Stable and efficient gene transfer into the mutant retinal pigment epithelial cells of the Mitfvit mouse using a lentiviral vector

References

• Dryja, T. P., Berson, E. L. (1995) Retinitis Pigmentosa and allied diseases: implications of genetic heterogenity. iInves. Ophtha. Vis. Sci.i b36,b 1197–1200.
   Google Scholar
• Sullivan, L. S., Daiger, S. P. (1996) Inherited retinal de-generation: exceptional genetic and clinical heterogene-ity. iMol. Med. Todayi September, 380–386.
   Google Scholar
• Bennett, J., Wilson, J., Sun, D., Forbes, B., Maguire, A. (1994) Adenovirus vector-mediated in vivo gene trans-fer into adult murine retina. iInvest. Ophthal. Vis. Sci.i b35,b 2535–2542.
   Google Scholar
• Li, T., Adamian, M., Roof, D. J., Berson, E. L., Dryja, T. P., Roessler, B. J., Davidson, B. L. (1994) In vivotransfer of a reporter gene to the retina mediated by an adenoviral vector. iInvest. Ophthal. Vis. Sci.i b35,b 2543–2549.
   Google Scholar
• Bennett, J., Tanabe, T., Sun, D., Zeng, Y., Kjeldbye, H., Gouras, P., Maguire, A. M. (1996) Photoreceptor cell rescue in retinal degeneration i(rd)i mice by iin vivoi gene therapy. iNature Med.i b2,b 649–654.
   Google Scholar
• Doering, L. C. (1994) Nervous system modification by transplants and gene transfer. iRio. Essay,i b16,b 825–831.
   Google Scholar
• Karpati, G., Lochmilller, H., Nalbantoglu, J., Durham,H. (1996) The principles of gene therapy for the ner-vous system. iTrends Neurosci,i b19,b 49–54.
   Google Scholar
• Hoffman, L. M., Maguire, A. M., Bennett, J. (1997) Cell-mediated immune response and stability of intraocular transgene expression after adenovirus-mediated delivery. iInvest. Ophthal. Vis. Sci.i b38(11),b 2224–2233.
   Google Scholar
• Kajiwara, K., Byrnes, A. P., Charlton, H. M., Wood, M.J. A., Wood, K. J. (1997) Immune responses to aden-oviral vectors during gene transfer in the brain. iHuman Gene Therapy,i b8,b 253–265.
   Google Scholar
• Majors, J. E. (1992) Retroviral vectors - strategies and applications. iVirology,i b3,b 285–295.
   Google Scholar
• Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F. H., Verma, I. M., Trono, D. (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. iScience,i b272,b 263–267.
   Google Scholar
• Naldini, L., Blömer, U., Gage, F. H., Trono, D., Verma,I. M. (1996) Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. iProc. Natl. Acad. Sci. USA,i b93,b 11382–11388.
   Google Scholar
• Blömer, U., Naldini, L., Kafri, T., Trono, D., Verma, I. M., Gage, F. H. (1997) Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. iAmer. Soc. Microbiol.i b71(9),b 6641–6649.
   Google Scholar
• Miyoshi, H., Takahashi, M., Gage, F. H., Verma, I. M. (1997) Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. iProc. Natl. Acad. Sci. USA,i b94,b 10319–10323.
   Google Scholar
• Smith, S. B. (1992) C57BL/6J-vit/vit mouse model of retinal degeneration: Light microscopic analysis andevaluation of rhodopsin levels. iExp. Eye. Res.i b55,b 903–910.
   Google Scholar
• Smith, S. B. (1995) Evidence of a difference in photo-receptor cell loss in the peripheral versus posterior re-gions of the vitiligo (C57BL/6J-mivit/mivit) mouse retina. iExp. Eye. Res.i b60,b 333–336.
   Google Scholar
• Sidman, R. L., Kosaras, B., Tang, M. (1996) Pigment epithelium and retinal phenotypes in the vitiligo, imivit,i Mutant Mouse. iInvest. Ophthal. Vis. Sci.i b37(6),b 1097–1115.
   Google Scholar
• Smith, S. B., Bora, N., McCool, D. J., Kutty, G., Wong, P., Kutty, K., Wiggert, B. (1995) Photoreceptor cells in the vitiligo mouse die by apoptosis. TRPM-2/clusterin is elevated in the neural retina, but also in the retinal pigment epithelium. iInvest. Ophthal. Vis. Sci.i b36,b 2193–2201.
   Google Scholar
• Boissy, R. E., Moellmann, G. E., Lerner, A. B. (1987) Morphology of melanocytes in hair bulbs and eyes of vitiligo mice. iAmer. J. Path.i b127,b 380–388.
   Google Scholar
• Nir, I., Ransom, N., Smith, S. B. (1995) Ultrastructural features of retinal dystrophy in mutant vitiligo mice. iExp. Eye Res.i b61,b 363–377.
   Google Scholar
• Smith, S. B., Duncan, T., Kutty, G., Kutty, R. K., Wiggert, B. (1994) Increase in retinyl palmitate concentration in eyes and livers and the concentration of interphotoreceptor retinoid-binding protein in eyes of vitiligo mutant mice. iBiochem. J.i b300,b 63–68.
   Google Scholar
• Smith, S. B., Cope, B. K., McCoy, J. R., McCool, D. J., Defoe, D. M. (1994) Reduction of phagosomes in the vitiligo mouse model of retinal degeneration. iInvest. Ophthal. Vis. Sci.i b35,b 3625–3632.
   Google Scholar
• Kosaras, B., Sidman, R. L. (1996) Phagosome number and distribution in retinal pigment epithelial cells of vitiligo mutant mice. iExp. Eye Res.i b63,b 151–158.
   Google Scholar
• Lamoreux, M. L., Boissy, R. E., Womack, J. E., Nordlund, J. J. (1992) The vit gene maps to the mi (Microphthalmia) locus of the laboratory mouse. iJ. Hered.i b83,b 435–439.
   Google Scholar
• Tang, M., Neumann, P. E., Kosaras, B., Taylor, B. A., Sidman, R. L. (1992) Vitiligo maps to mouse chromo-some 6 within or close to the mi locus. iMouse Genomei b90,b 441–443.
   Google Scholar
• Steingrimsson, E., Moore, K. J., Lamoreux, M. L., Ferre-D'Amare, A., Burley, S. K., Sanders, Zimring, D. C., Skow, L. C., Hodgkinson, C. A., Arnheiter, H., Copeland, N. G., Jenkins, N. (1994) Molecular basis of mouse imi-crophthalmia (mi)i mutations helps explain their devel-opmental and phenotypic consequences. iNature Genet-icsi b8,b 256–263.
   Google Scholar
• Moore, K. J. (1995) Insight into the imicrophthalmiai gene. iTrends In Geneticsi b11,b 442–448.
   Google Scholar
• Tang, M., Ruiz, M., Kosaras, B., Sidman, R. L. (1996) Increased Cell Gensis in Retinal Pigment Epithelium of Perinatal Vitiligo Mutant Mice. iInvest. Ophthal. Vis. Sci.i b37(6),b 1116–1124.
   Google Scholar
• Nakayama, A., Nguyen, M. T., Chen, C. C., Opdecamp, K., Hodgkinson, C. A., Arnheiter, H. (1998) Mutationsin imicrophthalmia,i the mouse homolog of the human deaf-ness gene, MITF, affect neuroepithelial and neural crest-derived melanocytes differently. iMech. of Develop.i b70,b 155–166.
   Google Scholar
• Bora, N., Conway, S., Liang, H., Smith, S. B. Early de-velopmental expression of the microphthalmia i(Miy)i gene in wild type mouse eyes and its dysregulation in the iMiri mutant allele. Develop Dynamics; in press.
   Google Scholar
• Chen, C., Okayama, H. (1987) High-efficiency transfor-mation of mammalian cells by plasmid DNA. iMol. Cell. Biol.i b7,b 2745–2752.
   Google Scholar
• Galileo, D. S., Majors, J., Horwitz, A. F., Sanes, J. R. (1992) Retrovirally introduced antisense integrin RNA inhibits neuroblast migration in vivo. iNeuron,i b9,b 1117–1131.
   Google Scholar
• O'Brien, P. J. (1993) iIn vivoi labeling of retinal compo-nents by intraocular injection. In: iMethods in Neuro-sciences,i iVolume 15: Photoreceptor Cells.i San Diego: Academic Press, Inc. 75–85.
   Google Scholar
• Smith, S. B., Titelman, R., Hamasaki, D. I. (1996) Ef-fects of basic fibroblast growth factor on the retinal de-generation of the mivit/mivit (Vitiligo) mouse: a morpho-logic and electrophysiologic study. iExp. Eye Res.i 565–577.
   PubMedGoogle Scholar
• da Cruz, L., Rakoczy, P., Perricaudet, M., Constable, I. J. (1996) Dynamics of gene transfer to retinal pigment epithelium. iInvest. Ophthal. Vis. Sci.i b37,b 2447–2454.
   Google Scholar
• Sullivan, D. M., Chung, D. C., Anglade, E., Nussenblatt,R. B., Csaky, K. G. (1996) Adenovirus-mediated gene transfer of ornithine aminotransferase in ultured human retinal pigment epithelium. iInvest. Ophthal. Vis. Sci.i b7,b 766–774.
   Google Scholar
• Schubert, C. A., Kimura, H., Spee, C., Hinton, D. R., Gordon, E. M., Anderson, W. F., Ryan, S. J. (1997) Retrovirus-mediated transfer of the suicide gene into retinal pigment epithelial cell iin vitro. Curr. Eye Res.i b16,b 656–662.
   Google Scholar
• Kido, M., Rich, K. A., Yang, G., Barron, E., Kohn, D. B., Al-Ubaidi, M. R., Blanks, J. C. (1996) Use of a retroviral vector with an internal opsin promoter to di-rect gene expression to retinal photoreceptor cells. iCurr. Eye Res.i b15,b 833–844.
   Google Scholar
• Dunaief, J. L., Kwun, R. C., Bhardwaj, N., Lopez, R., Gouras, P., Goff, S. P. (1995) Retroviral gene transfer into retinal pigment epithelial cells followed by trans-plantation into rat retina. iHuman Gene Therapy,i b6,b 1225–1229.
   Google Scholar
• Turner, D. L., Cepko, C. (1987) A common progenitor for neurons and glia persists in rat retina late in devel-opment. iNature,i b328,b 131–136.
   Google Scholar
• Turner, D. L., Snyder, E. Y., Cepko, C. (1990) Lineage-independent determination of cell type in the embryonic mouse retina. iNeuron,i b4,b 833–845.
   Google Scholar
• Blanks, J. C., Spee, C., Barron, E., Rich, K. A., Schmidt,S. (1997) Lineage study of degenerating photoreceptor cells in the irdi mouse retina. iCurr. Eye Res.i b16,b 733–737.
   Google Scholar
• McCormack, J. E., Martineau, D., DePolo, N., Maifert, S., Akvarian, L., Townsend, K., Lee, W., Irwin, M., Sajjadi, N., Jolly, D. J., Warner, J. (1997) Anti-vector immunoglobulin induced by retroviral vectors. iHuman Gene Therapy,i b8,b 123–1273.
   Google Scholar
• Koedood, M., Fichtel, A., Meier, P., Mitchell, P. J. (1995) Human cytomegalovirus (HCMV) immediate-early en-hancer/promoter specificity during embryogenesis defines target tissues of congenital HCMV infection. iJ. Virol.i b69,b 2194–2207.
   Google Scholar