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Adeno-Associated Virus Delivery of Viral Serpins for Ocular Diseases: Design and Validation

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Serpins

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1826))

Abstract

Adeno-associated virus (AAV) has become the preferred viral gene transfer platform for ocular gene therapy due to its known safety profile in human clinical trials. This viral vector has a 4.7 kbp (kilo base pair) carrying capacity (single-stranded DNA) and only retains the inverted terminal repeats (ITRs) from the original virus. Here we describe the design and testing of AAV vectors capable of delivering an anti-inflammatory serine protease inhibitor (serpin) derived from the myxoma virus. Myxoma is a rabbit species specific virus infection, a Leporipoxvirus. Myxomaviral proteins have been developed as therapeutic stand-alone immune-modulating proteins for inflammation-based disorders and the myxoma virus itself is under development as a viral oncolytic platform for cancer treatment. We fused the Serp2 gene with the GFP reporter gene through a self-cleaving peptide.

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Author information

Authors and Affiliations

  1. Department of Ophthalmology, University of Florida College of Medicine, Gainesville, FL, USA

    Cristhian J. Ildefonso

  2. Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL, USA

    Alfred S. Lewin

Authors
  1. Cristhian J. Ildefonso
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  2. Alfred S. Lewin
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Corresponding author

Correspondence to Cristhian J. Ildefonso .

Editor information

Editors and Affiliations

  1. Biodesign Center for Personalized Diagnostics and Center for Immunotherapy Vaccines and Virotherapy, Arizona State University, Tempe, AZ, USA

    Alexandra Lucas

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Ildefonso, C.J., Lewin, A.S. (2018). Adeno-Associated Virus Delivery of Viral Serpins for Ocular Diseases: Design and Validation. In: Lucas, A. (eds) Serpins. Methods in Molecular Biology, vol 1826. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-8645-3_16

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  • DOI: https://doi.org/10.1007/978-1-4939-8645-3_16

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-8644-6

  • Online ISBN: 978-1-4939-8645-3

  • eBook Packages: Springer Protocols

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