Cargando…

Gene Therapy for Huntington’s Disease: The Final Strategy for a Cure?

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 ot...

Descripción completa

Detalles Bibliográficos
Autores principales: Byun, Seulgi, Lee, Mijung, Kim, Manho
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Korean Movement Disorder Society 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8820879/
https://www.ncbi.nlm.nih.gov/pubmed/34781633
http://dx.doi.org/10.14802/jmd.21006
Descripción
Sumario: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.