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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
- 9,042 View
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- 17 Web of Science
- 15 Crossref
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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
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ACS Applied Bio Materials.2024; 7(8): 4975. CrossRef - 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; 39(5): 855. CrossRef - Exosomes for neurodegenerative diseases: diagnosis and targeted therapy
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Journal of Neurology.2024; 271(6): 3050. CrossRef - Emerging Perspectives on Prime Editor Delivery to the Brain
Eli BenDavid, Sina Ramezanian, Yaoyao Lu, Joël Rousseau, Avi Schroeder, Marc Lavertu, Jacques P. Tremblay
Pharmaceuticals.2024; 17(6): 763. CrossRef - Exploring molecular mechanisms, therapeutic strategies, and clinical manifestations of Huntington’s disease
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Livia Zhou, Yafeng Wang, Yiran Xu, Yaodong Zhang, Changlian Zhu
Journal of Neuroinflammation.2024;[Epub] CrossRef - Efficacy and Safety of Tetrabenazine in Reducing Chorea and Improving Motor Function in Individuals With Huntington's Disease: A Systematic Review
Nandini Vadlamani, Sabina Ibrahimli, Farees Ahmad Khan, Jason A Castillo, Kavya Sai Satya Amaravadi, Poornachandra Nalisetty, Safeera Khan
Cureus.2024;[Epub] CrossRef - Neurofilament light chain: a biomarker at the crossroads of clarity and confusion for gene-directed therapies
Michael Christian A Virata, Jesus Alfonso Catahay, Giuseppe Lippi, Brandon M Henry
Neurodegenerative Disease Management.2024; : 1. 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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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
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Pharmaceuticals.2023; 16(11): 1513. CrossRef - Mitochondrial organization and structure are compromised in fibroblasts from patients with Huntington’s disease
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Ultrastructural Pathology.2022; 46(5): 462. CrossRef - Pathogenesis of Huntington’s Disease: An Emphasis on Molecular Pathways and Prevention by Natural Remedies
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Brain Sciences.2022; 12(10): 1389. CrossRef
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