Synthetic RNA Modulators in Drug Discovery
Synthetic RNA Modulators in Drug Discovery
复制标题
药物发现中的合成 RNA 调节剂
DOI：
10.1021/acs.jmedchem.1c00154
复制
发表时间：
2021
影响因子：
7.3
通讯作者：
Suzuki Takayoshi
中科院分区：
文献类型：
--
作者：
Zamani Farzad;Suzuki Takayoshi
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，而功能失调的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并调节其功能的能力的化合物是开发下一代疗法的令人兴奋的方法。在过去的十年中，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，我们强调了最近的进展，靶向RNA与合成分子，我们讨论了这种方法的潜在价值的创新治疗药物的发展。
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.