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Review Article
- Gene Therapy for Huntington’s Disease: The Final Strategy for a Cure?
- Seulgi Byun, Mijung Lee, Manho Kim
- J Mov Disord. 2022;15(1):15-20. Published online November 17, 2021
- DOI: https://doi.org/10.14802/jmd.21006
- 6,155 View
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- 6 Citations
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Abstract
PDF - 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.
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Citations
Citations to this article as recorded by
- Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid
Jonathan B. Rosenberg, Edward K. Fung, Jonathan P. Dyke, Bishnu P. De, Howard Lou, James M. Kelly, Layla Reejhsinghani, Rodolfo J. Ricart Arbona, Dolan Sondhi, Stephen M. Kaminsky, Nathalie Cartier, Christian Hinderer, Juliette Hordeaux, James M. Wilson,
Human Gene Therapy.2023;[Epub] CrossRef - CRISPR: a tool with potential for genomic reprogramming in neurological disorders
Yogesh K. Dhuriya, Aijaz A. Naik
Molecular Biology Reports.2023; 50(2): 1845. CrossRef - Gene therapy for selected neuromuscular and trinucleotide repeat disorders – An insight to subsume South Asia for multicenter clinical trials
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IBRO Neuroscience Reports.2023; 14: 146. CrossRef - Huntington’s Disease Drug Development: A Phase 3 Pipeline Analysis
Hannah J. Van de Roovaart, Nguyen Nguyen, Timothy D. Veenstra
Pharmaceuticals.2023; 16(11): 1513. CrossRef - Mitochondrial organization and structure are compromised in fibroblasts from patients with Huntington’s disease
Marie Vanisova, Hana Stufkova, Michaela Kohoutova, Tereza Rakosnikova, Jana Krizova, Jiri Klempir, Irena Rysankova, Jan Roth, Jiri Zeman, Hana Hansikova
Ultrastructural Pathology.2022; 46(5): 462. CrossRef - Pathogenesis of Huntington’s Disease: An Emphasis on Molecular Pathways and Prevention by Natural Remedies
Zainab Irfan, Sofia Khanam, Varnita Karmakar, Sayeed Mohammed Firdous, Bothaina Samih Ismail Abou El Khier, Ilyas Khan, Muneeb U. Rehman, Andleeb Khan
Brain Sciences.2022; 12(10): 1389. CrossRef
- Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid
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