Target intelligence / Profile preview

Antisense noncoding mitochondrial RNA (ASncmtRNA)

Target
ASncmtRNA
Molecular classification
Long non-coding RNA, Mitochondrial RNA, Regulatory RNA, Other
01

Overview

Antisense noncoding mitochondrial RNA (ASncmtRNA) refers to a family of long non-coding RNAs transcribed antisense from the mitochondrial 16S rRNA gene. The ASncmtRNAs include at least two principal members, ASncmtRNA-1 and ASncmtRNA-2, and are characterized by extended double-stranded stem-loop structures[1][3][4]. They are present in normal proliferating cells but consistently downregulated in a variety of tumor cells and tissues[2][3][4][5][6]. Knockdown of these RNAs with chemically modified antisense oligonucleotides (such as Andes-1537 or ASO-1537S) induces cell cycle arrest and massive apoptosis selectively in tumor cells, but not in non-tumor, normal cells[2][3][5][6]. This selective vulnerability positions ASncmtRNAs as promising, novel therapeutic targets and potential universal biomarkers for cancer[2][3][5][6]. Mechanistically, targeting ASncmtRNAs alters the expression of key cell cycle regulators, activates cell death pathways, and influences microRNA biogenesis—most notably that of miR-4485-3p, which is derived from ASncmtRNA-2[1][3]. The ability of ASncmtRNAs to act outside the mitochondria, including in the cytosol and nucleus, further supports their broad regulatory roles[2][5]. Key clinical highlights: - ASncmtRNA knockdown is under investigation as a strategy for cancer therapy and has demonstrated efficacy in vitro and in animal models for numerous cancer types (including breast, bladder, renal, and melanoma)[2][3][5][6]. - Biomarker potential is suggested by universal downregulation of ASncmtRNAs across tumor samples, independent of origin[2][4][5][6]. No evidence suggests the target name or classification is incorrect; the molecule is not a receptor, enzyme, or transporter, but a regulatory long non-coding RNA with established significance in cancer biology.

Other names
ASncmtRNAsAntisense noncoding mitochondrial RNAsASncmtRNA-1ASncmtRNA-2
02

Mechanism of action

Antisense oligonucleotide-mediated knockdown induces apoptosis in tumor cells; Downregulation of cell cycle and survival proteins (e.g., cyclin B1, D1, CDK1, CDK4, survivin)

03

Biological functions

Cell cycle regulationApoptosisCell proliferationTumor suppressionRegulation of microRNA biogenesis (e.g., miR-4485-3p)
04

Disease associations

Cancer
05

Safety considerations

Potential off-target effects of antisense oligonucleotidesCell-type specificity (apoptosis induced in tumor but not normal cells)
06

Interacting drugs

Andes-1537 (antisense oligonucleotide targeting ASncmtRNAs)

1 more in the full profile.

07

Biomarkers

Downregulation or loss of ASncmtRNAs as a biomarker for tumor cells/cancersUpregulation of miR-4485-3p following ASncmtRNA-2 knockdown

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