Challenges to optimizing RNA nanostructures for large scale production and controlled therapeutic properties.
Challenges to optimizing RNA nanostructures for large scale production and controlled therapeutic properties.
复制标题
DOI：
10.2217/nnm-2020-0034
复制
发表时间：
2020-05-26
期刊：
影响因子：
--
通讯作者：
Afonin, Kirill A
中科院分区：
文献类型：
--
作者：
Chandler, Morgan;Panigaj, Martin;Afonin, Kirill A
核酸已被用来构建广泛的纳米结构，其设计、理化性质、细胞加工和生物医学应用各不相同。然而，核酸纳米组件的更广泛的治疗适应，特别是基于 RNA 的纳米颗粒，在走向临床（前）环境方面面临着一些挑战。其一，核酸的大批量合成仍处于开发阶段，多链和化学修饰的组装需要更大的生产能力，同时保持一致的医疗级产量。此外，这些纳米材料未知的免疫刺激带来了额外的挑战，为了优化临床批准的 RNA 纳米粒子的未来开发，必须克服这些挑战。
Nucleic acids have been utilized to construct an expansive collection of nanoarchitectures varying in design, physicochemical properties, cellular processingand biomedical applications. However, the broader therapeutic adaptation of nucleic acid nanoassemblies in general, and RNA-based nanoparticles in particular, have faced several challenges in moving towards (pre)clinical settings. For one, the large-batch synthesis of nucleic acids is still under development, with multi-stranded and chemically modified assemblies requiring greater production capacity while maintaining consistent medical-grade outputs. Furthermore, the unknown immunostimulation by these nanomaterials poses additional challenges, necessary to be overcome for optimizing future development of clinically approved RNA nanoparticles.