期刊
FUTURE NEUROLOGY
卷 13, 期 2, 页码 101-121出版社
FUTURE MEDICINE LTD
DOI: 10.2217/fnl-2017-0035
关键词
ASO; chorea; drug therapies; Huntington disease; mHtt; RNAi; VMAT2
资金
- US NIH through Center for Protein Folding Machinery [5PN2EY016525, 5U24RR021992]
- US NIH
Huntington disease (HD) is an autosomal dominantly inherited neurodegenerative disease that affects motor, cognitive and psychiatric functions, and ultimately leads to death. The pathology of the disease is based on an expansion of CAG repeats in exon 1 of the huntingtin gene on chromosome 4, which produces a mutant huntingtin protein (mHtt). This protein is involved in neurotoxicity and brain atrophy, and can form beta-sheets and abnormal mHtt aggregates. Currently, there are no approved effective treatments for HD, although tetrabenazine (Xenazine T) and deutetrabenazine (AUSTEDO T) have been approved for treatment of the motor symptom chorea in HD. This literature review aims to address the latest research on promising therapeutics based on influencing the hypothesized pathological mechanisms.
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