Abstract
| Original language | English |
|---|---|
| Title of host publication | Retinal Degenerations |
| 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 | |
| Publication status | Published - 1 Dec 2003 |
| Externally published | Yes |
Publication series
| Name | Advances in Experimental Medicine and Biology |
|---|---|
| Publisher | Springer |
| Volume | 533 |
| ISSN (Print) | 0065-2598 |
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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 proceeding › Chapter › Research › peer-review
TY - CHAP
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 - Chapter
SN - 978-1-4613-4909-9
T3 - Advances in Experimental Medicine and Biology
SP - 431
EP - 438
BT - Retinal Degenerations
A2 - LaVail, Matthew M.
A2 - Hollyfield, Joe G.
A2 - Anderson, Robert E.
PB - Springer
CY - Boston
ER -