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Target intelligence / Profile preview
Pathogenic Expanded CUG Repeat RNA (r(CUG)^exp)
- Target
- r(CUG)^exp
- Molecular classification
- Non-coding RNA, mRNA (mutated)
01
Overview
Pathogenic expanded CUG repeat RNAs are structured non-coding regions within certain transcripts that cause disease primarily through toxic gain-of-function at the RNA level. Their unique structure enables them to aberrantly interact with key cellular proteins leading to widespread dysregulation—most prominently seen in myotonic dystrophy type 1—and they represent an important target for therapeutic intervention using structure-specific molecules or nucleic acid-based drugs.
- Other names
- Expanded CUG repeatsCUG repeat RNAr(CUG)exp tractsMutant DMPK mRNA
02
Mechanism of action
Disrupts protein sequestration; promotes selective degradation/cleavage of mutant transcripts; modifies RNA structure
03
Biological functions
RNA-protein interactionSplicing regulation (disruption)Ribonucleoprotein aggregate formationMitochondrial dysfunction inductionCellular stress induction
04
Disease associations
Myotonic dystrophy type 1 (DM1)Huntington’s disease-like syndromes (some forms)Fragile X-associated tremor/ataxia syndrome (FXTAS - when different triplet repeats occur)
05
Safety considerations
Off-target effects of small moleculesNon-selective ASO bindingImmune response to ASOsDelivery of therapeutics to affected tissuesSpecificity of targeting pathological versus physiological repeats
06
Interacting drugs
Small molecules (CUG-binding)
1 more in the full profile.
07
Biomarkers
r(CUG)^exp levels in patient tissuesMBNL protein sequestration in nuclear fociMis-splicing patterns of target genesExpression levels of cell cycle control genes (e.g., TP53)Senescence mediators (e.g., IGFBP3)Matrix remodeling enzymes (MMPs)Antioxidant response elements (Nrf2 targets)
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