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细胞器靶向纳米颗粒

Organelle-Targeting Nanoparticles.

作者信息

Soukar John, Peppas Nicholas A, Gaharwar Akhilesh K

机构信息

Interdisiplinary program in Genetics and Genomics, Texas A&M University, College Station, TX, 77843, USA.

Department of Biomedical Engineering, College of Engineering, Texas A&M University, College Station, TX, 77843, USA.

出版信息

Adv Sci (Weinh). 2025 Feb;12(7):e2411720. doi: 10.1002/advs.202411720. Epub 2025 Jan 13.

Abstract

Organelles are specialized subunits within cells which carry out vital functions crucial to cellular survival and form a tightly regulated network. Dysfunctions in any of these organelles are linked to numerous diseases impacting virtually every organ system in the human body. Targeted delivery of therapeutics to specific organelles within the cell holds great promise for overcoming challenging diseases and improving treatment outcomes through the minimization of therapeutic dosage and off-target effects. Nanoparticles are versatile and effective tools for therapeutic delivery to specific organelles. Nanoparticles offer several advantageous characteristics, including a high surface area-to-volume ratio for efficient therapeutic loading and the ability to attach targeting moieties (tethers) that enhance delivery. The choice of nanoparticle shape, size, composition, surface properties, and targeting ligands depends on the desired target organelle and therapeutic effect. Various nanoparticle platforms have been explored for organelle targeting, such as liposomes, polymeric nanoparticles, dendrimers, and inorganic nanoparticles. In this review, current and emerging approaches to nanoparticle design are examined in the context of various diseases linked to organelle dysfunction. Specifically, advances in nanoparticle therapies targeting organelles such as the nucleus, mitochondria, lysosomes/endosomes, Golgi apparatus, and endoplasmic reticulum are comprehensively and critically discussed.

摘要

细胞器是细胞内的特殊亚单位,执行对细胞存活至关重要的重要功能,并形成一个严格调控的网络。这些细胞器中任何一个的功能障碍都与许多影响人体几乎每个器官系统的疾病有关。将治疗药物靶向递送至细胞内特定的细胞器,对于克服具有挑战性的疾病并通过最小化治疗剂量和脱靶效应来改善治疗效果具有巨大潜力。纳米颗粒是用于向特定细胞器进行治疗递送的通用且有效的工具。纳米颗粒具有几个有利特性,包括用于高效治疗性负载的高表面积与体积比以及连接可增强递送的靶向部分(连接体)的能力。纳米颗粒的形状、大小、组成、表面性质和靶向配体的选择取决于所需的靶细胞器和治疗效果。已经探索了各种纳米颗粒平台用于细胞器靶向,例如脂质体、聚合物纳米颗粒、树枝状大分子和无机纳米颗粒。在这篇综述中,在与细胞器功能障碍相关的各种疾病背景下,研究了纳米颗粒设计的当前和新兴方法。具体而言,全面且批判性地讨论了靶向细胞核、线粒体、溶酶体/内体、高尔基体和内质网等细胞器的纳米颗粒疗法的进展。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6d21/11831507/e2d2bab89a94/ADVS-12-2411720-g005.jpg

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