Marson Domenico, Laurini Erik, Aulic Suzana, Fermeglia Maurizio, Pricl Sabrina
Molecular Biology and Nanotechnology Laboratory (MolBNL@UniTS), Department of Engineering and Architecture, University of Trieste, 34127 Trieste, Italy.
Pharmaceutics. 2019 Jul 18;11(7):351. doi: 10.3390/pharmaceutics11070351.
Small interfering RNAs (siRNAs) represent a new approach towards the inhibition of gene expression; as such, they have rapidly emerged as promising therapeutics for a plethora of important human pathologies including cancer, cardiovascular diseases, and other disorders of a genetic etiology. However, the clinical translation of RNA interference (RNAi) requires safe and efficient vectors for siRNA delivery into cells. Dendrimers are attractive nanovectors to serve this purpose, as they present a unique, well-defined architecture and exhibit cooperative and multivalent effects at the nanoscale. This short review presents a brief introduction to RNAi-based therapeutics, the advantages offered by dendrimers as siRNA nanocarriers, and the remarkable results we achieved with bio-inspired, structurally flexible covalent dendrimers. In the companion paper, we next report our recent efforts in designing, characterizing and testing a series of self-assembled amphiphilic dendrimers and their related structural alterations to achieve unprecedented efficient siRNA delivery both in vitro and in vivo.
小干扰RNA（siRNA）代表了一种抑制基因表达的新方法；因此，它们迅速成为治疗多种重要人类疾病（包括癌症、心血管疾病和其他遗传病因引起的疾病）的有前景的疗法。然而，RNA干扰（RNAi）的临床转化需要安全有效的载体将siRNA递送至细胞。树枝状大分子是用于此目的的有吸引力的纳米载体，因为它们具有独特的、明确的结构，并在纳米尺度上表现出协同和多价效应。这篇简短的综述简要介绍了基于RNAi的疗法、树枝状大分子作为siRNA纳米载体的优势，以及我们用受生物启发的、结构灵活的共价树枝状大分子所取得的显著成果。在配套论文中，我们接下来报告了我们最近在设计、表征和测试一系列自组装两亲树枝状大分子及其相关结构改变方面所做的努力，以在体外和体内实现前所未有的高效siRNA递送。
