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ATXN2 antisense RNA (ATXN2-AS)
- Target
- ATXN2-AS
- Molecular classification
- Other (Natural antisense transcript, long noncoding RNA)
01
Overview
ATXN2 antisense RNA (ATXN2-AS) is a bidirectionally transcribed natural antisense transcript at the ATXN2 locus, detected in human brain and peripheral tissues. It contains a CUG trinucleotide repeat region that, when expanded, confers neurotoxicity and contributes to the pathogenesis of SCA2 and ALS. Expression of ATXN2-AS may modulate ATXN2 sense transcript levels and act as an independent pathogenic factor. Its unique repeat expansion profile and neurotoxic properties make ATXN2-AS a candidate therapeutic target, but its low abundance and potential regulatory role pose additional complexities for drug development.
- Other names
- ATXN2-ASATXN2 antisense transcript
02
Mechanism of action
RNA silencing (antisense oligonucleotide inhibition); RNA repeat expansion targeting
03
Biological functions
RNA regulation (bidirectional transcription and modulation of sense ATXN2 expression)Potential gene silencing by antisense RNA mechanismsContribution to neurotoxicity through repeat expansion
04
Disease associations
Neurodegenerative disease (Spinocerebellar ataxia type 2, Amyotrophic lateral sclerosis)
05
Safety considerations
Off-target modulation of sense ATXN2Potential for toxicity with altered antisense transcript levelsChallenges in targeting low-abundance, noncoding RNAs without affecting normal functions
06
Biomarkers
CUG repeat expansion in ATXN2-AS RNA found in patient tissue can act as a biomarker for disease (SCA2, ALS)
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