Huntingtin and Huntington’s Disease
Gu Zhen
Abstract
Gu Zhen
Abstract
Many of human diseases,including several neurodegenerative disorders are thought to be caused by misfolding and aggregation of a disease-causing protein. Huntington’s disease (HD) occurs due to an expansion of polyglutamine tract in the protein named huntingtin. Mutant huntingtin forms inclusions in the nucleus and aggregates in the cytoplasm in neurons. We reviewed the mechanisms of protein misfolding in Huntington’s disease and recent progress in developing therapies for HD aimed at preventing or correcting misfolding and aggregation of mutant huntingtin.
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Many of human diseases,including several neurodegenerative disorders are thought to be caused by misfolding and aggregation of a disease-causing protein. Huntington’s disease (HD) occurs due to an expansion of polyglutamine tract in the protein named huntingtin. Mutant huntingtin forms inclusions in the nucleus and aggregates in the cytoplasm in neurons. We reviewed the mechanisms of protein misfolding in Huntington’s disease and recent progress in developing therapies for HD aimed at preventing or correcting misfolding and aggregation of mutant huntingtin.
Key concepts: Huntingtin, Polyglutamine tract, Huntington's disease, Huntingtin Protein, Disease, Protein aggregation, Mutant, Neurodegeneration