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Pathogenic Repeat Expansion-Containing mRNAs (Repeat Expansion RNAs)
- Target
- Repeat Expansion RNAs
- Molecular classification
- mRNA, RNA
01
Overview
Pathogenic repeat expansion-containing mRNAs represent a class of transcripts whose abnormally long repetitive sequences drive disease through multiple converging mechanisms—primarily by forming toxic RNA structures that disrupt cell function directly or indirectly via aberrant protein production through unconventional translation pathways. Their instability underlies variable expressivity and anticipation seen clinically across affected families.
- Other names
- Expanded Repeat RNAsRepeat-Associated RNAsToxic Repeat RNAs
02
Mechanism of action
Reducing levels of toxic RNA/protein species, Disrupting pathological RNA-protein interactions, Inhibiting RAN translation, Restoring normal expression/function where loss-of-function occurs
03
Biological functions
Gene expression regulation (aberrant)RNA-protein interaction (disrupted)Translation (aberrant)RBP sequestration
04
Disease associations
Neurodegenerative diseaseNeurological disordersMyotonic dystrophySpinocerebellar ataxiaAmyotrophic lateral sclerosisFrontotemporal dementiaFragile X syndromeHuntington's disease
05
Safety considerations
Off-target effects of RNA-targeting therapiesImmune responses to RNA-targeting therapiesDelivery of therapeutics to the central nervous systemSpecificity of targeting expanded repeats without affecting normal transcripts
06
Biomarkers
Repeat length in affected tissuesLevels of RAN translation productsExpression levels of affected genes
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