Target intelligence / Profile preview

Mutant Huntingtin Protein (mHtt)

Target: mHtt
Molecular classification: Protein, Other

Overview

The mutant huntingtin protein (mHtt) is a mutated form of the huntingtin protein that causes Huntington's disease. The mutation involves an expansion of polyglutamine repeats in the N-terminal region, leading to protein misfolding, aggregation, and disruption of various cellular functions, including intracellular trafficking, gene transcription, and mitochondrial function. mHtt interacts with numerous proteins and impairs normal cellular processes, ultimately leading to neurodegeneration.

Biological functions

Intracellular traffickingGene transcription regulationMitochondrial functionAxonal transportVesicle trafficking

Disease associations

Neurodegenerative disease

Safety considerations

Off-target effects due to wide range of interactionsDelivery challenges to the brain

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