期刊
PARKINSONISM & RELATED DISORDERS
卷 21, 期 3, 页码 169-178出版社
ELSEVIER SCI LTD
DOI: 10.1016/j.parkreldis.2014.12.013
关键词
Huntington's disease; Therapy; Symptomatic relief; Disease-modifying treatment; Experimental medicine
Introduction: Huntington's disease (HD) is the most common hereditary neurodegenerative disorder. Despite the fact that both the gene and the mutation causing this monogenetic disorder were identified more than 20 years ago, disease-modifying therapies for HD have not yet been established. Review: While intense preclinical research and large cohort studies in HD have laid foundations for tangible improvements in understanding HD and caring for HD patients, identifying targets for therapeutic interventions and developing novel therapeutic modalities (new chemical entities and advanced therapies using DNA and RNA molecules as therapeutic agents) continues to be an ongoing process. The authors review recent achievements in HD research and focus on approaches towards disease-modifying therapies, ranging from huntingtin-lowering strategies to improving huntingtin clearance that may be promoted by posttranslational HTT modifications. Conclusion: The nature and number of upcoming clinical studies/trials in HD is a reason for hope for HD patients and their families. (C) 2014 Elsevier Ltd. All rights reserved.
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