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Target intelligence / Profile preview
Huntingtin (HTT)
- Target
- HTT
- Molecular classification
- HEAT repeat-containing protein, Protein interaction hub
01
Overview
The Huntingtin protein (Htt), encoded by the HTT gene, is a large, ubiquitously expressed protein with highest levels in the brain. It plays crucial roles in neuronal signaling, intracellular transport, apoptosis regulation, and DNA repair. A key feature is the polyglutamine (polyQ) tract in the N-terminal region. Expansion of this tract due to CAG repeats leads to the production of toxic huntingtin fragments that aggregate, causing Huntington's disease, a neurodegenerative disorder characterized by movement disorders and cognitive decline. HTT serves as a protein-protein interaction hub.
- Other names
- IT15Huntingtin Protein
02
Mechanism of action
Not applicable; HTT is the target, not the drug.
03
Biological functions
Chemical signaling within neuronsIntracellular transportProtein bindingApoptosis regulationDNA repair
04
Disease associations
Neurodegenerative diseaseHuntington's diseaseLopes-Maciel-Rodan syndrome (rare)
05
Safety considerations
Embryonic lethality with complete loss-of-function mutationsNeurotoxicity of mutant huntingtin fragmentsChallenges in targeting protein aggregates
06
Biomarkers
CAG repeat length in HTT geneHuntingtin protein levelsHuntingtin aggregates
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