The development of novel diagnostics and treatment for refractory rhabdomyosarcoma

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Presently, there is no body fluid biomarker of rhabdomyosarcoma (RMS). The normalized serum miR-206 expression level could be used to differentiate between RMS and non-RMS tumors, with a sensitivity of 1.0 and a specificity of 0.913. Serum miR-206 expression level can be a novel serum biomarker of RMS.We identified a PAX3-NCOA2 fusion gene in a case of embryonal rhabdomyosarcoma. To understand the role of this translocation in RMS tumorigenesis, we established two types of stable mouse myoblast C2C12 cell lines expressing PAX3-NCOA2 and PAX3-FKHR, respectively. Expression array analysis revealed that RPS6KA1 was commonly upregulated in both cell lines. We are going to validate RPS6KA inhibitor as a novel treatment for rhabdomyosarcoma.