High-Throughput Screening to Identify Modulators of Sarcospan

Cynthia Shu; Ekaterina Mokhonova; Rachelle H Crosbie

doi:10.1007/978-1-0716-2772-3_25

High-Throughput Screening to Identify Modulators of Sarcospan

Methods Mol Biol. 2023:2587:479-493. doi: 10.1007/978-1-0716-2772-3_25.

Authors

Cynthia Shu^{1

2}, Ekaterina Mokhonova^{1

2}, Rachelle H Crosbie^{3

4}

Affiliations

¹ Department of Integrative Biology and Physiology, University of California Los Angeles, Los Angeles, CA, USA.
² Department of Neurology, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, CA, USA.
³ Department of Integrative Biology and Physiology, University of California Los Angeles, Los Angeles, CA, USA. rcrosbie@physci.ucla.edu.
⁴ Department of Neurology, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, CA, USA. rcrosbie@physci.ucla.edu.

PMID: 36401045
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.

Keywords: Drug discovery; Duchenne muscular dystrophy; High-throughput screening; Sarcospan.

High-Throughput Screening to Identify Modulators of Sarcospan

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