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

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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Main Authors: Alper Yavas, Rudie Weij, Maaike van Putten, Eleni Kourkouta, Chantal Beekman, Jukka Puoliväli, Timo Bragge, Toni Ahtoniemi, Jeroen Knijnenburg, Marlies Elisabeth Hoogenboom, Yavuz Ariyurek, Annemieke Aartsma-Rus, Judith van Deutekom, Nicole Datson
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2020-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0244215

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