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Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results

Duchenne muscular dystrophy is a severe, progressive muscle-wasting disease that is caused by mutations that abolish the production of functional dystrophin protein. The exon skipping approach aims to restore the disrupted dystrophin reading frame, to allow the production of partially functional dys...

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Detalhes bibliográficos
Publicado no:Nucleic Acid Ther
Main Authors: van Putten, Maaike, Tanganyika-de Winter, Christa, Bosgra, Sieto, Aartsma-Rus, Annemieke
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc., publishers 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6461150/
https://ncbi.nlm.nih.gov/pubmed/30672725
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2018.0759
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