Network of Excellence for Functional Biomaterials, National University of Ireland-Galway, Ireland
Adv Drug Deliv Rev. 2011 Apr 30;63(4-5):197-208. doi: 10.1016/j.addr.2011.01.006. Epub 2011 Jan 15.
Tissue engineering aims to provide structural and biomolecular cues to compromised tissues through scaffolds. An emerging biomolecular cue is that of RNA interference by which the expression of genes can be silenced through a potent endogenous pathway. Recombinant viral-based approaches in RNAi delivery exist; however non-viral strategies offer many opportunities to exploit this mechanism of regulation in a safer way. Current RNAi therapies in clinical trials are without a vector (naked) or have slightly modified structures. Modification of these molecules with efficient backbone moieties for improved stability and potency, protecting and buffering them with delivery vehicles, and using scaffolds as reservoirs of delivery is at the frontier of current research. However, to enable an efficient sustained therapeutic effect scaffolds have a potentially significant role to play. This review presents non-viral delivery of RNAi that have been attempted via tissue engineered scaffolds. For RNAi to have a clinical impact, it is imperative to evaluate optimal delivery systems to ensure that the efficacy of this promising technology can be maximized.
组织工程旨在通过支架为受损组织提供结构和生物分子线索。一种新兴的生物分子线索是 RNA 干扰，通过这种内源性途径可以沉默基因的表达。目前存在基于重组病毒的 RNAi 传递方法；然而，非病毒策略为以更安全的方式利用这种调控机制提供了许多机会。目前临床试验中的 RNAi 疗法没有载体（裸）或只有略微修改的结构。通过使用高效的骨架部分来修饰这些分子，以提高其稳定性和效力，使用输送载体来保护和缓冲它们，并将支架用作输送的储库，这是当前研究的前沿。然而，为了实现有效的持续治疗效果，支架具有重要的潜在作用。本综述介绍了通过组织工程支架尝试的非病毒 RNAi 传递。为了使 RNAi 产生临床影响，必须评估最佳的输送系统，以确保这项有前途的技术的功效能够最大化。
