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更好药物的蓝图:纳米医学的结构革命

Blueprints for Better Drugs: The Structural Revolution in Nanomedicine.

作者信息

Mirkin Chad A, Langer Robert, Mrksich Milan, Margolin Adam A, Petrosko Sarah Hurst, Artzi Natalie

机构信息

Department of Chemistry, Northwestern University, Evanston, Illinois 60208, United States.

International Institute for Nanotechnology, Northwestern University, Evanston, Illinois 60208, United States.

出版信息

ACS Nano. 2025 May 27;19(20):18889-18901. doi: 10.1021/acsnano.5c06380. Epub 2025 May 13.

Abstract

Structural nanomedicines are engineered constructs that arrange therapeutic components into well-defined architectures to maximize efficacy. Their multivalent, multifunctional design offers key advantages over unstructured formulations, including targeted delivery, expanded therapeutic windows, and enhanced target engagement. The mRNA COVID-19 vaccines exemplify their transformative potential. However, structural precision varies, and more well-defined architectures will streamline optimization, manufacturing, and regulation. Unlike small molecule drugs, nanomedicines within a batch are not identical. Identifying the most effective, least toxic structures will advance our understanding of structure-function relationships and therapeutic mechanisms. This work highlights structural nanomedicines─small molecules, nucleic acids, and biologics─to galvanize the field and drive innovation toward even safer, more effective treatments that benefit patients.

摘要

结构性纳米药物是经过工程设计的构建体,它们将治疗成分排列成明确的架构以实现疗效最大化。其多价、多功能设计相较于非结构化制剂具有关键优势,包括靶向递送、扩大治疗窗口以及增强靶点结合。mRNA新冠疫苗就是其变革潜力的例证。然而,结构精度各不相同,更明确的架构将简化优化、制造和监管流程。与小分子药物不同,一批纳米药物中的分子并非完全相同。识别最有效、毒性最小的结构将增进我们对结构-功能关系及治疗机制的理解。这项工作重点介绍了结构性纳米药物——小分子、核酸和生物制剂——以激励该领域,并推动创新,朝着为患者带来更安全、更有效治疗的方向发展。

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