Strategic study of atelocollagen-mediated application of myostatin-targeting siRNA for therapeutic use for muscular atrophy diseases

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Muscular dystrophy is a severe muscle wasting disorder caused by gene mutations. The disease is lethal, but the treatment of the basic genetic defect has not been established. Recently, many studies have been performed towards establishing an effective treatment for muscular dystrophy.After local application of the Mst-siRNA/ ATCOL complex, masseter muscles were enlarged, while no significant change was observed on the contralateral side. Histological analysis showed that the myofibril sizes of the masseter muscles treated with the Mst-siRNA/ ATCOL complex were larger than those of the control side. The Mst-siRNA/ ATCOL treated muscles were further examined by a real-time PCR analysis, showing a significant down-regulation of Mst gene expression in the treated masseters of both wild type and mCAV-3Tg mice. From EMG records, the amounts of masseter muscle activity in mCAV-3Tg mice were increased by local administration of Mst-siRNA/ ATCOL complex. Furthermore, systemic application of the Mst-siRNA/ ATCOL complex induced considerable recovery of contractile forces for TA muscles in mCAV-3Tg mice.