95
Views
55
CrossRef citations to date
0
Altmetric
Research Article

Evaluation of recombinant adeno-associated virus as a gene transfer vector for the retina

, , , &
Pages 949-956 | Published online: 02 Jul 2009

References

  • Rosenfeld, P. J., McKusick, V. A., Amberger, J. S. and Dryja, T. P. (1994) Recent advances in the gene map of inherited eye disorders: primary hereditary diseases of the retina, choroid, and vitreous. J. Med. Genet. 31, 903–915.
  • Daiger, S., Sullivan, L. and Rodriguez, J. (1995) Correla-tion of phenotype with genotype in inherited retinal degen-eration. Behav. and Brain Sci. 18,452–467.
  • Travis, G. H., Groshan, K. R., Lloyd, M. and Bok, D. (1992) Complete rescue of photoreceptor dysplasia and degeneration in transgenic retinal degeneration slow (rds) mice. Neuron, 9, 113–119.
  • Lem, J., Flannery, J. G., Li, T., Applebury, M. L., Farber, D. B. and Simon, M. I. (1992) Retinal degeneration is res-cued in transgenic rd mice by expression of the cGMP phosphodiesterase beta subunit. Proc. Natl. Acad. Sci. USA, 89, 4422–4426.
  • Bennett, J., Wilson, J., Sun, D., Forbes, B. and Maguire, A. (1994) Adenovirus vector-mediated in vivo gene transfer into adult murine retina. Invest. Ophthalmol. Vis. Sci 35, 2535–2542.
  • Li, T., Adamian, M., Roof, D. J., Berson, E. L., Dryja, T. P., Roessler, B. J. and Davidson, B. L. (1994) In vivo transfer of a reporter gene to the retina mediated by an adenoviral vector. Invest. Ophthalmol. Vis. Sci. 35, 2543–2549.
  • Rosenfeld, M., Siegfried, W., Yoshimura, K., Yoneyama, K., Fukayama, M. and Stier, L. (1991) Adenovirus-mediated transfer of a recombinant ec1-antitrypsin gene to the lung epithelium in vivo. Science, 252, 431–434.
  • Davidson, B., Allen, E., Kozarsky, K., Wilson, J. and Roessler, B. (1993) A model system for in vivo gene trans-fer into the central nervous system using an adenovirual vector. Nat. Genet. 3, 219–223.
  • Akli, S., Caillaud, C., Vigne, E., Stratford-Perricaudet, L., Poenaru, L., Perricaudet, M., Kahn, A. and Peschanski, M. (1993) Transfer of a foreign gene into the brain using ade-novirus vectors. Nat. Genet. 3, 224–228.
  • Li, T. and Davidson, B. L. (1995) Phenotype correction in retinal pigment epithelium in murine mucopolysacchari-dosis: VII by adenovirus-mediated gene transfer. Proc. Natl. Acad. Sci. USA, 92, 7700–7704.
  • Bennett, J., Tanabe, T., Sun, D., Zeng, Y., Kjeldbye, H., Gouras, P. and Maguire, A. (1996) Photoreceptor cell res-cue in retinal degeneration (rd) mice by in vivo gene ther-apy. Nat. Med. 2, 649–654.
  • Yang, Y., Nunes, F. A., Berencsi, K., Furth, E. E., Gonczol, E. and Wilson, J. M. (1994) Cellular immunity to viral antigens limits El-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA, 91,4407–4411.
  • Dai, Y., Schwarz, E. M., Gu, D., Zhang, W. W., Sarvetnick, N. and Verma, I. M. (1995) Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Proc. Natl. Acad. Sci. USA, 92, 1401–1405.
  • Ali, R., Reichel, M., Thrasher, A., Kanuga, N., Hunt and Bhattacharya, S. (1996) In vivo gene delivery to the mouse retina. (Abstract). Invest. Ophthalmol. Vis. Sci. 37, S10.
  • Streilein, J. W. (1995) Immunological non-responsiveness and acquisition of tolerance in relation to immune privi-lege in the eye. Eye, 9, 236–240.
  • Muzyczka, N. (1992) Use of adeno-associated virus as a general transduction vector for mammalian cells. Curt: Top. Micro bio. Immunol. 158, 97–129.
  • Berns, K. I. and Linden, R. M. (1995) The cryptic life style of adeno-associated virus. Bioessays, 17, 237–245.
  • Hermonat, P. L. and Muzyczka, N. (1984) Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tis-sue culture cells. Proc. Natl. Acad. Sci. USA, 81, 6466–6470.
  • Samulski, R., Chang, L.-S. and Shenk, T. (1989) Helperfree stocks of recombinant adeno-associated viruses: nor-mal integration does not require viral gene expression. J. ViroL 63, 3822–3828.
  • Flotte, T. R., Solow, R., Owens, R. A., Afione, S., Zeitlin, P. L. and Carter, B. J. (1992) Gene expression from adeno-associated virus vectors in airway epithelial cells. Am. Resp. Cell & MoL Biol. 7,349–356.
  • Flotte, T. R., Afione, S. A., Conrad, C., McGrath, S. A., Solow, R., Oka, H., Zeitlin, P. L., Guggino, W. B. and Carter, B. J. (1993) Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. USA, 90, 10613–10617.
  • Thou, S. Z., Broxmeyer, H. E., Cooper, S., Harrington, M. A. and Srivastava, A. (1993) Adeno-associated virus 2-mediated gene transfer in murine hematopoietic progenitor cells. Exp. HematoL 21, 928–933.
  • Miller, J. L., Donahue, R. E., Sellers, S. E., Samulski, R. J., Young, N. S. and Nienhuis, A. W. (1994) Recombinant adeno-associated virus (rAAV)-mediated expression of a human gamma-globin gene in human progenitor-derived erythroid cells. Proc. Natl. Acad. Sci. USA, 91, 10183-10187. (Published Erratum appears in Proc. Natl. Acad. Sci USA, 92, 646.
  • Kaplitt, M., Leone, P., Samulski, R., Xiao, X., Pfaff, D., O'Malley, K. and During, M. (1994) Long-term gene expres-sion and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 8, 148–153.
  • Podsakoff, G., Wong, K. and Chatterjee, S. (1994) Effi-cient gene transfer into nondividing cells by adeno-associated virus-based vectors. J. ViroL 68, 5656–5666.
  • Hauswirth, W., Zolotukhin, S., Muzyczka, N. and Flan-nery, J. (1995) Adeno-associated virus delivery of an opsin promoter-driven reporter gene to the mouse and rabbit ret-ina. (Abstract). Invest. Ophthalmol. Vis. Sci 36, S845.
  • Ali, R. R., Reichel, M. B., Thrasher, A. J., Levinsky, R. J., Kinnon, C., Kanuga, N., Hunt, D. M. and Bhattacharya, S. S. (1996) Gene transfer into the mouse retina mediated by an adeno-associated viral vector. Hum. MoL Genet. 5, 591–594.
  • Zolotukhin, S., Potter, M., Hauswirth, W. W., Guy, J. and Muzyczka, N. (1996) A humanized green fluorescent pro-tein cDNA adapted for high-level expression in mamma-lian cells. J. ViroL 70, 4646–4654.
  • Russell, D., Miller, A. and Alexander, I. (1994) Adeno-associated virus vectors preferentially transduce cells in S phase. Proc. Natl. Acad. Sci. USA, 91, 8915–8919.
  • Flotte, T. R., Afione, S. A. and Zeitlin, P. L. (1994) Adeno-associated virus vector gene expression occurs in nondi-viding cells in the absence of vector DNA integration. Am. Resp. Cell & MoL Biol. 11,517–521.
  • Alexander, I., Russell, D., Spence, A. and Miller, A. (1996) Effects of gamma irradiation on the transduction of dividing and nondividing cells in brain and muscle of rats by adeno-associated virus vectors. Hum. Gene Ther 7, 841–850.
  • Russell, D. W., Alexander, I. E. and Miller, A. D. (1995) DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA, 92, 5719–5723.
  • Fisher, K. J., Gao, G. P., Weitzman, M. D., Dematteo, R., Burda, J. F. and Wilson, J. M. (1996) Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Vim. 70, 520–532.
  • Ferrari, F. K., Samulski, T., Shenk, T. and Samulski, R. J. (1996) Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. I ViroL 70, 3227–3234.
  • Travis, G. H., Brennan, M. B., Danielson, P. E., Kozak, C. A. and Sutcliffe, J. G. (1989) Identification of a photo-receptor-specific mRNA encoded by the gene responsible for retinal degeneration slow (rds). Nature, 338, 70–73.
  • Ponnazhagan, S., Mukherjee, P., Yoder, M. C., Wang, X.-S., Zhou, S. Z., Kaplan, J., Wadsworth, S. and Srivastava, A. (1997) Adeno-associated virus 2-mediated gene trans-fer in vivo: organ-tropism and expression of transduced sequences in mice. Gene. In Press.
  • Luo, F., Thou, S. Z., Cooper, S., Munshi, N. C., Boswell, H. S., Broxmeyer, H. E. and Srivastava, A. (1995) Adeno-associated virus 2-mediated gene transfer and functional expression of the human granulocyte-macrophage colony-stimulating factor. Exp. HematoL 23, 1261–1267.
  • Srivastava, A., Bruno, E., Briddell, R., Cooper, R., Srivas-tava, C., van Besien, K. and Hoffman, R. (1990) Parvovi-rus B19-induced perturbation of human megakaryocyto-poiesis in vitro. Blood, 76, 1997–2000.
  • Srivastava, C. H., Samulski, R. J., Lu, L., Larsen, S. H. and Srivastava, A. (1989) Construction of a recombinant human parvovirus B19: adeno-associated virus 2 (AAV) DNA inverted terminal repeats are functional in an AAV-B19 hybrid virus. Proc. Natl. Acad. Sci. USA, 86, 8078–8002.
  • Physicians 'Desk Reference, 50th Ed. (1996) P. 696. Med-ical Economics, Montvale, NJ, USA.
  • Beckloff, F., Lerner, H. and Frost, D. (1963) Hydroxyurea in biologic fluids: dose-concentration relationship. Cancer Chemother. Rep. 48, 57–58.
  • Rosenberg, W., Breakefield, X., DeAntonio, C. and Isac-son, 0. (1992) Authentic and artifactual detection of the E. coli lacZ gene product in the rat brain by histochemical methods. MoL Brain Res. 16, 311–315.
  • Drager, U. C. and Olsen, J. F. (1981) Ganglion cell distri-bution in the retina of the mouse. Invest. Ophthalmol. Vis. Sci. 20, 285–293.
  • Hofbauer, A. and Drager, U. C. (1985) Depth segregation of retinal ganglion cells projecting to mouse superior col-liculus. J. Comp. NeuroL 234, 465–474.
  • Baskar, J., Smith, P., Nilaver, G., Jupp, R., Hoffmann, S., Peffer, N., Tenney, D., Colberg-Poley, A., Ghazal, P. and Nelson, J. (1996) The enhancer domain of the human cytomegalovirus major immediate-early promoter deter-mines cell type-specific expression in transgenic mice. J. Viml. 70, 3207–3214.
  • Cayouette, M. and Gravel, C. (1996) Adenovirus-mediated gene transfer to retinal ganglion cells. Invest. OphthalmoL Vis. Sci. 37, 2022–2028.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.