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Design of Muscle Gene Therapy Expression Cassette

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Muscle Gene Therapy

Abstract

The first gene therapy drug approved by the European Regulatory Commission involves the transfer of a therapeutic gene to the muscle by adeno-associated viral vector (AAV). Now, muscle gene transfer is quickly becoming a therapy of choice for muscle and non-muscle diseases. Successful muscle gene therapy requires efficient expression of therapeutic proteins in the muscle without causing any toxicity and side effects. To achieve this, the expression cassette of therapeutic proteins needs to be designed rationally. A typical expression cassette usually contains a promoter to initiate transcription, the coding sequence of a transgene, and a termination signal to terminate transcription. Other cis-regulatory elements can be added into the 5′- and 3′-untranslated regions. In this chapter, we review the development of the components of the expression cassette in the context of muscle gene therapy.

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Authors and Affiliations

  1. Department of Molecular Microbiology and Immunology, School of Medicine, University of Missouri, Columbia, MO, USA

    Yi Lai & Dongsheng Duan

  2. Department of Neurology, School of Medicine, University of Missouri, Columbia, MO, USA

    Dongsheng Duan

  3. Department of Biomedical Sciences, College of Veterinary Medicine, University of Missouri, Columbia, MO, USA

    Dongsheng Duan

  4. Department of Bioengineering, University of Missouri, Columbia, MO, USA

    Dongsheng Duan

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  1. Yi Lai
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  2. Dongsheng Duan
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Correspondence to Yi Lai .

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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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Lai, Y., Duan, D. (2019). Design of Muscle Gene Therapy Expression Cassette. In: Duan, D., Mendell, J. (eds) Muscle Gene Therapy. Springer, Cham. https://doi.org/10.1007/978-3-030-03095-7_8

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