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Huntingtin mRNA (mutant) (mutant HTT mRNA)
- Target
- mutant HTT mRNA
- Molecular classification
- mRNA, Non-coding RNA, CAG repeat expansion
01
Overview
Mutant huntingtin mRNA is the messenger RNA transcribed from the HTT gene carrying an expanded CAG trinucleotide repeat, the genetic cause of Huntington’s disease (HD). This mutation leads to a polyglutamine expansion in the resulting protein, but also affects the properties and processing of the mRNA itself. It forms pathological nuclear foci and can sequester RNA-binding proteins, contributing to toxicity independently of the protein. It is a primary target for therapeutic intervention using allele-selective silencing/lowering therapies.
- Other names
- Mutant HTT mRNAmHTT mRNAExpanded CAG huntingtin mRNA
02
Mechanism of action
mRNA degradation, inhibition of translation
03
Biological functions
TranslationRNA splicingRNA processingGene expression regulation
04
Disease associations
Huntington's diseaseNeurodegenerative disease
05
Safety considerations
Off-target effectsImmune response to ASOs/siRNAsIncomplete allele selectivityPotential impact on wild-type HTT expression
06
Interacting drugs
Antisense oligonucleotides (ASOs)
1 more in the full profile.
07
Biomarkers
Mutant HTT mRNA levels in cerebrospinal fluidHTT1a isoform levelsSize of CAG repeat expansionNuclear RNA foci
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