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Rhabdomyosarcoma 2-associated transcript (non-protein coding) (RMST)

Target
RMST
Molecular classification
Long non-coding RNA (lncRNA), Other (regulatory RNA, not classified as receptor, enzyme, transporter, etc.)
01

Overview

Rhabdomyosarcoma 2-associated transcript (RMST) is a long non-coding RNA (lncRNA), typically comprising >200 nucleotides, and is not translated into protein. RMST is highly expressed in the brain and neural tissues, where it regulates neurogenesis, neuron differentiation, and survival through interactions with transcription factors (e.g., SOX2) and RNA-binding proteins (e.g., HuR, FUS, hnRNPA2/B1, hnRNPK). RMST also plays a key role in endothelial cell angiogenesis, promoting survival, proliferation, and migration, and its levels are significantly altered under hypoxic conditions. Pathologically, RMST acts as a tumor suppressor in several cancers and participates in neuropathic pain by regulating gene expression (notably DNMT3A methyltransferase) in damaged neurons. RMST knockdown or overexpression can modulate disease states, and RMST expression levels serve as biomarkers of prognosis and disease activity, but it is not currently a direct drug target—modulation involves RNA-based approaches.

Other names
NCRMSNCRNA00054LINC00054non-coding RNA in RMSnon-protein coding RNA 54long intergenic non-protein coding RNA 54non-coding RNA in rhabdomyosarcoma (RMS)rhabdomyosarcoma 2-associated transcript (non-coding RNA)rhabdomyosarcoma 2-associated transcript (non-protein coding)
02

Mechanism of action

Potential therapeutic manipulation of RMST expression (e.g., inhibition to suppress neuropathic pain, overexpression as tumor suppressor in cancer). Not classical drug-receptor/enzyme mechanisms—typically involves RNA interference, gapmers, or molecular tools targeting RNA.

03

Biological functions

Neurogenesis and neuronal differentiation (especially brain development, dopaminergic neurons)Regulation of angiogenesis in endothelial cellsCell survival, proliferation, migration, and differentiation (neural and endothelial)Regulation of mRNA stability and DNA methylation through interaction with RNA-binding proteins (e.g., HuR, FUS, hnRNPA2/B1, hnRNPK)Tumor suppressor function in various cancers, including inhibition of cell viability, proliferation, migration, and invasion
04

Disease associations

Cancer (glioblastoma, rhabdomyosarcoma, lung adenocarcinoma, triple-negative breast cancer, neuroblastoma, osteosarcoma, etc.)Neuropathic pain (peripheral nerve injury)Angiogenesis-related diseases (cardiovascular/neurovascular conditions)Brain injury (ischemia, stroke models)
05

Safety considerations

Therapeutic targeting of lncRNAs is nascent; off-target RNA effects, delivery challenges, and tissue specificity are notable issuesRMST is widely expressed in the brain and other tissues, raising concerns about unintended neurogenic/angiogenic effects
06

Interacting drugs

None reported directly
07

Biomarkers

RMST expression levels as biomarker for: Neuropathic pain state (in dorsal root ganglion neurons)RMST expression levels as biomarker for: Lung adenocarcinoma progression and prognosis (low RMST correlates with worse outcome)RMST expression levels as biomarker for: Triple-negative breast cancer survival (low RMST correlates with poor prognosis)

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