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Theranostics 2014; 4(12):1211-1232. doi:10.7150/thno.8491 This issue Cite

Review

Functional Nanostructures for Effective Delivery of Small Interfering RNA Therapeutics

Cheol Am Hong¹, Yoon Sung Nam^1,2,3✉

1. Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST), 291 Daehak-ro, Yuseong-gu, Daejeon, 305-701, Republic of Korea;
2. Department of Materials Science and Engineering, Korea Advanced Institute of Science and Technology (KAIST), 291 Daehak-ro, Yuseong-gu, Daejeon, 305-701, Republic of Korea;
3. KAIST Institute for NanoCentury and BioCentury, Korea Advanced Institute of Science and Technology (KAIST), 291 Daehak-ro, Yuseong-gu, Daejeon, 305-701, Republic of Korea.

✉ Corresponding author: Yoon Sung Nam, E-mail: yoonsungac.kr; Tel.: +82-42-350-3311; Fax: +82-42-350-3310.

Abstract

Small interfering RNA (siRNA) has proved to be a powerful tool for target-specific gene silencing via RNA interference (RNAi). Its ability to control targeted gene expression gives new hope to gene therapy as a treatment for cancers and genetic diseases. However, siRNA shows poor pharmacological properties, such as low serum stability, off-targeting, and innate immune responses, which present a significant challenge for clinical applications. In addition, siRNA cannot cross the cell membrane for RNAi activity because of its anionic property and stiff structure. Therefore, the development of a safe, stable, and efficient system for the delivery of siRNA therapeutics into the cytoplasm of targeted cells is crucial. Several nanoparticle platforms for siRNA delivery have been developed to overcome the major hurdles facing the therapeutic uses of siRNA. This review covers a broad spectrum of non-viral siRNA delivery systems developed for enhanced cellular uptake and targeted gene silencing in vitro and in vivo and discusses their characteristics and opportunities for clinical applications of therapeutic siRNA.

Keywords: gene delivery, gene silencing, nanoparticles, non-viral vectors, small interfering RNA (siRNA).

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