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Adeno-Associated Virus Mediated Gene Therapy for Retinal Degenerative Diseases

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Adeno-Associated Virus

Part of the book series: Methods in Molecular Biology ((MIMB,volume 807))

Abstract

Retinal gene therapy holds great promise for the treatment of inherited and noninherited blinding diseases such as retinitis pigmentosa and age-related macular degeneration. The most widely used vectors for ocular gene delivery are based on adeno-associated virus (AAV) because it mediates long-term transgene expression in a variety of retinal cell types and elicits minimal immune responses. Inherited retinal diseases are nonlethal and have a wide level of genetic heterogeneity. Many of the genes have now been identified and their function elucidated, providing a major step towards the development of gene-based treatments. Extensive preclinical evaluation of gene transfer strategies in small and large animal models is key to the development of successful gene-based therapies for the retina. These preclinical studies have already allowed the field to reach the point where gene therapy to treat inherited blindness has been brought to clinical trial.

In this chapter, we focus on AAV-mediated specific gene therapy for inherited retinal degenerative diseases, describing the disease targets, the preclinical studies in animal models and the recent success of the LCA-RPE65 clinical trials.

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Author information

Authors and Affiliations

  1. Department of Ophthalmology, Justus-Liebig-University Giessen, Gissen, Germany

    Knut Stieger

  2. Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA

    Therese Cronin

  3. Department of Ophthalmology, F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA

    Jean Bennett

  4. Laboratoire de Thérapie Génique, INSERM UMR649, Université de Nantes, Nantes, France

    Fabienne Rolling

Authors
  1. Knut Stieger
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  2. Therese Cronin
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  3. Jean Bennett
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  4. Fabienne Rolling
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Corresponding author

Correspondence to Jean Bennett .

Editor information

Editors and Affiliations

  1. Center of Excellence for Regenerative, Health Biotechnology (CERHB), University of Florida, Innovation Drive 13706, Alachua, 32615, Florida, USA

    Richard O. Snyder

  2. INSERM U649, Labo. Thérapie Génique, CHU Hôtel Dieu, bd. Jean Monnet 30, Nantes CX 1, 44035, France

    Philippe Moullier

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Stieger, K., Cronin, T., Bennett, J., Rolling, F. (2012). Adeno-Associated Virus Mediated Gene Therapy for Retinal Degenerative Diseases. In: Snyder, R., Moullier, P. (eds) Adeno-Associated Virus. Methods in Molecular Biology, vol 807. Humana Press. https://doi.org/10.1007/978-1-61779-370-7_8

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  • DOI: https://doi.org/10.1007/978-1-61779-370-7_8

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  • Publisher Name: Humana Press

  • Print ISBN: 978-1-61779-369-1

  • Online ISBN: 978-1-61779-370-7

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