期刊
TRENDS IN BIOTECHNOLOGY
卷 36, 期 5, 页码 562-575出版社
ELSEVIER SCIENCE LONDON
DOI: 10.1016/j.tibtech.2018.01.006
关键词
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资金
- NHMRC-ARC Dementia Fellowship [APP1111611]
- National Natural Science Foundation of China [NSFC 31640027, 31600809, U1604177]
- Program of China 1000-Talents Plan
Small interfering RNA (siRNA)-based gene silencing technology has demonstrated significant potential for treating brain-associated diseases. However, effective and safe systemic delivery of siRNA into the brain remains challenging because of biological barriers such as enzymatic degradation, short circulation lifetime, the blood-brain barrier (BBB), insufficient tissue penetration, cell endocytosis, and cytosolic transport. Nanotechnology offers intriguing potential for addressing these challenges in siRNA brain delivery in conjunction with chemical and biological modification strategies. In this review, we outline the challenges of systemic delivery of siRNA-based therapy for brain diseases, highlight recent advances in the development and engineering of siRNA nano-medicines for various brain diseases, and discuss our perspectives on this exciting research field for siRNA-based therapy towards more effective brain disease therapy.
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