@inproceedings{cf534abe8f2349a6a2e7a908434c2a70,
title = "Assessment of rAAV-mediated gene therapy in the Rpe65-/- mouse",
abstract = "There have been several reports demonstrating the potential of virus-mediated gene delivery for use in treating congenital retinal degenerations (Bennett and Maguire, 2000; Acland et al., 2001; Campochiaro et al., 2002; Narfstrom et al., 2002). A recent report on the restoration of vision to Briard dogs marked the first successful gene therapy for blindness in a large animal model, bringing the possibility of a human retinal gene therapy trial closer (Bennett and Maguire, 2000; Narfstrom et al., 2002). We wished to pursue this potential, by developing a successful gene therapy protocol for the treatment of retinal degeneration in a small animal model. Once established, this small animal model could be used to assess the clinical benefits and limitations of using gene therapy in treating congenital retinal degeneration.",
author = "Rakoczy, \{P. Elizabeth\} and Lai, \{Chooi May\} and Yu, \{Meaghan J T\} and Daniels, \{Dru M.\} and Meliha Brankov and Rae, \{Ben C.\} and Stoddart, \{Chris W.\} and Barnett, \{Nigel L.\} and Martin-Iverson, \{Matthew T.\} and Redmond, \{T. Michael\} and Kristina Narfstrom and Xiaohuai Zhou and Constable, \{Ian J.\}",
year = "2003",
month = dec,
day = "1",
doi = "10.1007/978-1-4615-0067-4\_55",
language = "English",
isbn = "978-1-4613-4909-9",
series = "Advances in Experimental Medicine and Biology",
publisher = "Springer",
pages = "431--438",
editor = "LaVail, \{Matthew M.\} and Hollyfield, \{Joe G.\} and Anderson, \{Robert E.\}",
booktitle = "Retinal Degenerations: Mechanisms and Experimental Therapy",
address = "Germany",
note = "RD 2002: 10th International Symposium on Retinal Degenerations ; Conference date: 30-09-2002 Through 05-10-2002",
}