Istituto per l'Endocrinologia ed Oncologia Sperimentale, CNR, Naples, Italy.
Department of Internal Medicine, University of Iowa, Iowa City, IA, USA.
Methods Mol Biol. 2021;2282:31-42. doi: 10.1007/978-1-0716-1298-9_3.
Despite the therapeutic utility of small interfering RNA (siRNA) molecules, the development of a safe and reliable method to selectively target diseased organs and tissues is still a critical need for their translation to the clinic. Here we describe how nucleic acid-based aptamers against cell surface epitopes may be used to address this issue. We discuss the most recent examples and advances in the field of aptamer siRNA delivery and provide a fast and simple protocol for the design and generation of aptamer-siRNA chimeras. The described approach is based on the annealing of the targeting aptamer, and the antisense strand through "stick" complementary sequences elongated at their 3' end, and the subsequent paring with the sense strand. Such a protocol allows a modular non-covalent generation of the constructs and permits an efficient delivery of the siRNA moiety into aptamer target cells.
尽管小干扰 RNA（siRNA）分子具有治疗作用，但开发一种安全可靠的方法来选择性地靶向患病器官和组织，对于将其转化为临床应用仍然是一个关键需求。在这里，我们描述了如何使用针对细胞表面表位的基于核酸的适体来解决这个问题。我们讨论了适体 siRNA 递送领域的最新实例和进展，并提供了一种快速简单的设计和生成适体-siRNA 嵌合体的方案。所描述的方法基于通过在 3'末端延伸的“粘性”互补序列使靶向适体和反义链退火，然后与有义链配对。这种方案允许构建体的模块化非共价生成，并允许将 siRNA 部分有效递送到适体靶细胞中。
