Univ Coimbra, Faculty of Pharmacy, Pólo das Ciências da Saúde, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Univ Coimbra, LAQV, REQUIMTE, Faculty of Pharmacy, University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Center for Neuroscience and Cell Biology (CNC), University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (IIIUC), University of Coimbra, Coimbra, Portugal.
Int J Pharm. 2021 Mar 15;597:120362. doi: 10.1016/j.ijpharm.2021.120362. Epub 2021 Feb 5.
The decoding of the human genome revolutionized the understanding of how genetics influence the interplay between health and disease, in a multidisciplinary perspective. Thus, the development of exogenous nucleic acids-based therapies has increased to overcome hereditary or acquired genetic-associated diseases. Gene drug delivery using non-viral systems, for instance micelleplexes, have been recognized as promising options for gene-target therapies. Micelleplexes are core-shell structures, at a nanometric scale, designed using amphiphilic block copolymers. These can self-assemble in an aqueous medium, leading to the formation of a hydrophilic and positively charged corona - that can transport nucleic acids, - and a hydrophobic core - which can transport poor water-soluble drugs. However, the performance of these types of carriers usually is hindered by several in vivo barriers. Fortunately, due to a significant amount of research, strategies to overcome these shortcomings emerged. With a wide range of structural features, good stability against proteolytic degradation, affordable characteristic, easy synthesis, low immunogenicity, among other advantages, peptides have increasingly gained popularity as target ligands for non-viral carriers. Hence, this review addresses the use of peptides with micelleplexes illustrating, through the analysis of in vitro and in vivo studies, the potential and future perspectives of this combination.
人类基因组的解码从多学科的角度彻底改变了我们对遗传如何影响健康与疾病之间相互作用的理解。因此，为了克服遗传性或获得性遗传相关疾病，基于外源核酸的治疗方法得到了发展。例如，使用非病毒系统的基因药物传递，已经被认为是基因靶向治疗的有前途的选择。胶束复合物是纳米级的核壳结构，使用两亲嵌段共聚物设计。这些可以在水介质中自组装，形成亲水性和带正电荷的冠——可以携带核酸，——和疏水性核——可以携带水溶性差的药物。然而，这些类型载体的性能通常受到多种体内障碍的阻碍。幸运的是，由于大量的研究，出现了克服这些缺点的策略。由于具有广泛的结构特征、对蛋白水解降解的稳定性、负担得起的特性、易于合成、低免疫原性等优点，肽类作为非病毒载体的靶向配体越来越受到关注。因此，本文综述了肽与胶束复合物的联合使用，通过对体外和体内研究的分析，说明了这种联合的潜力和未来前景。
