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Target intelligence / Profile preview
Mutant Huntingtin (mHTT)
- Target
- mHTT
- Molecular classification
- Protein, Aggregation-prone protein
01
Overview
Mutant huntingtin (mHTT) is the altered form of the huntingtin protein, resulting from an expanded CAG trinucleotide repeat in the HTT gene. This expansion leads to a polyglutamine (polyQ) tract exceeding 36 glutamines, causing Huntington's disease. mHTT disrupts various cellular processes including vesicular transport, transcription, and mitochondrial function, leading to neuronal dysfunction and cell death. It also forms toxic aggregates and exhibits aberrant interactions with other proteins. Therapeutic strategies aim to reduce mHTT levels, prevent aggregation, or mitigate its toxic effects.
- Other names
- mutant HTTexpanded HuntingtinpolyQ Huntingtin
02
Mechanism of action
N/A
03
Biological functions
Disruption of vesicular transportDisruption of endocytosisDisruption of autophagyTranscriptional dysregulationMitochondrial dysfunctionAggregate formationAberrant protein-protein interactions
04
Disease associations
Huntington's DiseaseNeurodegenerative disease
05
Safety considerations
Ubiquitous expression may lead to off-target effects.Difficult to selectively target mutant huntingtin over wild-type.Blood brain barrier penetration for therapeutics targeting the brain.
06
Biomarkers
CAG repeat length in HTT gene
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