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Engineering Exosomes for CNS Disorders: Advances, Challenges, and Therapeutic Potential
Eun Chae Lee1, Dongsic Choi2, Dong-Hun Lee3
1Department of Medical Sciences, Graduate School, The Catholic University of Korea, Seoul 06591, Republic of Korea.
International Journal of Molecular Sciences
|April 17, 2025
Summary
Exosomes show promise for treating central nervous system (CNS) diseases by delivering therapies across the blood-brain barrier. Challenges in production and standardization must be overcome for clinical use.
Area of Science:
- Neuroscience
- Biotechnology
- Pharmacology
Background:
- Central nervous system (CNS) drug development faces significant hurdles due to the brain's complex structure.
- Exosomes, nanoscale vesicles, offer a novel therapeutic delivery system due to their ability to cross the blood-brain barrier.
Purpose of the Study:
- To review recent advancements in exosome research for CNS diseases.
- To highlight exosomes' potential as carriers for therapeutic molecules like proteins, RNAs, and lipids.
- To discuss challenges and future directions for exosome-based CNS therapies.
Main Methods:
- Literature review of preclinical and clinical studies on exosomes in CNS disorders.
- Analysis of exosome cargo (proteins, RNAs, lipids) and their therapeutic implications.
- Examination of challenges in exosome production, isolation, and clinical translation.
Main Results:
- Exosomes demonstrate potential in delivering therapeutic agents to the CNS.
- Ongoing research elucidates exosome roles in neuroprotection and neurodegeneration.
- Key challenges include scalable manufacturing and standardized isolation protocols.
Conclusions:
- Exosomes represent a promising platform for targeted CNS therapies.
- Overcoming production and standardization challenges is crucial for clinical application.
- Further research into exosome-mediated mechanisms will drive innovative therapeutic strategies for CNS disorders.
