Lévy, Nicolas ; Krahn, Martin ; Bartoli, MarcGenes, 2022-07, Vol.13 (7), p.1277 [Periódico revisado por pares]Basel: MDPI AGTexto completo disponível |