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

Research output: Chapter in Book/Report/Conference proceedingChapterResearchpeer-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 languageEnglish
Title of host publicationRetinal Degenerations
EditorsMatthew M. LaVail, Joe G. Hollyfield, Robert E. Anderson
Place of PublicationBoston
PublisherSpringer
Chapter55
Pages431-438
Number of pages8
ISBN (Electronic)978-1-4615-0067-4
ISBN (Print)978-1-4613-4909-9
DOIs
Publication statusPublished - 1 Dec 2003
Externally publishedYes

Publication series

NameAdvances in Experimental Medicine and Biology
PublisherSpringer
Volume533
ISSN (Print)0065-2598

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Gene therapy
Genetic Therapy
Retinal Degeneration
Animals
Animal Models
Blindness
Clinical Protocols
Viruses
Restoration
Genes
Dogs

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 (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. 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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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.}",
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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. Advances in Experimental Medicine and Biology, vol. 533, Springer, Boston, pp. 431-438. 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. 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 proceedingChapterResearchpeer-review

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AU - Rakoczy, P. Elizabeth

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AU - Yu, Meaghan J T

AU - Daniels, Dru M.

AU - Brankov, Meliha

AU - Rae, Ben C.

AU - Stoddart, Chris W.

AU - Barnett, Nigel L.

AU - Martin-Iverson, Matthew T.

AU - Redmond, T. Michael

AU - Narfstrom, Kristina

AU - Zhou, Xiaohuai

AU - Constable, Ian J.

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N2 - 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.

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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M3 - Chapter

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T3 - Advances in Experimental Medicine and Biology

SP - 431

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BT - Retinal Degenerations

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A2 - Hollyfield, Joe G.

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PB - Springer

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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. Boston: Springer. 2003. p. 431-438. (Advances in Experimental Medicine and Biology). https://doi.org/10.1007/978-1-4615-0067-4_55