Assessment of rAAV-mediated gene therapy in the Rpe65-/- mouse

P. Elizabeth Rakoczy*, Chooi May Lai, Meaghan J T Yu, Dru M. Daniels, Meliha Brankov, Ben C. Rae, Chris W. Stoddart, Nigel L. Barnett, Matthew T. Martin-Iverson, T. Michael Redmond, Kristina Narfstrom, Xiaohuai Zhou, Ian J. Constable

*Corresponding author for this work

Research output: Chapter in Book/Report/Conference proceedingConference contributionResearchpeer-review

7 Citations (Scopus)


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.
Original languageEnglish
Title of host publicationRetinal Degenerations: Mechanisms and Experimental Therapy
Subtitle of host publicationProceedings of the X International Symposium on Retinal Degeneration, held September 30-0ctober 5, 2002, in Burgenstock, Switzerland
EditorsMatthew M. LaVail, Joe G. Hollyfield, Robert E. Anderson
Place of PublicationBoston
Number of pages8
ISBN (Electronic)978-1-4615-0067-4
ISBN (Print)978-1-4613-4909-9
Publication statusPublished - 1 Dec 2003
Externally publishedYes
EventRD 2002: 10th International Symposium on Retinal Degenerations - Burgenstock, Switzerland
Duration: 30 Sept 20025 Oct 2002

Publication series

NameAdvances in Experimental Medicine and Biology
ISSN (Print)0065-2598


ConferenceRD 2002: 10th International Symposium on Retinal Degenerations


Dive into the research topics of 'Assessment of rAAV-mediated gene therapy in the Rpe65-/- mouse'. Together they form a unique fingerprint.

Cite this