Polyglutamine (PolyQ) Diseases: Molecular pathology to treatment
نویسندگان
چکیده
منابع مشابه
New Targets for Therapy in Polyglutamine (polyQ) Expansion Diseases
The polyglutamine (polyQ) repeat disorders are a family of inherited disorders characterized by progressive neurodegeneration, as well as the formation of intracellular protein aggregates. Huntington's disease (HD) is the most prevalent disorder in the family of polyQ diseases. This family includes nine other neurodegenerative disorders: Dentatorubral-pallidoluysian atrophy (DRPLA), Bulbo-Spina...
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Proteins with expanded polyglutamine (polyQ) repeats are involved in human neurodegenerative diseases, via a gain-of-function mechanism of neuronal toxicity involving protein conformational changes that result in the formation and deposition of β-sheet-rich aggregates. Aggregation is dependent on the context and properties of the host protein, such as domain context and location of the repeat t...
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Considerable advances have been recently made in understanding the molecular aspects of pathogenesis and in developing therapeutic approaches for polyglutamine (polyQ) diseases. Studies on pathogenic mechanisms have extended our knowledge of mutant protein toxicity, confirmed the toxicity of mutant transcript and identified other toxic RNA and protein entities. One very promising therapeutic st...
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ژورنال
عنوان ژورنال: Biochemistry Letters
سال: 2015
ISSN: 1687-4773
DOI: 10.21608/blj.2015.63845