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siRNA therapeutics: a clinical reality

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  • Special Topic: Noncoding RNA: from dark matter to bright star
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Abstract

Since the revolutionary discovery of RNA interference (RNAi), a remarkable progress has been achieved in understanding and harnessing gene silencing mechanism; especially in small interfering RNA (siRNA) therapeutics. Despite its tremendous potential benefits, major challenges in most siRNA therapeutics remains unchanged—safe, efficient and target oriented delivery of siRNA. Twenty years after the discovery of RNAi, siRNA therapeutics finally charts its way into clinics. As we journey through the decades, we reminisce the history of siRNA discovery and its application in a myriad of disease treatments. Herein, we highlight the breakthroughs in siRNA therapeutics, with special feature on the first FDA approved RNAi therapeutics Onpattro (Patisiran) and the consideration of effective siRNA delivery system focusing on current siRNA nanocarrier in clinical trials. Lastly, we present some challenges and multiple barriers that are yet to be fully overcome in siRNA therapeutics.

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Acknowledgements

This work was supported by the National Key Research and Development Program of China (2016YFC1302300), the National Natural Science Foundation of China (81720108029, 81621004, and 81490750), Guangdong Science and Technology Department (2016B030229004), Guangzhou Science Technology and Innovation Commission (201803040015), and the Fountain-Valley Life Sciences Fund of University of Chinese Academy of Sciences Education Foundation.

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  1. Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, 510120, China

    Phei Er Saw & Er-Wei Song

  2. Breast Tumor Center, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, 510120, China

    Er-Wei Song

  3. Zhongshan School of Medicine, Breast Surgery, Guangzhou, 510080, China

    Er-Wei Song

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  1. Phei Er Saw
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Saw, P.E., Song, EW. siRNA therapeutics: a clinical reality. Sci. China Life Sci. 63, 485–500 (2020). https://doi.org/10.1007/s11427-018-9438-y

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