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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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Detalles Bibliográficos
Publicado en:Nucleic Acid Ther
Main Authors: van Putten, Maaike, Tanganyika-de Winter, Christa, Bosgra, Sieto, Aartsma-Rus, Annemieke
Formato: Artigo
Idioma:Inglês
Publicado: Mary Ann Liebert, Inc., publishers 2019
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Acceso en liña: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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