Nanotechnology-Based Strategies for siRNA Brain Delivery for Disease Therapy

Meng Zheng, Wei Tao, Yan Zou, Omid C. Farokhzad, Bingyang Shi*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

112 Citations (Scopus)


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 nanomedicines for various brain diseases, and discuss our perspectives on this exciting research field for siRNA-based therapy towards more effective brain disease therapy.

Original languageEnglish
Pages (from-to)562-575
Number of pages14
JournalTrends in Biotechnology
Issue number5
Publication statusPublished - 1 May 2018
Externally publishedYes


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