{Reference Type}: Journal Article
{Title}: High-Throughput Screening to Identify Modulators of Sarcospan.
{Author}: Shu C;Mokhonova E;Crosbie RH;
{Journal}: Methods Mol Biol
{Volume}: 2587
{Issue}: 0
{Year}: 2023
暂无{DOI}: 10.1007/978-1-0716-2772-3_25
{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.