%0 Journal Article
%T High-Throughput Screening to Identify Modulators of Sarcospan.
%A Shu C
%A Mokhonova E
%A Crosbie RH
%J Methods Mol Biol
%V 2587
%N 0
%D 2023
%M 36401045
暂无%R 10.1007/978-1-0716-2772-3_25
%X 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.