Department of Chemistry and Chemical Engineering, Chalmers University of Technology, Kemivägen 10, SE-41296 Gothenburg, Sweden.
LanteRNA (Stealth Labels Biotech AB), c/o Chalmers Ventures AB, Vera Sandbergs allé 8, SE-41296 Gothenburg, Sweden.
Nucleic Acids Res. 2024 Sep 23;52(17):10102-10118. doi: 10.1093/nar/gkae722.
RNA and its building blocks play central roles in biology and have become increasingly important as therapeutic agents and targets. Hence, probing and understanding their dynamics in cells is important. Fluorescence microscopy offers live-cell spatiotemporal monitoring but requires labels. We present two fluorescent adenine analogue nucleoside phosphates which show spontaneous uptake and accumulation in cultured human cells, likely via nucleoside transporters, and show their potential utilization as cellular RNA labels. Upon uptake, one nucleotide analogue, 2CNqAXP, localizes to the cytosol and the nucleus. We show that it could then be incorporated into de novo synthesized cellular RNA, i.e. it was possible to achieve metabolic fluorescence RNA labeling without using genetic engineering to enhance incorporation, uptake-promoting strategies, or post-labeling through bio-orthogonal chemistries. By contrast, another nucleotide analogue, pAXP, only accumulated outside of the nucleus and was rapidly excreted. Consequently, this analogue did not incorporate into RNA. This difference in subcellular accumulation and retention results from a minor change in nucleobase chemical structure. This demonstrates the importance of careful design of nucleoside-based drugs, e.g. antivirals to direct their subcellular localization, and shows the potential of fine-tuning fluorescent base analogue structures to enhance the understanding of the function of such drugs.
RNA 及其结构单元在生物学中起着核心作用,并且作为治疗剂和靶标变得越来越重要。因此,探测和理解它们在细胞中的动态变化非常重要。荧光显微镜提供了活细胞时空监测,但需要标记物。我们提出了两种荧光腺嘌呤类似物核苷磷酸,它们可以自发地被培养的人类细胞摄取和积累,可能是通过核苷转运体,并且显示了它们作为细胞 RNA 标记物的潜在用途。摄取后,一种核苷酸类似物 2CNqAXP 定位于细胞质和细胞核。我们表明,它随后可以被整合到新合成的细胞 RNA 中,即可以在不使用遗传工程增强整合、促进摄取的策略或通过生物正交化学进行标记后,实现代谢荧光 RNA 标记。相比之下,另一种核苷酸类似物 pAXP 仅在核外积累并迅速被排出。因此,该类似物没有整合到 RNA 中。这种在细胞内积累和保留方面的差异源于碱基化学结构的微小变化。这证明了核苷类药物设计的重要性,例如抗病毒药物来指导其亚细胞定位,并展示了精细调整荧光碱基类似物结构的潜力,以增强对这些药物功能的理解。