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基于脂质体的原位基因递送方法用于将视网膜和大脑皮层作为免疫赦免组织。

Niosome-Based Approach for In Situ Gene Delivery to Retina and Brain Cortex as Immune-Privileged Tissues.

作者信息

Al Qtaish Nuseibah, Gallego Idoia, Villate-Beitia Ilia, Sainz-Ramos Myriam, López-Méndez Tania Belén, Grijalvo Santiago, Eritja Ramón, Soto-Sánchez Cristina, Martínez-Navarrete Gema, Fernández Eduardo, Puras Gustavo, Pedraz José Luis

机构信息

NanoBioCel group, University of the Basque Country (UPV/EHU), E-01006 Vitoria-Gasteiz, Spain.

Networking Research Centre of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), E-01006 Vitoria-Gasteiz, Spain.

出版信息

Pharmaceutics. 2020 Feb 25;12(3):198. doi: 10.3390/pharmaceutics12030198.

Abstract

Non-viral vectors have emerged as a promising alternative to viral gene delivery systems due to their safer profile. Among non-viral vectors, recently, niosomes have shown favorable properties for gene delivery, including low toxicity, high stability, and easy production. The three main components of niosome formulations include a cationic lipid that is responsible for the electrostatic interactions with the negatively charged genetic material, a non-ionic surfactant that enhances the long-term stability of the niosome, and a helper component that can be added to improve its physicochemical properties and biological performance. This review is aimed at providing recent information about niosome-based non-viral vectors for gene delivery purposes. Specially, we will discuss the composition, preparation methods, physicochemical properties, and biological evaluation of niosomes and corresponding nioplexes that result from the addition of the genetic material onto their cationic surface. Next, we will focus on the in situ application of such niosomes to deliver the genetic material into immune-privileged tissues such as the brain cortex and the retina. Finally, as future perspectives, non-invasive administration routes and different targeting strategies will be discussed.

摘要

由于非病毒载体具有更安全的特性,它们已成为病毒基因递送系统的一种有前景的替代方案。在非病毒载体中,近年来,脂质体已显示出在基因递送方面的良好特性,包括低毒性、高稳定性和易于生产。脂质体制剂的三个主要成分包括负责与带负电荷的遗传物质进行静电相互作用的阳离子脂质、增强脂质体长期稳定性的非离子表面活性剂以及可添加以改善其物理化学性质和生物学性能的辅助成分。本综述旨在提供有关用于基因递送目的的基于脂质体的非病毒载体的最新信息。特别地,我们将讨论脂质体以及通过将遗传物质添加到其阳离子表面而形成的相应脂质复合物的组成、制备方法、物理化学性质和生物学评价。接下来,我们将重点关注此类脂质体在将遗传物质递送至免疫特权组织(如大脑皮层和视网膜)中的原位应用。最后,作为未来展望,将讨论非侵入性给药途径和不同的靶向策略。

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