miR-126 Inhibitor miRisten

**Semantic type:** Pharmacologic Substance

**Definition:** An inhibitor of microRNA miR-126 (MiR-126-3p) composed of an anti-miR-126 RNA antisense oligodeoxynucleotide (ODN) linked to a cytosine-phosphate-guanine (CpG) DNA ODN, with potential antineoplastic activity. Upon administration, miR-126 inhibitor miRisten targets, binds to and inhibits miR-126 in leukemia stem cells (LSCs) and endothelial cells (ECs), thereby preventing transcription and promoting elimination of miR-126 from the circulation. As miR-126 plays a key role in the self-renewal and persistence of cancer-causing LSCs, this may eliminate the LSCs. In addition, the depletion of miR-126 downregulates BCL-2 and disrupts mitochondrial function and metabolism, leading to increased levels of reactive oxygen species (ROS) and the induction of apoptosis in LSCs. miR-126, expressed in normal hematopoietic stem and progenitor cells (HSPC), maintains self-renewal capacity and renewal. Aberrantly increased in LSCs, miR-126 supports homeostasis, quiescence, and activity of LSCs, and enhances mitochondrial metabolism and mitochondrial dynamics in LSCs. CpG DNA ODN, a ligand for scavenger receptors (SRs), is highly expressed on the surface of LSCs and endothelial cells, and allows for efficient cell delivery of anti-miR-126 ODN.

**Synonyms:** - CpG(D19)-Anti-miR-126 - CpG-linked Anti-Mir-126 Oligonucleotide - MicroRNA-126 Inhibitor miRisten - ODN-based CpG-Anti-miRNA-126 - Oligodeoxynucleotide Anti-miR-126 Inhibitor miRisten - miRisten