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High-Throughput Screening to Identify Modulators of Sarcospan

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Muscular Dystrophy Therapeutics

Part of the book series: Methods in Molecular Biology ((MIMB,volume 2587))

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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Acknowledgments

This work was supported by the Muscle Cell Biology, Pathogenesis, and Therapeutics Training Grant (NIH T32 AR065972); Pilot and Feasibility Seed Grant program (NIH/NIAMS P30 AR057230); UCLA Department of Integrative Biology & Physiology Eureka Scholarship; Muscular Dystrophy Association (MDA 274143; Venture Philanthropy Program); NIH NIAMS (R01 AR048179); and NIH NHLBI (R01 HL126204).

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Authors and Affiliations

  1. Department of Integrative Biology and Physiology, University of California Los Angeles, Los Angeles, CA, USA

    Cynthia Shu, Ekaterina Mokhonova & Rachelle H. Crosbie

  2. Department of Neurology, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, CA, USA

    Cynthia Shu, Ekaterina Mokhonova & Rachelle H. Crosbie

Authors
  1. Cynthia Shu
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  2. Ekaterina Mokhonova
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  3. Rachelle H. Crosbie
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Corresponding author

Correspondence to Rachelle H. Crosbie .

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Editors and Affiliations

  1. Department of Medical Genetics, University of Alberta, Edmonton, AB, Canada

    Rika Maruyama

  2. Department of Medical Genetics, University of Alberta, Edmonton, AB, Canada

    Toshifumi Yokota

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Shu, C., Mokhonova, E., Crosbie, R.H. (2023). High-Throughput Screening to Identify Modulators of Sarcospan. In: Maruyama, R., Yokota, T. (eds) Muscular Dystrophy Therapeutics. Methods in Molecular Biology, vol 2587. Humana, New York, NY. https://doi.org/10.1007/978-1-0716-2772-3_25

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  • DOI: https://doi.org/10.1007/978-1-0716-2772-3_25

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  • Publisher Name: Humana, New York, NY

  • Print ISBN: 978-1-0716-2771-6

  • Online ISBN: 978-1-0716-2772-3

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