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Updated: Jan 26, 2026

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System
Published on: May 12, 2013
Non-viral nucleic acid delivery to the central nervous system and brain tumors
Shanshan Wang1, Rongqin Huang1
1Department of Pharmacy, Zhongshan Hospital, and School of Pharmacy, Fudan University, Shanghai, China.
Abstract:
Gene therapy is a rapidly emerging remedial route for many serious incurable diseases, such as central nervous system (CNS) diseases. Currently, nucleic acid medicines, including DNAs encoding therapeutic or destructive proteins, small interfering RNAs or microRNAs, have been successfully delivered to the CNS with gene delivery vectors using various routes of administration and have subsequently exhibited remarkable therapeutic efficiency. Among these vectors, non-viral vectors are favorable for delivering genes into the CNS as a result of their many special characteristics, such as low toxicity and pre-existing immunogenicity, high gene loading efficiency and easy surface modification. In this review, we highlight the main types of therapeutic genes that have been applied in the therapy of CNS diseases and then outline non-viral gene delivery vectors.
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