AI Article Synopsis
- Huntington's disease (HD) is a genetic condition linked to abnormal CAG expansions and altered microRNA (miRNA) levels, which disrupt normal gene regulation.
- Researchers developed an exosome-based delivery method to administer miR-124, a crucial miRNA that is downregulated in HD, aiming to restore gene function.
- In experiments with R6/2 transgenic HD mice, injecting the exosomes (Exo-124) reduced the expression of a target gene, but there were no notable improvements in behavior, indicating more research is needed.
Article 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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Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5288667 | PMC |
| http://dx.doi.org/10.14802/jmd.16054 | DOI Listing |
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