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Can I read Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy on EtoBox?
Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy by Dwi U Kemaladewi; Willem MH Hoogaars; Sandra H van Heiningen; Samuel Terlouw; David JJ de Gorter; Johan T den Dunnen; Gert Jan B van Ommen; Annemieke Aartsma-Rus; Peter ten Dijke; Peter AC 't Hoen is a Biochemistry, Genetics and Molecular Biology article available to read on EtoBox.
What is Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy about?
## Background Myostatin is a potent muscle growth inhibitor that belongs to the Transforming Growth Factor-β (TGF-β) family. Mutations leading to non functional myostatin have been associated with hypermuscularity in several organisms. By contrast, Duchenne muscular dystrophy (DMD) is characterized by a loss of muscle fibers and impaired regeneration. In this study, we aim to knockdown myostatin by means of exon skipping, a technique which has been successfully applied to reframe the genetic defect of dystrophin gene in DMD patients. ## Methods We targeted myostatin exon 2 using antisense oligonucleotides (AON) in healthy and DMD-derived myotubes cultures. We assessed the exon skipping level, transcriptional expression of myostatin and its target genes, and combined myostatin and several dystrophin AONs. These AONs were also applied in the __mdx__ mice models via intramuscular injections. ## Results Myostatin AON induced exon 2 skipping in cell cultures and to a lower extent in the __mdx__ mice. It was accompanied by decrease in myostatin mRNA and enhanced __MYOG__ and __MYF5__ expression. Furthermore, combination of myostatin and dystrophin AONs induced simultaneous skipping of bo
Who reads Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy?
It is typically read by researchers, students, and practitioners in Biochemistry, Genetics and Molecular Biology.
- Author
- Dwi U Kemaladewi; Willem MH Hoogaars; Sandra H van Heiningen; Samuel Terlouw; David JJ de Gorter; Johan T den Dunnen; Gert Jan B van Ommen; Annemieke Aartsma-Rus; Peter ten Dijke; Peter AC 't Hoen
- Publisher
- BioMed Central; Springer (Biomed Central Ltd.); London: BioMed Central, [2008]-; Springer Science and Business Media LLC (ISSN 1755-8794)
- Published
- 2011
- Language
- EN
- Field
- Biochemistry, Genetics and Molecular Biology (Life Sciences)