THREE-DIMENSIONAL ARCHITECTURES OF COMPLEX SELF-ASSEMBLING RNA PARTICLES

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 通过利用丰富的天然RNA结构基序，目前有可能产生具有有趣的潜在治疗活性的复杂的人工RNA纳米结构。最近，我们使用了一种计算机辅助的方法来设计和设计多功能的、可编程的、三维(3D)自组装的RNA颗粒，并对其几何、大小和组成进行精确控制。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3D RNA颗粒可能采用小立方体或长方体的形状。然而，为了明确地验证他们的整体3D架构，低温电磁表征是必要的。 这些RNA颗粒可以潜在地作为纳米支架用于各种治疗功能，如RNA适体和siRNAs。因此，它们在生物医学应用方面具有巨大的潜力。

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.