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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2020
Materias:
Original Papers
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7049912/
https://www.ncbi.nlm.nih.gov/pubmed/31821107
http://dx.doi.org/10.1089/nat.2019.0824

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