THREE-DIMENSIONAL ARCHITECTURES OF COMPLEX SELF-ASSEMBLING RNA PARTICLES

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 通过利用天然RNA结构基序的丰富库，目前有可能产生具有有趣的潜在治疗活性的复杂人工RNA纳米结构。最近，我们采用了计算机辅助方法来设计和工程多功能，可编程，三维（3D）自组装粒子由RNA制成，精确控制其几何形状，大小和组成。这些结构复杂的RNA颗粒可以以良好的产率生产。正如我们的理论建模方法所预期的那样，各种生化表征技术表明，这些RNA的3D颗粒可能采用小立方体或长方体的形状。然而，冷冻EM表征是必要的，以明确地验证其整体3D架构。 这些RNA颗粒可以潜在地用作各种治疗功能的纳米支架，例如RNA适体和siRNA。因此，它们在生物医学应用方面具有巨大潜力。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. By taking advantage of the rich repertoire of natural RNA structural motifs, it is presently possible to generate complex artificial RNA nano-structures with interesting potential therapeutic activities. Recently, we have employed a computer-aided approach to design and engineer versatile, programmable, three-dimensional (3D) self-assembling particles made of RNA with precise control over their geometry, size and composition. These structurally complex RNA particles can be produced with good yields. As anticipated by our theoretical modeling approach, various biochemical characterization techniques suggest that these 3D particles of RNA might adopt the shape of small cubes or cuboids. However, cryo-EM characterization is necessary to validate non-ambiguously their overall 3D architectures. These RNA particles can potentially serve as nano-scaffolds for various therapeutic functions such as RNA aptamers and siRNAs. They have therefore a great potential for biomedical applications.