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PHOX2B antisense RNA 1 (PHOX2B-AS1)
- Target
- PHOX2B-AS1
- Molecular classification
- Long non-coding RNA (lncRNA), Other (RNA gene)
01
Overview
PHOX2B antisense RNA 1 is a long non-coding RNA transcribed from the PHOX2B locus and acts primarily by favoring translation of PHOX2B mRNA into protein without changing mRNA levels. PHOX2B-AS1 and PHOX2B mRNA show predominantly cytoplasmic localization. Functional studies (e.g., gapmer-mediated knockdown) demonstrate that reducing PHOX2B-AS1 leads to a decrease in PHOX2B protein levels, indicating PHOX2B-AS1 promotes PHOX2B translation rather than its transcription. Aberrant expression or function of PHOX2B-AS1 may contribute to the pathogenesis of congenital central hypoventilation syndrome (CCHS), a disorder due to impaired development or function of autonomic and respiratory neural circuits. There are currently no drugs that directly target PHOX2B-AS1, but its regulatory influence on PHOX2B makes it a subject of interest in neurodevelopmental disease research.
- Other names
- PHOX2B-AS1PHOX2B Antisense RNA 1
02
Mechanism of action
No established drugs target PHOX2B-AS1, so mechanisms of action are not defined
03
Biological functions
Post-transcriptional regulation (regulation of PHOX2B protein translation)Regulation of gene expression (by modulating PHOX2B levels)
04
Disease associations
Congenital central hypoventilation syndrome (CCHS, also called Haddad syndrome)Other neurodevelopmental disorders (by inference from PHOX2B's role)
05
Safety considerations
There are no direct safety concerns or therapeutic challenges known, as PHOX2B-AS1 is not currently a drug target
06
Biomarkers
PHOX2B-AS1 expression (potential, but not established in clinical use) in pathologies such as CCHS
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