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Neutralizing Antibodies Against Adeno-Associated Virus (AAV): Measurement and Influence on Retinal Gene Delivery

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Retinal Gene Therapy

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1715))

Abstract

Adeno-associated viral vectors have become widely used in the clinic for retinal gene therapy. Thanks to AAVs impeccable safety profile and positive functional outcomes in its clinical application, interest in retinal gene therapy has increased exponentially over the past decade. Although early clinical trials have shown there is little influence of neutralizing antibodies on the performance of AAV when vector is administered into the subretinal space, recent findings suggest neutralizing antibodies may play a role when AAV is delivered via the intravitreal route. These findings highlight the importance of microenvironment on gene delivery and stress the need for a versatile assay to screen subjects for the presence of AAV-neutralizing antibodies. Measuring NAb titers against AAV prior and after gene therapy will help us better understand the impact of preexisting immunity on gene transfer, especially when the vector is administered intravitreally.

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Author information

Authors and Affiliations

  1. INSERM, U968, Paris, France

    Mélissa Desrosiers & Deniz Dalkara

  2. Sorbonne Universités, UPMC Univ Paris 06, UMR_S 968, Institut de la Vision, Paris, France

    Mélissa Desrosiers & Deniz Dalkara

  3. CNRS, UMR_7210, Paris, France

    Mélissa Desrosiers & Deniz Dalkara

Authors
  1. Mélissa Desrosiers
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  2. Deniz Dalkara
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Corresponding author

Correspondence to Deniz Dalkara .

Editor information

Editors and Affiliations

  1. Department of Ophthalmology, Leiden University Medical Center (LUMC), Leiden, The Netherlands

    Camiel J.F. Boon

  2. Department of Ophthalmology, Leiden University Medical Center (LUMC), Leiden, The Netherlands

    Jan Wijnholds

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Desrosiers, M., Dalkara, D. (2018). Neutralizing Antibodies Against Adeno-Associated Virus (AAV): Measurement and Influence on Retinal Gene Delivery. In: Boon, C., Wijnholds, J. (eds) Retinal Gene Therapy. Methods in Molecular Biology, vol 1715. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-7522-8_16

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  • DOI: https://doi.org/10.1007/978-1-4939-7522-8_16

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-7521-1

  • Online ISBN: 978-1-4939-7522-8

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