The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice

• Main Authors: Ingrid E C Verhaart (Author), Laura van Vliet-van den Dool (Author), Jessica A Sipkens (Author), Sjef J de Kimpe (Author), Ingrid G M Kolfschoten (Author), Judith C T van Deutekom (Author), Lia Liefaard (Author), Jim E Ridings (Author), Steve R Hood (Author), Annemieke Aartsma-Rus (Author)
• Format: Book
• Published: Elsevier, 2014-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Antisense-mediated exon skipping is currently in clinical development for Duchenne muscular dystrophy (DMD) to amend the consequences of the underlying genetic defect and restore dystrophin expression. Due to turnover of compound, transcript, and protein, chronic treatment with effector molecules (a...