Target intelligence / Profile preview

USP30 antisense RNA 1 (USP30-AS1)

Target
USP30-AS1
Molecular classification
Long non-coding RNA (lncRNA), Antisense RNA, Other
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Overview

USP30 antisense RNA 1 (USP30-AS1) is a human long non-coding RNA (lncRNA) transcribed antisense to the USP30 gene locus. It functions as an epigenetic and post-transcriptional regulator, controlling diverse cellular processes, including mitochondrial homeostasis and mitophagy. USP30-AS1 plays distinct, context-dependent roles in cancer and immune responses: it serves as a negative regulator of mitophagy in glioblastoma, correlating with poor survival, and maintains leukemia cell viability in acute myeloid leukemia, acting as a pro-survival oncogenic regulator[1]. In viral infection settings, USP30-AS1 is robustly upregulated as an interferon-stimulated gene and may participate in the JAK-STAT pathway response; its upregulation suppresses viral protein synthesis, thus modulating host–pathogen interactions[2]. Mechanistically, USP30-AS1 can act in cis to regulate the neighboring USP30 gene and can modulate immune evasion in leukemia by interfering with HLA-I trafficking[1]. Upregulation or knockdown affects cell proliferation and apoptosis depending on the tissue context, and it has value as a disease biomarker in multiple settings[1][2].

Other names
USP30-AS1lncRNA USP30-AS1Long non-coding RNA USP30 antisense RNA 1
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Biological functions

Regulation of mitochondrial homeostasisEpigenetic and post-transcriptional gene regulationModulates immune response (interferon-stimulated)Regulation of mitophagyApoptosis modulationCell proliferation regulation
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Disease associations

CancerGlioblastomaAcute myeloid leukemiaInfection (specifically, influenza A virus and interferon response)Other
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Safety considerations

Therapeutic targeting may alter fundamental cellular pathways, such as apoptosis and immune response, raising concern for unintended effects on normal cells[1][2].The complexity and tissue specificity of lncRNA function introduces challenges for targeted therapy and off-target effects may be difficult to predict[1][2].
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Biomarkers

Biomarker for prognosis in glioblastoma (high levels correlate with poor survival)Biomarker for adverse prognosis in acute myeloid leukemia (overexpressed via hypomethylation and correlates with poor outcome)

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