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Gene- and Cell-Based Treatment Strategies for the Eye pp 65–76Cite as

Neovascular Age-Related Macular Degeneration: Secretion Gene Therapy

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Part of the book series: Essentials in Ophthalmology ((ESSENTIALS))

Abstract

Age-related macular degeneration (ARMD) is the major cause of blindness in the developed world. The neovascular form of ARMD is characterized by the development and growth of new leaky blood vessels into the retina. Although wet-ARMD is now treatable with frequent intravitreal injections of antiangiogenic molecules, there is an unmet need for the long-term treatment of the disease. This chapter describes the development of a naturally occurring antiangiogenic agent using a recombinant adeno-associated virus for its long-term delivery. Firstly, data demonstrating the significance of vascular endothelial growth factor in the development of wet-ARMD is presented, followed by a description of the selection of the antiangiogenic agent and the delivery system. A detailed description of the construct, production, validation, and biological and pharmacological assessment follows. In addition, preclinical in vitro and in vivo data on mice, rats, and monkeys are presented. Finally, an early-stage clinical trial in human subjects is described.

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Compliance with Ethical Requirements

Professors Elizabeth Rakoczy and Ian Constable provide consultancy for Avalanche Biotechnologies, Inc.

All procedures followed were in accordance with the ethical standards of the responsible committee on human experimentation (institutional and national) and with the Helsinki Declaration of 1975, as revised in 2000. Informed consent was obtained from all patients for being included in the study.

All institutional and national guidelines for the care and use of laboratory animals were followed. All experiments were approved by The University of Western Australia.

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Authors and Affiliations

  1. Department of Molecular Ophthalmology, Centre for Ophthalmology and Visual Sciences, The University of Western Australia, Crawley, WA, Australia

    Elizabeth P. Rakoczy PhD & Chooi-May Lai PhD

  2. Department of Ophthalmology, Lions Eye Institute, Sir Charles Gairdner Hospital, University of Western Australia, Crawley, WA, Australia

    Ian J. Constable MBBS, DSc, FRANZCO

Authors
  1. Elizabeth P. Rakoczy PhD
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  2. Chooi-May Lai PhD
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  3. Ian J. Constable MBBS, DSc, FRANZCO
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Corresponding author

Correspondence to Elizabeth P. Rakoczy PhD .

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Editors and Affiliations

  1. Department of Molecular Ophthalmology, Centre for Ophthalmology and Visual Sciences, The University of Western Australia, Crawley, West Australia, Australia

    Elizabeth P. Rakoczy

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Rakoczy, E.P., Lai, CM., Constable, I.J. (2015). Neovascular Age-Related Macular Degeneration: Secretion Gene Therapy. In: Rakoczy, E. (eds) Gene- and Cell-Based Treatment Strategies for the Eye. Essentials in Ophthalmology. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-662-45188-5_6

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  • DOI: https://doi.org/10.1007/978-3-662-45188-5_6

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