{"title":"New directions in therapeutics for Huntington disease.","authors":"Katya T Potkin, Steven G Potkin","doi":"10.2217/fnl-2017-0035","DOIUrl":null,"url":null,"abstract":"<p><p>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 <i>huntingtin</i> gene on chromosome 4, which produces a mutant huntingtin protein (mHtt). This protein is involved in neurotoxicity and brain atrophy, and can form β-sheets and abnormal mHtt aggregates. Currently, there are no approved effective treatments for HD, although tetrabenazine (Xenazine™) and deutetrabenazine (AUSTEDO™) 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.</p>","PeriodicalId":12606,"journal":{"name":"Future Neurology","volume":"13 2","pages":"101-121"},"PeriodicalIF":0.6000,"publicationDate":"2018-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/fnl-2017-0035","citationCount":"25","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Future Neurology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.2217/fnl-2017-0035","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2018/5/29 0:00:00","PubModel":"Epub","JCR":"Q4","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
引用次数: 25
Abstract
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 β-sheets and abnormal mHtt aggregates. Currently, there are no approved effective treatments for HD, although tetrabenazine (Xenazine™) and deutetrabenazine (AUSTEDO™) 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.
期刊介绍：
The neurological landscape is changing rapidly. From the technological perspective, advanced molecular approaches and imaging modalities have greatly increased our understanding of neurological disease, with enhanced prospects for effective treatments in common but very serious disorders such as stroke, epilepsy, multiple sclerosis and Parkinson’s disease. Nevertheless, at the same time, the healthcare community is increasingly challenged by the rise in neurodegenerative diseases consequent upon demographic changes in developed countries.
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