May 2008
Volume 49, Issue 13
Free
ARVO Annual Meeting Abstract  |   May 2008
Efficient Middle Retinal Transduction With Adeno-Associated Virus Serotype-9
Author Affiliations & Notes
  • B. Lei
    University of Missouri-Columbia, Columbia, Missouri
    Vision Sci/Vet Med & Surg,
    Mason Eye Institute, Ophthalmology,
  • Y. Yue
    University of Missouri-Columbia, Columbia, Missouri
    Molecular Microbiology and Immunology,
  • K. Zhang
    University of Missouri-Columbia, Columbia, Missouri
    Vision Sci/Vet Med & Surg,
  • D. Duan
    University of Missouri-Columbia, Columbia, Missouri
    Molecular Microbiology and Immunology,
  • Footnotes
    Commercial Relationships  B. Lei, None; Y. Yue, None; K. Zhang, None; D. Duan, None.
  • Footnotes
    Support  RPB, NIH AR-49419, MDA 4291.
Investigative Ophthalmology & Visual Science May 2008, Vol.49, 879. doi:https://doi.org/
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    • Get Citation

      B. Lei, Y. Yue, K. Zhang, D. Duan; Efficient Middle Retinal Transduction With Adeno-Associated Virus Serotype-9. Invest. Ophthalmol. Vis. Sci. 2008;49(13):879. doi: https://doi.org/.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose: : The newly developed viral vector adeno-associated virus serotype-9 (AAV-9) has shown high transduction efficiency in a variety of tissues, including the heart, liver, lung, kidney, and skeletal muscle. We studied AAV-9-mediated gene transfer in the mouse retina.

Methods: : 1×109 viral genome particles of AAV-9 AV.RSV.AP (single injection in 1 µl) were administered either subretinally or intravitreally to young (17-day-old) and adult (2 to 3-month-old) mice. Transgene expression was examined by histochemical staining at 20 and 50 days after injection.

Results: : Subretinal injection yielded widespread transduction throughout the retina. High expression was observed in the middle retina outer plexiform layer (OPL) and Müller cells in both young and adult mice. Expressions were also observed in the retinal pigment epithelium (RPE) layer, inner plexiform layer (IPL), and retinal ganglion cell (RGC) layer. Transduction was not as efficient with the intravitreal approach. No expression was observed in young mice. In adult mice, expression was localized at the injection site.

Conclusions: : Our data suggest that AAV-9 is a potent vector to deliver the therapeutic gene(s) to the whole retina. The middle retina has been a challenging structure to target by the available gene therapy vectors. Efficient AAV-9 transduction of the mid-retina OPL is particularly encouraging for developing treatments for diseases that primarily affect this layer.

Keywords: gene transfer/gene therapy • adenovirus • retina: distal (photoreceptors, horizontal cells, bipolar cells) 
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