Zamani Farzad, Suzuki Takayoshi
The Institute of Scientific and Industrial Research, Osaka University, Mihogaoka 8-1, Ibaraki, Osaka 567-0047, Japan.
J Med Chem. 2021 Jun 10;64(11):7110-7155. doi: 10.1021/acs.jmedchem.1c00154. Epub 2021 Jun 1.
RNAs are involved in an enormous range of cellular processes, including gene regulation, protein synthesis, and cell differentiation, and dysfunctional RNAs are associated with disorders such as cancers, neurodegenerative diseases, and viral infections. Thus, the identification of compounds with the ability to bind RNAs and modulate their functions is an exciting approach for developing next-generation therapies. Numerous RNA-binding agents have been reported over the past decade, but the design of synthetic molecules with selectivity for specific RNA sequences is still in its infancy. In this perspective, we highlight recent advances in targeting RNAs with synthetic molecules, and we discuss the potential value of this approach for the development of innovative therapeutic agents.
RNA参与了大量的细胞过程，包括基因调控、蛋白质合成和细胞分化，功能失调的RNA与癌症、神经退行性疾病和病毒感染等疾病有关。因此，鉴定具有结合RNA并调节其功能能力的化合物是开发下一代疗法的一种令人兴奋的方法。在过去十年中已经报道了许多RNA结合剂，但对特定RNA序列具有选择性的合成分子的设计仍处于起步阶段。从这个角度来看，我们强调了用合成分子靶向RNA的最新进展，并讨论了这种方法在开发创新治疗药物方面的潜在价值。
