Open AccessGene Therapy for Huntington’s Disease: The Final Strategy for a Cure?
Author(s) -
Seulgi Byun,
Mijung Lee,
Manho Kim
Publication year - 2022
Publication title -
journal of movement disorders
Language(s) - English
Resource type - Journals
eISSN - 2093-4939
pISSN - 2005-940X
DOI - 10.14802/jmd.21006
Subject(s) - medicine , clinical trial , disease , huntington's disease , genetic enhancement , bioinformatics , intensive care medicine , gene , genetics , biology
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.