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Target intelligence / Profile preview
Expanded CAG Repeat RNA Transcript (CAG RNA)
- Target
- CAG RNA
- Molecular classification
- RNA, Non-coding RNA, mRNA
01
Overview
Expanded CAG repeat RNA transcripts are mRNA or noncoding RNA molecules containing abnormally long stretches of CAG trinucleotide repeats. They contribute to the pathogenesis of polyglutamine diseases through RNA toxicity, nuclear foci formation, and repeat-associated non-AUG (RAN) translation.
- Other names
- Expanded CAG RNACAG repeat expansionToxic CAG RNA
02
Mechanism of action
Binding to expanded CAG repeats, blocking harmful RNA interactions, and/or reducing overall transcript levels.
03
Biological functions
RNA Splicing RegulationProtein SequestrationGene Expression RegulationRAN Translation
04
Disease associations
Neurodegenerative diseasePolyglutamine diseaseHuntington's DiseaseSpinocerebellar Ataxia
05
Safety considerations
Off-target effects of ASOsPotential for exon skippingDelivery to the central nervous system
06
Interacting drugs
Antisense Oligonucleotides (ASOs)
1 more in the full profile.
07
Biomarkers
CAG repeat lengthNuclear foci formationMBNL1 sequestration
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