Programa de Pós-Graduação em Ciências Farmacêuticas da Universidade Federal do Rio Grande do Sul (UFRGS), Faculdade de Farmácia, Av. Ipiranga 2752, 90610-000, Porto Alegre, RS, Brazil.
Programa de Pós-Graduação em Ciências Farmacêuticas da Universidade Federal do Rio Grande do Sul (UFRGS), Faculdade de Farmácia, Av. Ipiranga 2752, 90610-000, Porto Alegre, RS, Brazil; Programa de Pós-Graduação em Ciências Farmacêuticas da Universidade Federal do Pampa (UNIPAMPA), Faculdade de Farmácia, Estrada BR 472 Km7, Prédio 700, 97500970, Uruguaiana, RS, Brazil.
Int J Pharm. 2017 Dec 20;534(1-2):356-367. doi: 10.1016/j.ijpharm.2017.10.030. Epub 2017 Oct 13.
Since the first clinical studies, knowledge in the field of gene therapy has advanced significantly, and these advances led to the development and subsequent approval of the first gene medicines. Although viral vectors-based products offer efficient gene expression, problems related to their safety and immune response have limited their clinical use. Thus, design and optimization of nonviral vectors is presented as a promising strategy in this scenario. Nonviral systems are nanotechnology-based products composed of polymers or lipids, which are usually biodegradable and biocompatible. Cationic liposomes are the most studied nonviral carriers and knowledge about these systems has greatly evolved, especially in understanding the role of phospholipids and cationic lipids. However, the search for efficient delivery systems aiming at gene therapy remains a challenge. In this context, cationic nanoemulsions have proved to be an interesting approach, as their ability to protect and efficiently deliver nucleic acids for diverse therapeutic applications has been demonstrated. This review focused on cationic nanoemulsions designed for gene therapy, providing an overview on their composition, physicochemical properties, and their efficacy on biological response in vitro and in vivo.
自首次临床研究以来,基因治疗领域的知识取得了显著进展,这些进展推动了第一批基因药物的开发和随后的批准。虽然基于病毒载体的产品提供了高效的基因表达,但与它们的安全性和免疫反应相关的问题限制了它们的临床应用。因此,非病毒载体的设计和优化在这种情况下呈现出一种有前途的策略。非病毒系统是基于纳米技术的聚合物或脂质产品,通常是可生物降解和生物相容的。阳离子脂质体是非病毒载体中研究最多的一种,人们对这些系统的了解已经有了很大的发展,特别是在理解磷脂和阳离子脂质的作用方面。然而,寻找旨在实现基因治疗的高效递药系统仍然是一个挑战。在这方面,阳离子纳米乳剂已被证明是一种很有前途的方法,因为它们具有保护和有效递送核酸用于多种治疗应用的能力。本文综述了用于基因治疗的阳离子纳米乳剂,概述了它们的组成、理化性质以及它们在体外和体内对生物反应的功效。