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

doi:10.1007/978-1-4615-0067-4_55

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 proceeding › Conference contribution › Research › peer-review

6 Citations (Scopus)

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.

Original language	English
Title of host publication	Retinal Degenerations: Mechanisms and Experimental Therapy
Subtitle of host publication	Proceedings of the X International Symposium on Retinal Degeneration, held September 30-0ctober 5, 2002, in Burgenstock, Switzerland
Editors	Matthew M. LaVail, Joe G. Hollyfield, Robert E. Anderson
Place of Publication	Boston
Publisher	Springer
Chapter	55
Pages	431-438
Number of pages	8
ISBN (Electronic)	978-1-4615-0067-4
ISBN (Print)	978-1-4613-4909-9
DOIs	https://doi.org/10.1007/978-1-4615-0067-4_55
Publication status	Published - 1 Dec 2003
Externally published	Yes
Event	RD 2002: 10th International Symposium on Retinal Degenerations - Burgenstock, Switzerland Duration: 30 Sep 2002 → 5 Oct 2002

Publication series

Name	Advances in Experimental Medicine and Biology
Publisher	Springer
Volume	533
ISSN (Print)	0065-2598

Conference

Conference	RD 2002: 10th International Symposium on Retinal Degenerations
Country	Switzerland
City	Burgenstock
Period	30/09/02 → 5/10/02

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Cite this

Rakoczy, P. E., Lai, C. M., Yu, M. J. T., Daniels, D. M., Brankov, M., Rae, B. C., ... Constable, I. J. (2003). Assessment of rAAV-mediated gene therapy in the Rpe65-/- mouse. In M. M. LaVail, J. G. Hollyfield, & R. E. Anderson (Eds.), Retinal Degenerations: Mechanisms and Experimental Therapy: Proceedings of the X International Symposium on Retinal Degeneration, held September 30-0ctober 5, 2002, in Burgenstock, Switzerland (pp. 431-438). (Advances in Experimental Medicine and Biology; Vol. 533). Boston: Springer. https://doi.org/10.1007/978-1-4615-0067-4_55

Rakoczy, P. Elizabeth ; Lai, Chooi May ; Yu, Meaghan J T ; Daniels, Dru M. ; Brankov, Meliha ; Rae, Ben C. ; Stoddart, Chris W. ; Barnett, Nigel L. ; Martin-Iverson, Matthew T. ; Redmond, T. Michael ; Narfstrom, Kristina ; Zhou, Xiaohuai ; Constable, Ian J. / Assessment of rAAV-mediated gene therapy in the Rpe65-/- mouse. Retinal Degenerations: Mechanisms and Experimental Therapy: Proceedings of the X International Symposium on Retinal Degeneration, held September 30-0ctober 5, 2002, in Burgenstock, Switzerland . editor / Matthew M. LaVail ; Joe G. Hollyfield ; Robert E. Anderson. Boston : Springer, 2003. pp. 431-438 (Advances in Experimental Medicine and Biology).

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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",

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language = "English",

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series = "Advances in Experimental Medicine and Biology",

publisher = "Springer",

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Rakoczy, PE, Lai, CM, Yu, MJT, Daniels, DM, Brankov, M, Rae, BC, Stoddart, CW, Barnett, NL, Martin-Iverson, MT, Redmond, TM, Narfstrom, K, Zhou, X & Constable, IJ 2003, Assessment of rAAV-mediated gene therapy in the Rpe65-/- mouse. in MM LaVail, JG Hollyfield & RE Anderson (eds), Retinal Degenerations: Mechanisms and Experimental Therapy: Proceedings of the X International Symposium on Retinal Degeneration, held September 30-0ctober 5, 2002, in Burgenstock, Switzerland . Advances in Experimental Medicine and Biology, vol. 533, Springer, Boston, pp. 431-438, RD 2002: 10th International Symposium on Retinal Degenerations, Burgenstock, Switzerland, 30/09/02. https://doi.org/10.1007/978-1-4615-0067-4_55

Assessment of rAAV-mediated gene therapy in the Rpe65-/- mouse. / Rakoczy, P. Elizabeth; Lai, Chooi May; Yu, Meaghan J T; Daniels, Dru M.; Brankov, Meliha; Rae, Ben C.; Stoddart, Chris W.; Barnett, Nigel L.; Martin-Iverson, Matthew T.; Redmond, T. Michael; Narfstrom, Kristina; Zhou, Xiaohuai; Constable, Ian J.

Retinal Degenerations: Mechanisms and Experimental Therapy: Proceedings of the X International Symposium on Retinal Degeneration, held September 30-0ctober 5, 2002, in Burgenstock, Switzerland . ed. / Matthew M. LaVail; Joe G. Hollyfield; Robert E. Anderson. Boston : Springer, 2003. p. 431-438 (Advances in Experimental Medicine and Biology; Vol. 533).

Research output: Chapter in Book/Report/Conference proceeding › Conference contribution › Research › peer-review

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AU - Brankov, Meliha

AU - Rae, Ben C.

AU - Stoddart, Chris W.

AU - Barnett, Nigel L.

AU - Martin-Iverson, Matthew T.

AU - Redmond, T. Michael

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AB - 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.

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Rakoczy PE, Lai CM, Yu MJT, Daniels DM, Brankov M, Rae BC et al. Assessment of rAAV-mediated gene therapy in the Rpe65-/- mouse. In LaVail MM, Hollyfield JG, Anderson RE, editors, Retinal Degenerations: Mechanisms and Experimental Therapy: Proceedings of the X International Symposium on Retinal Degeneration, held September 30-0ctober 5, 2002, in Burgenstock, Switzerland . Boston: Springer. 2003. p. 431-438. (Advances in Experimental Medicine and Biology). https://doi.org/10.1007/978-1-4615-0067-4_55

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10.1007/978-1-4615-0067-4_55

Link to publication in Scopus