Shu Cynthia, Mokhonova Ekaterina, Crosbie Rachelle H
Department of Integrative Biology and Physiology, University of California Los Angeles, Los Angeles, CA, USA.
Department of Neurology, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, CA, USA.
Methods Mol Biol. 2023;2587:479-493. doi: 10.1007/978-1-0716-2772-3_25.
High-throughput screening enables the discovery of disease-modifying small molecules. Here, we describe the development of a scalable, cell-based assay to screen for small molecules that modulate sarcospan for the treatment of Duchenne muscular dystrophy. We detail the hit validation pipeline, which includes secondary screening, gene/protein quantification, and an in vitro membrane stability assay.
高通量筛选能够发现可改变疾病进程的小分子。在此，我们描述了一种可扩展的、基于细胞的检测方法的开发，用于筛选调节肌膜跨度蛋白以治疗杜氏肌营养不良症的小分子。我们详细介绍了命中验证流程，其中包括二次筛选、基因/蛋白质定量以及体外膜稳定性检测。
