期刊
TRENDS IN BIOCHEMICAL SCIENCES
卷 38, 期 8, 页码 378-385出版社
ELSEVIER SCIENCE LONDON
DOI: 10.1016/j.tibs.2013.05.003
关键词
Huntington's disease; molecular mechanisms; therapeutic approaches
资金
- NIA NIH HHS [R37 AG026647, R01 AG026647] Funding Source: Medline
- NIGMS NIH HHS [R01 GM038109] Funding Source: Medline
Huntington's disease (HD) is a progressive neurodegenerative disorder for which no disease modifying treatments exist. Many molecular changes and cellular consequences that underlie HD are observed in other neurological disorders, suggesting that common pathological mechanisms and pathways may exist. Recent findings have enhanced our understanding of the way cells regulate and respond to expanded polyglutamine proteins such as mutant huntingtin. These studies demonstrate that in addition to effects on folding, aggregation, and clearance pathways, a general transcriptional mechanism also dictates the expression of polyglutamine proteins. Here, we summarize the key pathways and networks that are important in HD in the context of recent therapeutic advances and highlight how their interplay may be of relevance to other protein folding disorders.
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