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Related Experiment Video

Updated: Jun 21, 2026

Using Adeno-associated Virus as a Tool to Study Retinal Barriers in Disease
10:14

Using Adeno-associated Virus as a Tool to Study Retinal Barriers in Disease

Published on: April 19, 2015

Adeno-associated virus serotype-9 efficiently transduces the retinal outer plexiform layer.

Bo Lei1, Keqing Zhang, Yongping Yue

  • 1Department of Ophthalmology, The First Affiliated Hospital of Chongqing Medical University, 1 You Yi Road, Yu Zhong District, Chongqing, China. bolei99@126.com

Molecular Vision
|July 24, 2009
PubMed
Summary

Adeno-associated virus serotype-9 (AAV-9) effectively delivers genes to the mouse retina across all ages. Subretinal AAV-9 injection shows promise for treating outer plexiform layer diseases without causing acute retinal damage.

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Area of Science:

  • Ophthalmology
  • Gene Therapy
  • Neuroscience

Background:

  • Adeno-associated virus serotype-9 (AAV-9) is a leading candidate for gene delivery due to its safety and efficacy.
  • Retinal gene therapy aims to restore vision by delivering therapeutic genes to specific retinal cells.

Purpose of the Study:

  • To evaluate the efficacy and safety of AAV-9 mediated gene delivery in the mouse retina.
  • To assess AAV-9 transduction across different age groups and in a disease model.

Main Methods:

  • Subretinal injection of three distinct AAV-9 vectors (AAV-9.RSV.AP, AAV-9.CMV.eGFP, AAV-9.CMV.R4-23/C) into young, adult, and old C57BL/6J mice.
  • Administration of AAV-9.CMV.R4-23/C to mdx(3cv) mice, a model for Duchenne muscular dystrophy (DMD), to evaluate micro-dystrophin expression.

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Viral Tracing of Genetically Defined Neural Circuitry
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Last Updated: Jun 21, 2026

Using Adeno-associated Virus as a Tool to Study Retinal Barriers in Disease
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Published on: April 19, 2015

Viral Tracing of Genetically Defined Neural Circuitry
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  • Analysis of transgene expression via histochemical and immunofluorescence staining, and assessment of retinal function using electroretinograms (ERGs).

    • Main Results:

    • Widespread retinal transduction was observed in all age groups, with robust expression in the retinal pigment epithelium, outer nuclear layer, Müller cells, and the outer plexiform layer (OPL).
    • Efficient micro-dystrophin expression was confirmed in the OPL of mdx(3cv) mice, mirroring normal dystrophin localization.
    • Subretinal AAV-9.RSV.AP delivery did not result in any observed morphological or ERG abnormalities at five weeks post-injection.

    Conclusions:

    • AAV-9 serves as a potent vector for retinal gene delivery, demonstrating broad transduction capabilities.
    • Subretinal AAV-9 administration is safe, causing no significant acute retinal damage.
    • AAV-9-mediated OPL transduction presents a promising therapeutic strategy for diseases affecting this retinal layer.