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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Publicat a:Nucleic Acid Ther
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc., publishers 2020
Matèries:
Original Papers
Accés en línia: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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