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Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy

In the last two decades, antisense oligonucleotides (AONs) that induce corrective exon skipping have matured as promising therapies aimed at tackling the dystrophin deficiency that underlies the severe and progressive muscle fiber degeneration in Duchenne muscular dystrophy (DMD) patients. Pioneerin...

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Detalles Bibliográficos
Autores principales:	van Deutekom, Judith, Beekman, Chantal, Bijl, Suzanne, Bosgra, Sieto, van den Eijnde, Rani, Franken, Dennis, Groenendaal, Bas, Harquouli, Bouchra, Janson, Anneke, Koevoets, Paul, Mulder, Melissa, Muilwijk, Daan, Peterburgska, Galyna, Querido, Bianca, Testerink, Janwillem, Verheul, Ruurd, de Visser, Peter, Weij, Rudie, Aartsma-Rus, Annemieke, Puoliväli, Jukka, Bragge, Timo, O'Neill, Charles, Datson, Nicole A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2023
Materias:	Original Papers
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10277991/ https://www.ncbi.nlm.nih.gov/pubmed/37036788 http://dx.doi.org/10.1089/nat.2022.0063

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