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Detailed genetic and functional analysis of the hDMDdel52/mdx mouse model

Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disorder caused by reading frame disrupting mutations in the DMD gene leading to absence of functional dystrophin. Antisense oligonucleotide (AON)-mediated exon skipping is a therapeutic approach aimed at restoring the reading...

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Detalles Bibliográficos
Autores principales:	Yavas, Alper, Weij, Rudie, van Putten, Maaike, Kourkouta, Eleni, Beekman, Chantal, Puoliväli, Jukka, Bragge, Timo, Ahtoniemi, Toni, Knijnenburg, Jeroen, Hoogenboom, Marlies Elisabeth, Ariyurek, Yavuz, Aartsma-Rus, Annemieke, van Deutekom, Judith, Datson, Nicole
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2020
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7757897/ https://www.ncbi.nlm.nih.gov/pubmed/33362201 http://dx.doi.org/10.1371/journal.pone.0244215

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