AI Article Synopsis
- High-throughput screening is a method used to find small molecules that can potentially treat diseases, specifically focusing on Duchenne muscular dystrophy.
- This study discusses creating a scalable cell-based assay that targets the modulation of a protein called sarcospan.
- The process of validating successful candidates involves several steps, including additional screenings and tests to measure gene/protein levels and membrane stability.
Article 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.
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| http://dx.doi.org/10.1007/978-1-0716-2772-3_25 | DOI Listing |
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