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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Udgivet i:PLoS One
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2020
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7757897/
https://ncbi.nlm.nih.gov/pubmed/33362201
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0244215
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