Abstract
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder, which is characterized by the loss of muscle fibers and replacement by fibrotic tissue due to the lack of the dystrophin protein. Myostatin and Transforming Growth Factor (TGF)-beta play important roles in regulation of muscle differentiation and fibrosis, and are emerging as attractive therapeutic targets for DMD treatment. Both cytokines signal primarily via the overlapping Smad2/3-dependent signaling pathways. In this study we targeted myostatin/TGF-beta type 1 receptors Acvr1b (ALK4) and Tgfbr1 (ALK5) using antisense oligoribonucleotides (AON) targeting regions in the pre-mRNA encoding ligand binding and/or kinase domains of the receptors.
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