J Mov Disord.
2022 Jan;15(1):15-20. 10.14802/jmd.21006.
Gene Therapy for Huntington’s Disease: The Final Strategy for a Cure?
- Affiliations
-
- 1Department of Neurology, Seoul National University Hospital, Seoul, Korea
- 2Neurobiology Section, Division of Biological Science, University of California San Diego, La Jolla, CA, USA
- 3Protein Metabolism and Dementia & Neuroscience Center, Seoul National University College of Medicine, Seoul, Korea
- KMID: 2525084
- DOI: http://doi.org/10.14802/jmd.21006
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.
