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Design of AAV Vectors for Delivery of Large or Multiple Transgenes

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Adeno-Associated Virus Vectors

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1950))

Abstract

Adeno-associated virus (AAV)-mediated gene therapy has evolved from bench to bedside, and now is the therapy of choice for certain inherited diseases. However, the small packaging capacity of AAV vectors prevents this technique from treating genetic diseases with mutations of large genes. Multiple strategies, including split AAV gene delivery and oversized AAV gene delivery, have been explored to deliver large gene expression cassettes. These strategies have gained some success in animal experiments. In this chapter, we review the progress of AAV-mediated delivery of large expression cassettes. We also review using AAV to deliver multiple transgenes.

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Acknowledgments

This work was supported by grants from Jackson Freel DMD Research Fund (D.D.), National Institutes of Health (AR-69085) (D.D.), Department of Defense (MD150133) (D.D.), Hope for Javier (D.D.), and Duchenne Parent Project (the Netherlands) (Y.L.).

Disclosure: D.D. is a member of the scientific advisory board for Solid Biosciences and an equity holder of Solid Biosciences. D.D. has received research supports from Solid Biosciences, which are unrelated to this book chapter.

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Authors and Affiliations

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

    Aman Patel, Junling Zhao, Dongsheng Duan & Yi Lai

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

    Dongsheng Duan

  3. Department of Neurology, School of 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. Aman Patel
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  2. Junling Zhao
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  3. Dongsheng Duan
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  4. Yi Lai
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Correspondence to Yi Lai .

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Editors and Affiliations

  1. Department of Neurosciences, University of California, San Diego, La Jolla, CA, USA

    Michael J. Castle

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Patel, A., Zhao, J., Duan, D., Lai, Y. (2019). Design of AAV Vectors for Delivery of Large or Multiple Transgenes. In: Castle, M. (eds) Adeno-Associated Virus Vectors. Methods in Molecular Biology, vol 1950. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-9139-6_2

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  • DOI: https://doi.org/10.1007/978-1-4939-9139-6_2

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