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

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: 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
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
EventRD 2002: 10th International Symposium on Retinal Degenerations - Burgenstock, Switzerland
Duration: 30 Sep 20025 Oct 2002

Publication series

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

Conference

ConferenceRD 2002: 10th International Symposium on Retinal Degenerations
CountrySwitzerland
CityBurgenstock
Period30/09/025/10/02

Fingerprint

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: 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).
@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 = "12",
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",

}

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 proceedingConference contributionResearchpeer-review

TY - GEN

T1 - Assessment of rAAV-mediated gene therapy in the Rpe65-/- mouse

AU - Rakoczy, P. Elizabeth

AU - Lai, Chooi May

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.

PY - 2003/12/1

Y1 - 2003/12/1

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.

UR - http://www.scopus.com/inward/record.url?scp=0347532844&partnerID=8YFLogxK

U2 - 10.1007/978-1-4615-0067-4_55

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

M3 - Conference contribution

SN - 978-1-4613-4909-9

T3 - Advances in Experimental Medicine and Biology

SP - 431

EP - 438

BT - Retinal Degenerations: Mechanisms and Experimental Therapy

A2 - LaVail, Matthew M.

A2 - Hollyfield, Joe G.

A2 - Anderson, Robert E.

PB - Springer

CY - Boston

ER -

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