PAX3-FOXO1 drives miR-486-5p and represses miR-221 contributing to pathogenesis of alveolar rhabdomyosarcoma
PAX3-FOXO1 drives miR-486-5p and represses miR-221 contributing to pathogenesis of alveolar rhabdomyosarcoma
Rhabdomyosarcoma is the most common soft-tissue sarcoma in childhood and histologically resembles developing skeletal muscle. Alveolar rhabdomyosarcoma (ARMS) is an aggressive subtype with a higher rate of metastasis and poorer prognosis. The majority of ARMS tumors (80%) harbor a PAX3-FOXO1 or less commonly a PAX7-FOXO1 fusion gene. The presence of either the PAX3-FOXO1 or PAX7-FOXO1 fusion gene foretells a poorer prognosis resulting in clinical re-classification as either fusion-positive (FP-RMS) or fusion-negative RMS (FN-RMS). The PAX3/7-FOXO1 fusion genes result in the production of a rogue transcription factors that drive FP-RMS pathogenesis and block myogenic differentiation. Despite knowing the molecular driver of FP-RMS, targeted therapies have yet to make an impact for patients, highlighting the need for a greater understanding of the molecular consequences of PAX3-FOXO1 and its target genes including microRNAs. Here we show FP-RMS patient-derived xenografts and cell lines display a distinct microRNA expression pattern. We utilized both loss- and gain-of function approaches in human cell lines with knockdown of PAX3-FOXO1 in FP-RMS cell lines and expression of PAX3-FOXO1 in human myoblasts and identified microRNAs both positively and negatively regulated by the PAX3-FOXO1 fusion protein. We demonstrate PAX3-FOXO1 represses miR-221/222 that functions as a tumor suppressing microRNA through the negative regulation of CCND2, CDK6, and ERBB3. In contrast, miR-486-5p is transcriptionally activated by PAX3-FOXO1 and promotes FP-RMS proliferation, invasion, and clonogenic growth. Inhibition of miR-486-5p in FP-RMS xenografts decreased tumor growth, illustrating a proof of principle for future therapeutic intervention. Therefore, PAX3-FOXO1 regulates key microRNAs that may represent novel therapeutic vulnerabilities in FP-RMS.
- St. Jude Children's Research Hospital United States
Muscle Neoplasms, Oncogene Proteins, Fusion, Gene Expression Profiling, Mice, SCID, Microarray Analysis, Article, Gene Expression Regulation, Neoplastic, Mice, MicroRNAs, Cell Transformation, Neoplastic, HEK293 Cells, Gene Knockdown Techniques, Animals, Humans, Paired Box Transcription Factors, Female, Child, Cells, Cultured, Rhabdomyosarcoma, Alveolar, Cell Proliferation
Muscle Neoplasms, Oncogene Proteins, Fusion, Gene Expression Profiling, Mice, SCID, Microarray Analysis, Article, Gene Expression Regulation, Neoplastic, Mice, MicroRNAs, Cell Transformation, Neoplastic, HEK293 Cells, Gene Knockdown Techniques, Animals, Humans, Paired Box Transcription Factors, Female, Child, Cells, Cultured, Rhabdomyosarcoma, Alveolar, Cell Proliferation
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