Current preclinical small interfering RNA (siRNA)-based conjugate systems for RNA therapeutics.

Current preclinical small interfering RNA (siRNA)-based conjugate systems for RNA therapeutics.
Adv Drug Deliv Rev. 2015 Oct 27;
Authors: Lee SH, Kang YY, Jang HE, Mok H
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.
PMID: 26514375 [PubMed - as supplied by publisher]

Source: Advanced Drug Delivery Reviews - October 27, 2015 Category: Drugs & Pharmacology Authors: Lee SH, Kang YY, Jang HE, Mok H Tags: Adv Drug Deliv Rev Source Type: research