Chinese Journal of Tissue Engineering Research ›› 2025, Vol. 29 ›› Issue (23): 5013-5021.doi: 10.12307/2025.084

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Hot topics on exosomes as drug delivery system in central nervous system diseases

Lin Huijie1, Huang Yun1, Huang Zhihua2, 3, Jiang Lixia2, 4   

  1. 1First Clinical Medical School of Gannan Medical University, Ganzhou 341000, Jiangxi Province, China; 2Key Laboratory of Ministry of Education for Prevention and Treatment of Cardiovascular and Cerebrovascular Diseases, Gannan Medical University, Ganzhou 341000, Jiangxi Province, China; 3School of Basic Medicine, Gannan Medical University, Ganzhou 341000, Jiangxi Province, China; 4Clinical Laboratory of First Affiliated Hospital, Gannan Medical University, Ganzhou 341000, Jiangxi Province, China
  • Received:2023-12-19 Accepted:2024-05-17 Online:2025-08-18 Published:2024-09-30
  • Contact: Jiang Lixia, Master’s supervisor, Professor, Key Laboratory of Ministry of Education for Prevention and Treatment of Cardiovascular and Cerebrovascular Diseases, Gannan Medical University, Ganzhou 341000, Jiangxi Province, China; Clinical Laboratory of First Affiliated Hospital, Gannan Medical University, Ganzhou 341000, Jiangxi Province, China
  • About author:Lin Huijie, Master candidate, First Clinical Medical School of Gannan Medical University, Ganzhou 341000, Jiangxi Province, China
  • Supported by:
    Open Project of Key Laboratory of Ministry of Education for Prevention and Treatment of Cardiovascular and Cerebrovascular Diseases of Gannan Medical University, No. XN201921 (to JLX); Jiangxi Provincial Health Commission Project, No. 202130621 (to JLX); Graduate Innovation Special Fund Project, No. YC2023-S939 (to LHJ); Graduate Innovation Special Fund Project, No. YC2022-S964 (to HY)

Abstract: BACKGROUND: The use of exosomes as drug carriers can not only precisely target the therapeutic site, but also increase the local concentration, opening up a new way for drugs to enter the central nervous system.
OBJECTIVE: To explore the biogenesis and biological functions of exosomes and summarize the current state-of-the-art regarding extracellular vesicles as drug carriers in the treatment of central nervous system diseases.
METHODS: The first author searched Web of Science, PubMed, and CNKI for relevant literature from January 1976 to January 2024. The English search terms were “exosomes, extracellular vesicles, central nervous system, drug delivery, ischemic stroke, Alzheimer’s disease, Parkinson’s disease, spinal cord injury, brain tumor.” The Chinese search terms were “exosomes, extracellular vesicles, central nervous system diseases, drug delivery, stroke, Alzheimer’s disease, Parkinson’s disease, spinal cord injury, brain tumor.” Finally, 94 articles were included for analysis. 
RESULTS AND CONCLUSION: (1) Exosomes can easily cross the blood-brain barrier and deliver proteins, metabolites, and nucleic acids to recipient cells to regulate cellular metabolism. Since exosomes are small vesicles secreted by cells, they have a much lower circulating immunogenicity and can be less likely to be recognized and cleared by macrophages in the internal circulation. (2) Exosomes can be engineered to deliver different therapeutically ingredients, including RNA, proteins, chemotherapeutic drugs, and immunomodulators, and are capable of delivering them to the desired target areas. Engineered modified exosomes have better targeting properties. Furthermore, this exosome-mediated delivery is extremely low in immunogenicity and is expected to provide a safer and more effective method for precision therapy of central nervous system diseases in the future.

Key words: ">central nervous system disease, exosomes, drug delivery, stroke, Alzheimer’s disease, Parkinson’s disease, spinal cord injury, brain tumor

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