Department of Chemistry, University of Connecticut, Storrs, CT 06269, USA.
Department of Chemistry and Chemical Biology, Northeastern University, Boston, MA, 02115 USA.
Trends Biotechnol. 2019 Sep;37(9):983-994. doi: 10.1016/j.tibtech.2019.01.008. Epub 2019 Mar 14.
Nanoscale structures of therapeutic nucleic acids have shown enormous potential to help clinicians realize the promise of personaliz ed medicine using gene-specific treatments. With the advent of better sequencing through bioinformatic approaches and advancements in nucleic acid stabilization chemistries, the field of synthetic nucleic acid nanomaterials has advanced tremendously. This review focuses on an emerging strategy geared at gene silencing without the use of traditional polycation-based transfection agents and discusses how such nanostructures are being chemically tailored to navigate biological systems to improve their circulation time and biodistribution. We also address important challenges moving forward, including quantification of delivery and the multiplexing of sequences for regulating gene networks - a goal well suited for this unique class of materials.
治疗性核酸的纳米结构显示出巨大的潜力，可以帮助临床医生利用针对特定基因的治疗方法实现个性化医疗的承诺。随着生物信息学方法测序技术的进步和核酸稳定化学的发展，合成核酸纳米材料领域取得了巨大的进展。本综述专注于一种新兴的基因沉默策略，该策略不使用传统的基于聚阳离子的转染试剂，并讨论了如何通过化学方法对这些纳米结构进行修饰，以使其在生物系统中导航，从而提高其循环时间和生物分布。我们还讨论了前进过程中的重要挑战，包括对递药进行定量和对调控基因网络的序列进行多重化——这是一个非常适合这种独特材料的目标。
