J Mov Disord.
2017 Jan;10(1):45-52. 10.14802/jmd.16054.
Exosome-Based Delivery of miR-124 in a Huntington's Disease Model
- Affiliations
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- 1Department of Neurology, Neuroscience Research Center, Seoul National University Hospital, Seoul, Korea. kimmanho@snu.ac.kr stemcell.snu@gmail.com
- 2Program in Neuroscience, Neuroscience Research Institute of SNUMRC, Seoul National University, Seoul, Korea.
- 3Neuroscience Research Institute, Seoul National University College of Medicine, Seoul, Korea.
- 4Protein Metabolism Medical Research Center, Seoul National University College of Medicine, Seoul, Korea.
- KMID: 2367608
- DOI: http://doi.org/10.14802/jmd.16054
Abstract
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.
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