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用于 siRNA 递送的阳离子树状大分子自组装过程表征的 ITC。

ITC for Characterization of Self-Assembly Process of Cationic Dendrons for siRNA Delivery.

机构信息

Molecular Biology and Nanotechnology Laboratory (MolBNL@UniTs), Department of Engineering and Architecture, University of Trieste, Trieste, Italy.

Biotechnology Development Unit, International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.

出版信息

Methods Mol Biol. 2021;2282:245-266. doi: 10.1007/978-1-0716-1298-9_15.

Abstract

siRNAs are emerging as promising therapeutic agents due to their ability to inhibit specific genes in many diseases. However, these tools require specific vehicles in order to be safely delivered to the targeted site. Among different siRNA delivery systems, self-assembled nanomicelles based on amphiphilic cationic dendrons (ACDs) have recently outperformed nanovectors based on covalent carriers. This chapter describes how isothermal titration calorimetry (ITC) can be exploited as one of the best techniques to investigate the self-assembly process of ACDs. Specifically, ITC can provide, as such or via specific analysis methods, a full thermodynamic characterization of these nanomicelles, including their critical micellar concentration, micelle aggregation number, degree of counterion binding, Gibbs free energy of micellization, and its enthalpic and entropic components.

摘要

siRNAs 作为有前途的治疗剂正在兴起,因为它们能够抑制许多疾病中的特定基因。然而,这些工具需要特定的载体才能安全地递送到靶向部位。在不同的 siRNA 递送系统中,基于两亲性阳离子树状大分子(ACDs)的自组装纳米胶束最近在基于共价载体的纳米载体中表现出色。本章介绍了如何利用等温滴定量热法(ITC)作为研究 ACD 自组装过程的最佳技术之一。具体来说,ITC 可以提供此类或通过特定分析方法,对这些纳米胶束进行全面的热力学特性分析,包括临界胶束浓度、胶束聚集数、抗衡离子结合度、胶束化吉布斯自由能及其焓和熵分量。

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