Photoactivatable molecular probes to study RNA-protein interactions

由于其丰富的结构多样性和广泛的生物学功能，以小分子为靶点的RNA正成为药物发现的重要策略。RNA的大部分细胞功能由蛋白质控制，这些相互作用的失调可能导致一系列人类疾病。因此，人们对识别与特定RNA结构结合的蛋白质的有效方法越来越感兴趣。本项目旨在开发一种新型的分子探针(基于有机小分子和/或金属-有机分子)来选择性地标记RNA结合蛋白。该项目中开发的新探针将用于研究RNA结构中的重复扩展，其中几个与各种神经和神经肌肉疾病有关。

Due to their rich structural diversity and broad range of biological functions, targeting RNA with small molecules is becoming an important strategy in drug discovery. Most cellular functions of RNA are controlled by proteins and dysregulation of these interactions can lead to a range of human diseases. Therefore, there is an increasing interest in efficient methods to identify proteins that bind to specific RNA structures. This project aims to develop a new class of molecular probe (based on small organic and/or metal-organic molecules) for the selective photolabeling of RNA-binding proteins. The new probes developed in this project will be applied to study repeat expansions in RNA structures, several of which are responsible for various neurological and neuromuscular diseases.