Aptamer-based targeted drug delivery and disease therapy in preclinical and clinical applications.

Adv Drug Deliv Rev

The Key Laboratory of Zhejiang Province for Aptamers and Theranostics , Zhejiang Cancer Hospital, Hangzhou Institute of Medicine (HIM), Chinese Academy of Sciences, Hangzhou, Zhejiang 310022, China; Institute of Molecular Medicine (IMM), Renji Hospital, Shanghai Jiao Tong University School of Medici

Published: August 2025


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Article Abstract

The advent of precision medicine has created an urgent need for advanced drug-targeting strategies and refined drug delivery systems. Aptamers, characterized by their exceptional affinity and specificity, low molecular weight, negligible immunogenicity, remarkable stability, cost-effectiveness, and structural versatility, are emerging as promising candidates in targeted therapeutics, both in preclinical research and clinical applications. This review provides a comprehensive analysis of the latest advancements in aptamer-based therapeutic strategies, encompassing three key application domains: direct therapeutic agents, targeted ligand engineering, and controlled drug release. We will summarize the preclinical applications of aptamers for various disease therapies, including eye disorders, cancers, coagulation, and inflammation. Particular emphasis is placed on emerging clinical-stage aptamer therapeutics undergoing rigorous evaluation for these diseases. Furthermore, we will discuss the potential challenges and unlimited opportunities for the clinical transformation and commercialization of aptamers.

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http://dx.doi.org/10.1016/j.addr.2025.115680DOI Listing

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