- Gene Therapy for Huntington’s Disease: The Final Strategy for a Cure?
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Seulgi Byun, Mijung Lee, Manho Kim
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J Mov Disord. 2022;15(1):15-20. Published online November 17, 2021
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DOI: https://doi.org/10.14802/jmd.21006
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 Abstract
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- 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  - Translational Approach using Advanced Therapy Medicinal Products for Huntington's Disease
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Current Reviews in Clinical and Experimental Pharmacology.2025; 20(1): 14. CrossRef - Exploring the role of ferroptosis pathways in Huntington's disease: Insight of pathophysiology to emerging treatment
Mohd Haris Jamal, Meghna Dhupar, Khadga Raj Aran
Brain Disorders.2025; 18: 100207. CrossRef - Oligonucleotide-based therapeutics for neurodegenerative disorders: Focus on antisense oligonucleotides
Heba M. Mansour, Aiman S. El-Khatib
European Journal of Pharmacology.2025; 998: 177529. 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.2024; 7(8): 4975. CrossRef - The Huntington's Disease Gene Discovery
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Journal of Neurology.2024; 271(6): 3050. CrossRef - Emerging Perspectives on Prime Editor Delivery to the Brain
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Pharmaceuticals.2024; 17(6): 763. CrossRef - Exploring molecular mechanisms, therapeutic strategies, and clinical manifestations of Huntington’s disease
Alaa Shafie, Amal Adnan Ashour, Saleha Anwar, Farah Anjum, Md. Imtaiyaz Hassan
Archives of Pharmacal Research.2024; 47(6): 571. CrossRef - A comprehensive review of AAV-mediated strategies targeting microglia for therapeutic intervention of neurodegenerative diseases
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; 14(6): 227. 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
Nalaka Wijekoon, Lakmal Gonawala, Pyara Ratnayake, Darshana Sirisena, Harsha Gunasekara, Athula Dissanayake, Sunethra Senanayake, Ajantha Keshavaraj, Yetrib Hathout, Harry W.M. Steinbusch, Chandra Mohan, Ashwin Dalal, Eric Hoffman, K.Ranil D de Silva
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
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