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44 | Targeted delivery of mir-486 to skeletal muscle: efficacy, tropism, and integrin dependency of MYOAAV2A vs. AAV9 Chiara Ricciardi1, S. Perni1, V. Sorrentino1|2 | 1Department of Molecular and Developmental Medicine, University of Siena, Italy; 2Interdepartmental Program of Molecular Diagnosis and Pathogenetic Mechanisms of Rare Genetic Diseases, Azienda Ospedaliera Universitaria Senese, Italy.
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29 | RNA-based combinatorial multi-target therapy for the treatment of Duchenne muscular dystrophy Mathieu Horeau, C. Pezzini, M. Sandri | Department of Biomedical Sciences, University of Padova, Italy.
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41 | SRT2104 improves muscle and cognitive outcomes and has the potential to enhance μDys gene therapy in Duchenne muscular dystrophy Laura Lociuro1, A. Arcari2, O. Gjana1, G. Scolari1, S.R. Casati1|2, S. Zecchini2, M. Giovarelli2, C. Perrotta2, C. De Palma1 | 1Department of Medical Biotechnology and Translational Medicine (BioMeTra), Università degli Studi di Milano, Segrate (MI), Italy; 2Department of Biomedical and Clinical Sciences "Luigi Sacco" (DIBIC), Università degli Studi di Milano, Italy.
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15 | GDF5 therapeutic potential for Duchenne muscular dystrophy C. Gentil, A. Bourguiba, A. Vergnol, B. Cadot, Z. Guesmia, L. Saillard, P. Meunier, S. Falcone, L. Giordani, France Pietri-Rouxel | Sorbonne Université, INSERM, Institut de Myologie, Centre de Recherche en Myologie, Paris, France.
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Abstract 022 | Lecture: new insights leading to improved designs of micro-dystrophins for use in adeno-associated viruses vectors David Hammers, Cora Hart, Matthew Lee, H. Lee Sweeney, and UF Animal Assessment Core | University of Florida Myology Institute, Gainesville, Florida, USA.
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01 | Investigating skeletal muscle-endothelial cell crosstalk in plasticity and disease José Fernández-Martínez1|2, A. Gensel1|2, E. Germinario2, R.E. Oberkersch3, M.M. Santoro3, B. Blaauw1|2 | 1Veneto Institute of Molecular Medicine, Padua, Italy; 2Department of Biomedical Sciences, University of Padua, Italy; 3Department of Biology, University of Padua, Italy. | José Fernández-Martínez and A. Gensel contributed equally to this work.
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