Abstract
Gene therapy has been reported to show potential as an alternative treatment to conventional therapy. In ocular research, recombinant adeno-associated virus (rAAV) has been chosen as a vector of interest due to its low immunogenicity, its broad host range and its ability to result in long-term transduction (Ali et al., 1997; Bennett and Maguire, 2000).
This is a preview of subscription content, log in via an institution to check access.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
Preview
Unable to display preview. Download preview PDF.
References
Ali RR, Reichel MB, Hunt DM and Bhattacharya SS (1997). Gene therapy for inherited retinal degeneration. Br J Ophthalmol 81:795–801.
Auricchio A, Kobinger G, Anand V, et al. (2001). Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum Mol Genet 10:3075–81.
Bennett J and Maguire AM (2000). Gene therapy for ocular disease. Mol Ther 1:501–5.
Boulanger A, Liu S, Henningsgaard AA, et al. (2000). The upstream region of the Rpe65 gene confers retinal pigment epithelium-specific expression in vivo and in vitro and contains critical octamer and E-box binding sites. J Biol Chem 275:31274–82.
Cavailles V, Augereau P and Rochefort H (1993). Cathepsin D gene is controlled by a mixed promoter, and estrogens stimulate only TATA-dependent transcription in breast cancer cells. Proc Natl Acad Sci U S A 90:203–7.
Flannery JG, Zolotukhin S, Vaquero MI, et al. (1997). Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc Natl Acad Sci U S A 94:6916–21.
Guy J, Qi X, Muzyczka N and Hauswirth WW (1999). Reporter expression persists 1 year after adeno-associated virus-mediated gene transfer to the optic nerve. Arch Ophthalmol 117:929–37.
Jomary C, Chatelain G, Michel D, et al. (1999). Effect of targeted expression of clusterin in photoreceptor cells on retinal development and differentiation. J Cell Sci 112:1455–64.
Loser P, Jennings GS, Strauss M and Sandig V (1998). Reactivation of the previously silenced cytomegalovirus major immediate-early promoter in the mouse liver: involvement of NFkappaB. J Virol 72:180–90.
Nicoletti A, Kawase K and Thompson DA (1998). Promoter analysis of RPE65, the gene encoding a 61-kDa retinal pigment epithelium-specific protein. Invest Ophthalmol Vis Sci 39:637–44.
Prosch S, Stein J, Staak K, et al. (1996). Inactivation of the very strong HCMV immediate early promoter by DNA CpG methylation in vitro. Biol Chem Hoppe Seyler 377:195–201.
Rakoczy PE, Sarks SH, Daw N and Constable IJ (1999). Distribution of cathepsin D in human eyes with or without age-related maculopathy. Exp Eye Res 69:367–74.
Rolling F, Shen WY, Tabarias H, et al. (1999). Evaluation of adeno-associated virus-mediated gene transfer into the rat retina by clinical fluorescence photography. Hum Gene Ther 10:641–8.
Sadowski I, Ma J, Triezenberg S and Ptashne M (1988). GAL4-VP16 is an unusually potent transcriptional activator. Nature 335:563–4.
Sanftner MLH, Abel H, Hauswirth WW and Flannery JG (2001). Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa. Mol Ther 4:622–9.
Sheikh MS, Augereau P, Chalbos D, et al. (1996). Retinoid regulation of human cathepsin D gene expression. J Steroid Biochem Mol Biol 57:283–91.
Stone D, David A, Bolognani F, et al. (2000). Viral vectors for gene delivery and gene therapy within the endocrine system. J Endocrinol 164:103–18.
Wang F, Porter W, Xing W, et al. (1997). Identification of a functional imperfect estrogen-responsive element in the 5′-promoter region of the human cathepsin D gene. Biochemistry 36:7793–801.
Weber M, Rabinowitz J, Provost N, et al. (2003). Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery. Mol Ther 7:774–81.
Author information
Authors and Affiliations
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2006 Springer Science+Business Media, Inc.
About this paper
Cite this paper
Sutanto, E.N., Zhang, D., Lai1, Y.K.Y., Shen, WY., Rakoczy, P.E. (2006). Potential Use of Cellular Promoter(s) to Target RPE in AAV-Mediated Delivery. In: Hollyfield, J.G., Anderson, R.E., LaVail, M.M. (eds) Retinal Degenerative Diseases. Advances in Experimental Medicine and Biology, vol 572. Springer, Boston, MA. https://doi.org/10.1007/0-387-32442-9_37
Download citation
DOI: https://doi.org/10.1007/0-387-32442-9_37
Publisher Name: Springer, Boston, MA
Print ISBN: 978-0-387-28464-4
Online ISBN: 978-0-387-32442-5
eBook Packages: MedicineMedicine (R0)