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

• Asıl Yazarlar: Ingrid E C Verhaart (Yazar), Laura van Vliet-van den Dool (Yazar), Jessica A Sipkens (Yazar), Sjef J de Kimpe (Yazar), Ingrid G M Kolfschoten (Yazar), Judith C T van Deutekom (Yazar), Lia Liefaard (Yazar), Jim E Ridings (Yazar), Steve R Hood (Yazar), Annemieke Aartsma-Rus (Yazar)
• Materyal Türü: Kitap
• Baskı/Yayın Bilgisi: Elsevier, 2014-01-01T00:00:00Z.
• Konular: article
• Online Erişim: 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...