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Target intelligence / Profile preview
Huntingtin protein mutant form (mHTT)
- Target
- mHTT
- Molecular classification
- Protein, Transcription factor
01
Overview
The mutant form of huntingtin protein (mHTT) arises from an expanded CAG trinucleotide repeat in the HTT gene, leading to an abnormally long polyglutamine tract. This causes Huntington’s disease (HD), characterized by motor dysfunction, psychiatric symptoms, and cognitive decline. mHTT aggregates, disrupts transcription, impairs DNA repair, activates apoptosis, and impairs intracellular trafficking, leading to neuronal dysfunction and neurodegeneration.
- Other names
- Mutant HuntingtinmHTTExpanded PolyQ Huntingtin
02
Mechanism of action
Inhibition of mHTT aggregation; modulation of splicing/expression levels
03
Biological functions
Protein aggregationTranscriptional dysregulationImpaired DNA repairApoptosis activationImpaired intracellular traffickingSynaptic dysfunction
04
Disease associations
Neurodegenerative diseaseHuntington's Disease
05
Safety considerations
Potential for off-target effectsDelivery to the brainLong-term effects of mHTT reduction/inhibition
06
Biomarkers
CAG repeat length in HTT gene
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