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Muscle Gene Therapy pp 239–252Cite as

Biomarkers for Muscle Disease Gene Therapy

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Abstract

Molecular biomarkers are becoming increasingly attractive in drug development programs for muscle diseases especially for Duchenne muscular dystrophy (DMD). Robust and validated blood and muscle biomarkers that are sensitive to drug treatment (e.g., pharmacodynamic biomarkers) and that can predict later clinical outcomes (e.g., surrogate biomarkers) will likely aid in developing effective therapies for DMD at several levels. Peripheral blood biomarkers can serve as more objective and acute readouts of drug effects relative to clinical outcome measures. Furthermore, they may minimize the burden on patients and families regarding participating in trials and can help with go-no-go decisionmaking at early stages of drug development possibly reducing the length and cost of the clinical development program. Lastly, they may provide insights into pathobiochemical pathways and help define novel therapeutic targets. In this chapter, we will focus mainly on molecular biomarker advances in DMD with the emphasis on their utility to assess efficacy of gene therapy in this disease.

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Authors and Affiliations

  1. Binghamton University School of Pharmacy & Pharmaceutical Sciences, Binghamton, NY, USA

    Yetrib Hathout, Kanneboyina Nagaraju & Eric P. Hoffman

  2. Solid Biosciences, Cambridge, MA, USA

    Kristy J. Brown

Authors
  1. Yetrib Hathout
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  2. Kristy J. Brown
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  3. Kanneboyina Nagaraju
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  4. Eric P. Hoffman
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Corresponding author

Correspondence to Yetrib Hathout .

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Editors and Affiliations

  1. Department of Molecular Microbiology and Immunology, School of Medicine, University of Missouri, Columbia, MO, USA; Department of Neurology, School of Medicine, University of Missouri, Columbia, MO, USA, Department of Biomedical Sciences College of Veterinary Medicine, University of Missouri, Columbia, MO, USA; Department of Bioengineering, University of Missouri, Columbia, MO, USA

    Dongsheng Duan

  2. Department of Pediatrics Nationwide Children’s Hospital and Research Institute, Department of Neurology Nationwide Children’s Hospital and Research Institute, The Ohio State University, Columbus, OH, USA

    Jerry R. Mendell

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Hathout, Y., Brown, K.J., Nagaraju, K., Hoffman, E.P. (2019). Biomarkers for Muscle Disease Gene Therapy. In: Duan, D., Mendell, J. (eds) Muscle Gene Therapy. Springer, Cham. https://doi.org/10.1007/978-3-030-03095-7_14

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