Department of Chemistry and Biochemistry, College of Arts and Sciences, UCLA, Los Angeles, CA, 90095, USA.
Department of Chemistry and Biochemistry, College of Arts and Sciences, UCLA, Los Angeles, CA, 90095, USA; Department of Biological Chemistry, David Geffen School of Medicine, UCLA, Los Angeles, CA, 90095, USA; Molecular Biology Institute, UCLA, Los Angeles, CA, 90095, USA; DOE Institute for Genomics and Proteomics, UCLA, Los Angeles, CA, 90095, USA; Jonsson Comprehensive Cancer Center, UCLA, Los Angeles, CA, 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, UCLA, Los Angeles, CA, 90095, USA.
Curr Opin Chem Biol. 2021 Jun;62:13-23. doi: 10.1016/j.cbpa.2020.12.006. Epub 2021 Jan 31.
RNA binding proteins (RBPs) are a large and diverse class of proteins that regulate all aspects of RNA biology. As RBP dysregulation has been implicated in a number of human disorders, including cancers and neurodegenerative disease, small molecule chemical probes that target individual RBPs represent useful tools for deciphering RBP function and guiding the production of new therapeutics. While RBPs are often thought of as tough-to-drug, the discovery of a number of small molecules that target RBPs has spurred considerable recent interest in new strategies for RBP chemical probe discovery. Here we review current and emerging technologies for high throughput RBP-small molecule screening that we expect will help unlock the full therapeutic potential of this exciting protein class.
RNA 结合蛋白（RBPs）是一大类多样化的蛋白质，它们调节 RNA 生物学的各个方面。由于 RBP 失调与许多人类疾病有关，包括癌症和神经退行性疾病，因此针对个别 RBP 的小分子化学探针是破译 RBP 功能和指导新型治疗药物开发的有用工具。虽然 RBP 通常被认为是难以成药的，但发现了一些针对 RBP 的小分子，这激发了人们对 RBP 化学探针发现的新策略的浓厚兴趣。在这里，我们综述了当前和新兴的高通量 RBP-小分子筛选技术，我们预计这些技术将有助于释放这一令人兴奋的蛋白质类别的全部治疗潜力。
