Delivery of adeno-associated virus vectors to the fetal retina: Impact of viral capsid proteins on retinal neuronal progenitor transduction

Enrico M. Surace, Alberto Auricchio, Samuel J. Reich, Tonia Rex, Ernest Glover, Stacey Pineles, Waixing Tang, Erin O'Connor, Arkady Lyubarsky, Andrey Savchenko, Edward N Pugh Jr, Albert M. Maguire, James M. Wilson, Jean Bennett

Research output: Contribution to journalArticle

36 Scopus citations

Abstract

The development of fetal ocular gene transfer may be useful as a therapeutic tool for the prevention of retinal genetic disorders with congenital or early clinical manifestations. In this study we explored the neural progenitor transduction patterns of adeno-associated virus (AAV) vectors following delivery to the developing retina. Recombinant vectors with the same genome carrying the enhanced green fluorescent protein (EGFP) transgene packaged in capsids of differing serotypes (serotypes 1, 2, and 5, termed AAV2/1, AAV2/2, and AAV2/5, respectively) were created. Delivery of the AAV vectors during early retinal development resulted in efficient and stable transduction of retinal progenitors. Vector surface proteins and the developmental status of the retina profoundly affected viral tropism and transgene distribution. The procedure is not detrimental to retinal development and function and therefore provides a safe delivery vehicle for potential therapeutic applications and a means of assessing the mechanisms of retina development and disease.

Original languageEnglish (US)
Pages (from-to)7957-7963
Number of pages7
JournalJournal of Virology
Volume77
Issue number14
DOIs
StatePublished - Jul 2003
Externally publishedYes

ASJC Scopus subject areas

  • Immunology

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    Surace, E. M., Auricchio, A., Reich, S. J., Rex, T., Glover, E., Pineles, S., Tang, W., O'Connor, E., Lyubarsky, A., Savchenko, A., Pugh Jr, E. N., Maguire, A. M., Wilson, J. M., & Bennett, J. (2003). Delivery of adeno-associated virus vectors to the fetal retina: Impact of viral capsid proteins on retinal neuronal progenitor transduction. Journal of Virology, 77(14), 7957-7963. https://doi.org/10.1128/JVI.77.14.7957-7963.2003