Gene Therapy for Huntington’s Disease: The Final Strategy for a Cure?
Open Access
- 31 January 2022
- journal article
- review article
- Published by The Korean Movement Disorder Society in Journal of Movement Disorders
- Vol. 15 (1) , 15-20
Abstract
Huntington’s disease (HD) has become a target of the first clinical trials for gene therapy among movement disorders with a genetic origin. More than 100 clinical trials regarding HD have been tried, but all failed, although there were some improvements limited to symptomatic support. Compared to other neurogenetic disorders, HD is known to have a single genetic target. Thus, this is an advantage and its cure is more feasible than any other movement disorder with heterogeneous genetic causes. In this review paper, the authors attempt to cover the characteristics of HD itself while providing an overview of the gene transfer methods currently being researched, and will introduce an experimental trial with a preclinical model of HD followed by an update on the ongoing clinical trials for patients with HD.Keywords
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