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

• मुख्य लेखकों: Ingrid E C Verhaart (लेखक), Laura van Vliet-van den Dool (लेखक), Jessica A Sipkens (लेखक), Sjef J de Kimpe (लेखक), Ingrid G M Kolfschoten (लेखक), Judith C T van Deutekom (लेखक), Lia Liefaard (लेखक), Jim E Ridings (लेखक), Steve R Hood (लेखक), Annemieke Aartsma-Rus (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2014-01-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...