| Literature DB >> 26514375 |
Soo Hyeon Lee1, Yoon Young Kang2, Hyo-Eun Jang2, Hyejung Mok3.
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.Entities:
Keywords: Clinical translation; Conjugate; Preclinical study; siRNA; siRNA dose
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Year: 2015 PMID： 26514375 DOI： 10.1016/j.addr.2015.10.009
Source DB: PubMed Journal: Adv Drug Deliv Rev ISSN： 0169-409X Impact factor: 15.470