Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium

@article{Meur2007RestorationOV,
  title={Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium},
  author={Guyl{\`e}ne Le Meur and Knut Stieger and Alexander D. Smith and Michel Weber and J Y Deschamps and Delphine Nivard and Alexandra Mendes-Madeira and Nathalie Provost and Yann P{\'e}r{\'e}on and Yan Ch{\'e}rel and Robin R Ali and Christian P. Hamel and Philippe Moullier and Fabienne Rolling},
  journal={Gene Therapy},
  year={2007},
  volume={14},
  pages={292-303}
}
Previous studies have tested gene replacement therapy in RPE65-deficient dogs using recombinant adeno-associated virus 2/2 (rAAV2/2), -2/1 or -2/5 mediated delivery of the RPE65 gene. They all documented restoration of dark- and light-adapted electroretinography responses and improved psychophysical outcomes. Use of a specific RPE65 promoter and a rAAV vector that targets transgene expression specifically to the RPE may, however, provide a safer setting for the long-term therapeutic expression… CONTINUE READING

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