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Exploring Cordycepin as a Neuroprotective Agent in Huntington's Disease: In Vitro and In Vivo Insights
Chih-Wei Tung1,2, Siew Chin Chan1,2, Pei-Hsun Cheng1
1Department of Physiology, College of Medicine, National Cheng Kung University, Tainan, Taiwan.
Pharmacology Research & Perspectives
|April 9, 2025
Summary
Cordycepin, derived from Cordyceps sinensis, shows promise in protecting neurons and reducing harmful protein aggregates in Huntington's disease (HD) models. Further research is needed, but it may offer supplementary therapeutic benefits for this challenging neurodegenerative disorder.
Area of Science:
- Neuroscience
- Pharmacology
- Genetics
Background:
- Huntington's disease (HD) is a fatal neurodegenerative disorder caused by a mutation in the Huntingtin (HTT) gene.
- Current treatments for HD are limited, highlighting the need for novel therapeutic strategies.
- Cordyceps sinensis and its compound cordycepin have demonstrated neuroprotective properties in other neurodegenerative conditions.
Purpose of the Study:
- To investigate the neuroprotective potential of cordycepin in cellular and animal models of Huntington's disease.
- To evaluate the effects of cordycepin on cell survival, mutant Huntingtin (HTT) aggregation, and neuronal function in HD.
- To assess the impact of cordycepin on disease progression markers, including body weight and protein aggregates, in an HD mouse model.
Main Methods:
- In vitro studies utilizing HD cell models to assess cordycepin's effects on cell viability and HTT aggregation.
- In vivo experiments involving R6/2 HD transgenic mice treated with cordycepin.
- Evaluation of body weight, pathological aggregates, and behavioral changes in treated mice.
Main Results:
- Cordycepin treatment improved cell survival and neuronal formation in HD cell models.
- A slight reduction in mutant HTT aggregates was observed in both in vitro and in vivo models.
- Mice treated with cordycepin showed a modest increase in body weight, but no significant behavioral improvements were noted.
Conclusions:
- Cordycepin exhibits neuroprotective effects and can reduce mutant protein aggregates in HD models.
- While not a standalone cure, cordycepin shows potential as an adjunctive therapy for Huntington's disease.
- Further investigation into the mechanisms underlying cordycepin's effects is warranted.

