Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice

Duchenne muscular dystrophy (DMD) is a severe childhood muscle disease primarily caused by the lack of functional dystrophin at the muscle fiber membranes. Multiple therapeutic approaches are currently in (pre)clinical development, aimed at restoring expression of (truncated) dystrophin. Key questio...

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Detalhes bibliográficos
Publicado no:Nucleic Acid Ther
Main Authors: Datson, Nicole A., Bijl, Suzanne, Janson, Anneke, Testerink, Janwillem, van den Eijnde, Rani, Weij, Rudie, Puoliväli, Jukka, Lehtimäki, Kimmo, Bragge, Timo, Ahtoniemi, Toni, van Deutekom, Judith C.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc., publishers 2020
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Original Papers
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7049912/
https://ncbi.nlm.nih.gov/pubmed/31821107
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2019.0824
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