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Biotechnologies for Gene Therapy pp 61–83Cite as

Muscular Dystrophy Therapy Using Viral Vector-based CRISPR/Cas

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Abstract

Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic disorder caused by mutations in the DMD gene, which encodes the dystrophin protein. A lack of dystrophin disrupts the skeletal musculature, resulting in severe muscle degeneration. Currently, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system offers an evolved and precise tool of programmed genomic modification and is being widely studied as a therapeutic tool for treating various genetic diseases. Recently, CRISPR-mediated DMD therapy has been intensively studied as a means of correcting or bypassing disease-causing mutations, resulting in the permanent repair of mutated DMD gene and rescues of dystrophin expression. However, delivery methods remain a major barrier for CRISPR-mediated genome editing. Various viral vectors have been utilized as vehicles for sequences encoding CRISPR/Cas components. Efforts have been made to optimize the viral vector systems for efficient delivery of these components to treat DMD. Herein, we review diverse aspects of several viral vectors combined with CRISPR/Cas systems for DMD therapy and discuss their therapeutic potential and the challenges ahead.

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Acknowledgements

This work was supported by grants from the National Research Foundation of Korea grant funded by the Korea government [2022R1A2C1013352 and 2020R1A4A1016142].

Conflicts of Interest

The authors declare no conflicts of interest.

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Authors and Affiliations

  1. Department of Life and Nanopharmaceutical Sciences, Graduate School, Kyung Hee University, Seoul, Republic of South Korea

    Eunyoung Choi & Taeyoung Koo

  2. Department of Biomedical and Pharmaceutical Sciences, Graduate School, Kyung Hee University, Seoul, Republic of South Korea

    Taeyoung Koo

  3. Department of Pharmaceutical Science, College of Pharmacy, Kyung Hee University, Seoul, Republic of South Korea

    Taeyoung Koo

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  1. Eunyoung Choi
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Correspondence to Taeyoung Koo .

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Editors and Affiliations

  1. Department of Biomedical Engineering, College of Engineering and Polymer Science, The University of Akron, Akron, OH, USA

    Yang H. Yun

  2. Department of Cancer Biology and Genetics College of Medicine, The Ohio State University, Columbus, OH, USA

    Kristine E. Yoder

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Choi, E., Koo, T. (2022). Muscular Dystrophy Therapy Using Viral Vector-based CRISPR/Cas. In: Yun, Y.H., Yoder, K.E. (eds) Biotechnologies for Gene Therapy. Springer, Cham. https://doi.org/10.1007/978-3-030-93333-3_4

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