Mendes Bárbara B, Conniot João, Avital Aviram, Yao Dongbao, Jiang Xingya, Zhou Xiang, Sharf-Pauker Noga, Xiao Yuling, Adir Omer, Liang Haojun, Shi Jinjun, Schroeder Avi, Conde João
NOVA Medical School, Faculdade de Ciências Médicas, Universidade Nova de Lisboa, Lisbon, Portugal.
Centre for Toxicogenomics and Human Health, Genetics, Oncology and Human Toxicology, NOVA Medical School, Faculdade de Ciências Médicas, Universidade Nova de Lisboa, Lisbon, Portugal.
Nat Rev Methods Primers. 2022;2. doi: 10.1038/s43586-022-00104-y. Epub 2022 Apr 14.
There is growing need for a safe, efficient, specific and non-pathogenic means for delivery of gene therapy materials. Nanomaterials for nucleic acid delivery offer an unprecedented opportunity to overcome these drawbacks; owing to their tunability with diverse physico-chemical properties, they can readily be functionalized with any type of biomolecules/moieties for selective targeting. Nucleic acid therapeutics such as antisense DNA, mRNA, small interfering RNA (siRNA) or microRNA (miRNA) have been widely explored to modulate DNA or RNA expression Strikingly, gene therapies combined with nanoscale delivery systems have broadened the therapeutic and biomedical applications of these molecules, such as bioanalysis, gene silencing, protein replacement and vaccines. Here, we overview how to design smart nucleic acid delivery methods, which provide functionality and efficacy in the layout of molecular diagnostics and therapeutic systems. It is crucial to outline some of the general design considerations of nucleic acid delivery nanoparticles, their extraordinary properties and the structure-function relationships of these nanomaterials with biological systems and diseased cells and tissues.
对于安全、高效、特异且无致病性的基因治疗材料递送手段的需求日益增长。用于核酸递送的纳米材料为克服这些缺点提供了前所未有的机遇；由于其具有多种物理化学性质的可调控性，它们能够轻易地用任何类型的生物分子/部分进行功能化修饰以实现选择性靶向。诸如反义DNA、信使核糖核酸（mRNA）、小干扰核糖核酸（siRNA）或微小核糖核酸（miRNA）等核酸疗法已被广泛研究以调控DNA或RNA的表达。引人注目的是，与纳米级递送系统相结合的基因疗法拓宽了这些分子在生物分析、基因沉默、蛋白质替代和疫苗等方面的治疗及生物医学应用。在此，我们概述如何设计智能核酸递送方法，这些方法在分子诊断和治疗系统的布局中提供功能性和有效性。概述核酸递送纳米颗粒的一些一般设计考量、它们的非凡特性以及这些纳米材料与生物系统以及患病细胞和组织的结构 - 功能关系至关重要。
