1. Current preclinical small interfering RNA (siRNA)-based conjugate systems for RNA therapeutics
- Author
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Hyo-Eun Jang, Hyejung Mok, Soo Hyeon Lee, and Yoon Young Kang
- Subjects
0301 basic medicine ,Small interfering RNA ,Polymers ,business.industry ,Aptamer ,Nanostructured materials ,Rational design ,Pharmaceutical Science ,RNA ,Computational biology ,Pharmacology ,Lipids ,Nanostructures ,Clinical trial ,03 medical and health sciences ,RNAi Therapeutics ,030104 developmental biology ,Humans ,Medicine ,RNA Interference ,RNA, Small Interfering ,Peptides ,business ,Conjugate - Abstract
Recent promising clinical results of RNA therapeutics have drawn big attention of academia and industries to RNA therapeutics and their carrier systems. To improve their feasibility in clinics, systemic evaluations of currently available carrier systems under clinical trials and preclinical studies are needed. In this review, we focus on recent noticeable preclinical studies and clinical results regarding siRNA-based conjugates for clinical translations. Advantages and drawbacks of siRNA-based conjugates are discussed, compared to particle-based delivery systems. Then, representative siRNA-based conjugates with aptamers, peptides, carbohydrates, lipids, polymers, and nanostructured materials are introduced. To improve feasibility of siRNA conjugates in preclinical studies, several considerations for the rational design of siRNA conjugates in terms of cleavability, immune responses, multivalent conjugations, and mechanism of action are also presented. Lastly, we discuss lessons from previous preclinical and clinical studies related to siRNA conjugates and perspectives of their clinical applications.
- Published
- 2016
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