Abstract：
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.
摘要：
高通量筛选能够发现改善疾病的小分子。这里,我们描述了一个可扩展的发展，基于细胞的测定法，以筛选调节肌肉生长的小分子，用于治疗Duchenne肌营养不良。我们详细说明了命中验证管道，其中包括二次筛查，基因/蛋白质定量，和体外膜稳定性测定。
