Indication Target/marker/ pathway Summary Licensing status Publication and contact information Cancer Cancer MicroRNA-380-5p (miR-380-5p) Studies in patient samples and in mice suggest that antagonizing miR-380-5p could help treat neuroblastoma. About 76% of primary neuroblastoma samples showed miR-380-5p overexpression relative to expression in normal brain tissue. In a mouse neuroblastoma model, twiceweekly injections of chemically modified anti-miR-380-5p oligonucleotides significantly

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... sed tumor size and weight compared with injection of control oligonucleotides (p=0.01). Next steps include identifying other tumors in which antagonizing miR-380-5p could have a therapeutic effect. Regulus Therapeutics Inc. has miRNA-targeting compounds in preclinical development for multiple indications including cancer.