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 to this article as recorded by
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 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
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Objective
Huntington’s disease (HD) is a genetic neurodegenerative disease that is caused by abnormal CAG expansion. Altered microRNA (miRNA) expression also causes abnormal gene regulation in this neurodegenerative disease. The delivery of abnormally downregulated miRNAs might restore normal gene regulation and have a therapeutic effect.
Methods
We developed an exosome-based delivery method to treat this neurodegenerative disease. miR-124, one of the key miRNAs that is repressed in HD, was stably overexpressed in a stable cell line. Exosomes were then harvested from these cells using an optimized protocol. The exosomes (Exo-124) exhibited a high level of miR-124 expression and were taken up by recipient cells.
Results
When Exo-124 was injected into the striatum of R6/2 transgenic HD mice, expression of the target gene, RE1-Silencing Transcription Factor, was reduced. However, Exo-124 treatment did not produce significant behavioral improvement.
Conclusion
This study serves as a proof of concept for exosome-based delivery of miRNA in neurodegenerative diseases.
Citations
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