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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
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  • 10 Web of Science
  • 9 Crossref
AbstractAbstract 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.

Citations

Citations to this article as recorded by  
  • The Huntington's Disease Gene Discovery
    Gustavo L. Franklin, Hélio A.G. Teive, Fernando Spina Tensini, Carlos Henrique Ferreira Camargo, Nayra de Souza Carvalho de Lima, Diego de Castro de dos Santos, Alex T. Meira, Sarah J. Tabrizi
    Movement Disorders.2024; 39(2): 227.     CrossRef
  • Optimizing Screening for Intrastriatal Interventions in Huntington's Disease Using Predictive Models
    Matthew J. Barrett, Ahmed Negida, Nitai Mukhopadhyay, Jin K. Kim, Huma Nawaz, Jefin Jose, Claudia Testa
    Movement Disorders.2024;[Epub]     CrossRef
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    Human Gene Therapy.2023;[Epub]     CrossRef
  • CRISPR: a tool with potential for genomic reprogramming in neurological disorders
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    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
  • Bioinspired Approaches for Central Nervous System Targeted Gene Delivery
    Jyotish Kumar, Afroz Karim, Ummy Habiba Sweety, Hemen Sarma, Md Nurunnabi, Mahesh Narayan
    ACS Applied Bio Materials.2023;[Epub]     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

JMD : Journal of Movement Disorders