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用于心脏归巢的模块化生物正交脂质纳米颗粒修饰平台。

Modular Bioorthogonal Lipid Nanoparticle Modification Platforms for Cardiac Homing.

机构信息

School of Cellular and Molecular Medicine, University of Bristol, Biomedical Sciences Building, University Walk, Bristol BS8 1TD, U.K.

School of Physiology, Pharmacology and Neuroscience, University of Bristol, Biomedical Sciences Building, University Walk, Bristol BS8 1TD, U.K.

出版信息

J Am Chem Soc. 2023 Oct 18;145(41):22659-22670. doi: 10.1021/jacs.3c07811. Epub 2023 Oct 9.

Abstract

Lipid nanoparticles (LNPs) are becoming widely adopted as vectors for the delivery of therapeutic payloads but generally lack intrinsic tissue-homing properties. These extracellular vesicle (EV) mimetics can be targeted toward the liver, lung, or spleen via charge modification of their lipid headgroups. Homing to other tissues has only been achieved via covalent surface modification strategies using small-molecule ligands, peptides, or monoclonal antibodies─methods that are challenging to couple with large-scale manufacturing. Herein, we design a novel modular artificial membrane-binding protein (AMBP) platform for the modification of LNPs postformation. The system is composed of two protein modules that can be readily coupled using bioorthogonal chemistry to yield the AMBP. The first is a membrane anchor module comprising a supercharged green fluorescent protein (scGFP) electrostatically conjugated to a dynamic polymer surfactant corona. The second is a functional module containing a cardiac tissue fibronectin homing sequence from the bacterial adhesin CshA. We demonstrate that LNPs modified using the AMBP exhibit a 20-fold increase in uptake by fibronectin-rich C2C12 cells under static conditions and a 10-fold increase under physiologically relevant shear stresses, with no loss of cell viability. Moreover, we show targeted localization of the AMBP-modified LNPs in zebrafish hearts, highlighting their therapeutic potential as a vector for the treatment of cardiac disease and, more generally, as a smart vector.

摘要

脂质纳米颗粒(LNPs)作为治疗有效载荷的载体已被广泛采用,但通常缺乏内在的组织归巢特性。通过改变其脂质头部的电荷,这些细胞外囊泡(EV)模拟物可以靶向肝脏、肺或脾脏。其他组织的归巢仅通过使用小分子配体、肽或单克隆抗体的共价表面修饰策略来实现,这些方法与大规模制造相结合具有挑战性。在此,我们设计了一种用于 LNPs 形成后的修饰的新型模块化人工膜结合蛋白(AMBP)平台。该系统由两个蛋白质模块组成,可通过生物正交化学轻松偶联,生成 AMBP。第一个是膜锚定模块,由超荷电绿色荧光蛋白(scGFP)通过静电结合到动态聚合物表面活性剂冠上组成。第二个是功能模块,包含来自细菌黏附素 CshA 的心脏组织纤维连接蛋白同源序列。我们证明,在用 AMBP 修饰的 LNPs 下,在静态条件下富含纤维连接蛋白的 C2C12 细胞中的摄取增加了 20 倍,在生理相关的切应力下增加了 10 倍,而细胞活力没有损失。此外,我们还在斑马鱼心脏中显示了 AMBP 修饰的 LNPs 的靶向定位,突出了它们作为心脏疾病治疗载体的治疗潜力,更普遍地说,作为一种智能载体。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3ee0/10591475/d0ca56ba0d7a/ja3c07811_0001.jpg

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