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Optimized Subretinal Injection Technique for Gene Therapy Approaches

  • Protocol
Book cover Retinal Degeneration

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1834))

Abstract

Gene therapy for inherited eye diseases requires local viral vector delivery by intraocular injection. Since large animal models are lacking for most of these diseases, genetically modified mouse models are commonly used in preclinical proof-of-concept studies. However, because of the relatively small mouse eye, adverse effects of the subretinal delivery procedure itself may interfere with the therapeutic outcome. The method described here aims to provide the details relevant to perform a transscleral pars plana virus-mediated gene transfer to achieve an optimized therapeutic effect in the small mouse eye.

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Acknowledgments

This work was supported by Deutsche Forschungsgemeinschaft (Se837/5-2, Se837/6-1, Se837/6-2, Se837/7-1 from M. W. S.) and Tistou & Charlotte Kerstan Foundation (RD-CURE).

Author information

Authors and Affiliations

  1. Division of Ocular Neurodegeneration, Institute for Ophthalmic Research, Centre for Ophthalmology, Eberhard Karls Universität Tübingen, Tübingen, Germany

    Regine Mühlfriedel, Marina Garcia Garrido, Vithiyanjali Sothilingam & Mathias W. Seeliger

  2. Department of Pharmacy—Center for Drug Research, Center for Integrated Protein Science Munich (CIPSM), Ludwig-Maximilians-Universität München, Munich, Germany

    Stylianos Michalakis, Christian Schön & Martin Biel

Authors
  1. Regine Mühlfriedel
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  2. Stylianos Michalakis
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  3. Marina Garcia Garrido
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  4. Vithiyanjali Sothilingam
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  5. Christian Schön
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  6. Martin Biel
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  7. Mathias W. Seeliger
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Corresponding author

Correspondence to Regine Mühlfriedel .

Editor information

Editors and Affiliations

  1. Institute of Human Genetics, University of Regensburg, Regensburg, Germany

    Bernhard H. F. Weber

  2. Department of Ophthalmology, University of Cologne, Cologne, Germany

    Thomas Langmann

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Mühlfriedel, R. et al. (2019). Optimized Subretinal Injection Technique for Gene Therapy Approaches. In: Weber, B.H.F., Langmann, T. (eds) Retinal Degeneration. Methods in Molecular Biology, vol 1834. Humana, New York, NY. https://doi.org/10.1007/978-1-4939-8669-9_26

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  • DOI: https://doi.org/10.1007/978-1-4939-8669-9_26

  • Publisher Name: Humana, New York, NY

  • Print ISBN: 978-1-4939-8668-2

  • Online ISBN: 978-1-4939-8669-9

  • eBook Packages: Springer Protocols

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