This Article
Right arrow Full Text
Right arrow Full Text (PDF)
Right arrow Alert me when this article is cited
Right arrow Alert me if a correction is posted
Services
Right arrow Similar articles in this journal
Right arrow Similar articles in PubMed
Right arrow Alert me to new issues of the journal
Right arrow Download to citation manager
Right arrowReprints and Permissions
Right arrow Copyright Information
Right arrow Books from ASM Press
Right arrow MicrobeWorld
Citing Articles
Right arrow Citing Articles via HighWire
Right arrow Citing Articles via Google Scholar
Google Scholar
Right arrow Articles by Takahashi, M.
Right arrow Articles by Gage, F. H.
Right arrow Search for Related Content
PubMed
Right arrow PubMed Citation
Right arrow Articles by Takahashi, M.
Right arrow Articles by Gage, F. H.

 Previous Article  |  Next Article 

Journal of Virology, September 1999, p. 7812-7816, Vol. 73, No. 9
0022-538X/99/$04.00+0
Copyright © 1999, American Society for Microbiology. All rights reserved.

Rescue from Photoreceptor Degeneration in the rd Mouse by Human Immunodeficiency Virus Vector-Mediated Gene Transfer

Masayo Takahashi,dagger Hiroyuki Miyoshi, Inder M. Verma, and Fred H. Gage*

Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, California 92037

Received 5 February 1999/Accepted 3 June 1999

Retinitis pigmentosa (RP) is the most common inherited retinal disease, in which photoreceptor cells degenerate, leading to blindness. Mutations in the rod photoreceptor cGMP phosphodiesterase beta  subunit (PDEbeta ) gene are found in patients with autosomal recessive RP as well as in the rd mouse. We have recently shown that lentivirus vectors based on human immunodeficiency virus (HIV) type 1 achieve stable and efficient gene transfer into retinal cells. In this study, we evaluated the potential of HIV vector-mediated gene therapy for RP in the rd mouse. HIV vectors containing a gene encoding a hemagglutinin (HA)-tagged PDEbeta were injected into the subretinal spaces of newborn rd mouse eyes. One to three rows of photoreceptor nuclei were observed in the eyes for at least 24 weeks postinjection, whereas no photoreceptor cells remained in the eyes of control animals at 6 weeks postinjection. Expression of HA-tagged PDEbeta in the rescued photoreceptor cells was confirmed by two-color confocal immunofluorescence analysis using anti-HA and anti-opsin antibodies. HIV vector-mediated gene therapy appears to be a promising means for the treatment of recessive forms of inherited retinal degeneration.

* Corresponding author. Mailing address: Laboratory of Genetics, The Salk Institute for Biological Studies, 10010 N. Torrey Pines Rd., La Jolla, CA 92037. Phone: (619) 453-4100, ext. 1012. Fax: (619) 597-0824. E-mail: fgage{at}salk.edu.

dagger Present address: Department of Ophthalmology and Visual Sciences, Graduate School of Medicine, Kyoto University, Sakyo-ku, Kyoto 606, Japan.

Journal of Virology, September 1999, p. 7812-7816, Vol. 73, No. 9
0022-538X/99/$04.00+0
Copyright © 1999, American Society for Microbiology. All rights reserved.


This article has been cited by other articles:

  • Tan, M. H., Smith, A. J., Pawlyk, B., Xu, X., Liu, X., Bainbridge, J. B., Basche, M., McIntosh, J., Tran, H. V., Nathwani, A., Li, T., Ali, R. R. (2009). Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors. Hum Mol Genet 18: 2099-2114 [Abstract] [Full Text]  
  • Davis, R. J., Tosi, J., Janisch, K. M., Kasanuki, J. M., Wang, N.-K., Kong, J., Tsui, I., Cilluffo, M., Woodruff, M. L., Fain, G. L., Lin, C.-S., Tsang, S. H. (2008). Functional Rescue of Degenerating Photoreceptors in Mice Homozygous for a Hypomorphic cGMP Phosphodiesterase 6 b Allele (Pde6bH620Q). IOVS 49: 5067-5076 [Abstract] [Full Text]  
  • Pang, J.-j., Boye, S. L., Kumar, A., Dinculescu, A., Deng, W., Li, J., Li, Q., Rani, A., Foster, T. C., Chang, B., Hawes, N. L., Boatright, J. H., Hauswirth, W. W. (2008). AAV-Mediated Gene Therapy for Retinal Degeneration in the rd10 Mouse Containing a Recessive PDE{beta} Mutation. IOVS 49: 4278-4283 [Abstract] [Full Text]  
  • Sasahara, M., Otani, A., Oishi, A., Kojima, H., Yodoi, Y., Kameda, T., Nakamura, H., Yoshimura, N. (2008). Activation of Bone Marrow-Derived Microglia Promotes Photoreceptor Survival in Inherited Retinal Degeneration. Am. J. Pathol. 172: 1693-1703 [Abstract] [Full Text]  
  • DeMarco, P. J. Jr, Yarbrough, G. L., Yee, C. W., McLean, G. Y., Sagdullaev, B. T., Ball, S. L., McCall, M. A. (2007). Stimulation via a Subretinally Placed Prosthetic Elicits Central Activity and Induces a Trophic Effect on Visual Responses. IOVS 48: 916-926 [Abstract] [Full Text]  
  • Peeters, L., Sanders, N. N., Braeckmans, K., Boussery, K., Van de Voorde, J., De Smedt, S. C., Demeester, J. (2005). Vitreous: A Barrier to Nonviral Ocular Gene Therapy. IOVS 46: 3553-3561 [Abstract] [Full Text]  
  • Gauthier, R., Joly, S., Pernet, V., Lachapelle, P., Di Polo, A. (2005). Brain-Derived Neurotrophic Factor Gene Delivery to Muller Glia Preserves Structure and Function of Light-Damaged Photoreceptors. IOVS 46: 3383-3392 [Abstract] [Full Text]  
  • Akagi, T., Akita, J., Haruta, M., Suzuki, T., Honda, Y., Inoue, T., Yoshiura, S., Kageyama, R., Yatsu, T., Yamada, M., Takahashi, M. (2005). Iris-Derived Cells from Adult Rodents and Primates Adopt Photoreceptor-Specific Phenotypes. IOVS 46: 3411-3419 [Abstract] [Full Text]  
  • Doi, K., Kong, J., Hargitai, J., Goff, S. P., Gouras, P. (2004). Transient Immunosuppression Stops Rejection of Virus-Transduced Enhanced Green Fluorescent Protein in Rabbit Retina. J. Virol. 78: 11327-11333 [Abstract] [Full Text]  
  • Liu, S.-L., Halbert, C. L., Miller, A. D. (2004). Jaagsiekte Sheep Retrovirus Envelope Efficiently Pseudotypes Human Immunodeficiency Virus Type 1-Based Lentiviral Vectors. J. Virol. 78: 2642-2647 [Abstract] [Full Text]  
  • Haruta, M., Sasai, Y., Kawasaki, H., Amemiya, K., Ooto, S., Kitada, M., Suemori, H., Nakatsuji, N., Ide, C., Honda, Y., Takahashi, M. (2004). In Vitro and In Vivo Characterization of Pigment Epithelial Cells Differentiated from Primate Embryonic Stem Cells. IOVS 45: 1020-1025 [Abstract] [Full Text]  
  • Croyle, M. A., Callahan, S. M., Auricchio, A., Schumer, G., Linse, K. D., Wilson, J. M., Brunner, L. J., Kobinger, G. P. (2004). PEGylation of a Vesicular Stomatitis Virus G Pseudotyped Lentivirus Vector Prevents Inactivation in Serum. J. Virol. 78: 912-921 [Abstract] [Full Text]  
  • Nour, M., Quiambao, A. B., Peterson, W. M., Al-Ubaidi, M. R., Naash, M. I. (2003). P2Y2 Receptor Agonist INS37217 Enhances Functional Recovery after Detachment Caused by Subretinal Injection in Normal and rds Mice. IOVS 44: 4505-4514 [Abstract] [Full Text]  
  • Wu, W.-C., Lai, C.-C., Chen, S.-L., Xiao, X., Chen, T.-L., Tsai, R. J.-F., Kuo, S.-W., Tsao, Y.-P. (2002). Gene Therapy for Detached Retina by Adeno-Associated Virus Vector Expressing Glial Cell Line-Derived Neurotrophic Factor. IOVS 43: 3480-3488 [Abstract] [Full Text]  
  • Sandrin, V., Boson, B., Salmon, P., Gay, W., Negre, D., Le Grand, R., Trono, D., Cosset, F.-L. (2002). Lentiviral vectors pseudotyped with a modified RD114 envelope glycoprotein show increased stability in sera and augmented transduction of primary lymphocytes and CD34+ cells derived from human and nonhuman primates. Blood 100: 823-832 [Abstract] [Full Text]  
  • Strettoi, E., Porciatti, V., Falsini, B., Pignatelli, V., Rossi, C. (2002). Morphological and Functional Abnormalities in the Inner Retina of the rd/rd Mouse. J. Neurosci. 22: 5492-5504 [Abstract] [Full Text]  
  • Vorburger, S. A., Hunt, K. K. (2002). Adenoviral Gene Therapy. The Oncologist 7: 46-59 [Abstract] [Full Text]  
  • Auricchio, A., Kobinger, G., Anand, V., Hildinger, M., O'Connor, E., Maguire, A. M., Wilson, J. M., Bennett, J. (2001). Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum Mol Genet 10: 3075-3081 [Abstract] [Full Text]  
  • Vollrath, D., Feng, W., Duncan, J. L., Yasumura, D., D'Cruz, P. M., Chappelow, A., Matthes, M. T., Kay, M. A., LaVail, M. M. (2001). Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk. Proc. Natl. Acad. Sci. USA 10.1073/pnas.221364198v1 [Abstract] [Full Text]  
  • Kordower, J. H., Emborg, M. E., Bloch, J., Ma, S. Y., Chu, Y., Leventhal, L., McBride, J., Chen, E.-Y., Palfi, S., Roitberg, B. Z., Brown, W. D., Holden, J. E., Pyzalski, R., Taylor, M. D., Carvey, P., Ling, Z., Trono, D., Hantraye, P., Déglon, N., Aebischer, P. (2000). Neurodegeneration Prevented by Lentiviral Vector Delivery of GDNF in Primate Models of Parkinson's Disease. Science 290: 767-773 [Abstract] [Full Text]  
  • Yvert, G., Lindenberg, K. S., Picaud, S., Landwehrmeyer, G. B., Sahel, J.-A., Mandel, J.-L. (2000). Expanded polyglutamines induce neurodegeneration and trans-neuronal alterations in cerebellum and retina of SCA7 transgenic mice. Hum Mol Genet 9: 2491-2506 [Abstract] [Full Text]  
  • LaVail, M. M., Yasumura, D., Matthes, M. T., Drenser, K. A., Flannery, J. G., Lewin, A. S., Hauswirth, W. W. (2000). Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy. Proc. Natl. Acad. Sci. USA 10.1073/pnas.210319397v1 [Abstract] [Full Text]  
  • Hauswirth, W. W., Beaufrere, L. (2000). Ocular Gene Therapy: Quo Vadis?. IOVS 41: 2821-2826 [Full Text]  
  • Buchschacher, G. L. Jr, Wong-Staal, F. (2000). Development of lentiviral vectors for gene therapy for human diseases. Blood 95: 2499-2504 [Abstract] [Full Text]  
  • An, D. S., Wersto, R. P., Agricola, B. A., Metzger, M. E., Lu, S., Amado, R. G., Chen, I. S. Y., Donahue, R. E. (2000). Marking and Gene Expression by a Lentivirus Vector in Transplanted Human and Nonhuman Primate CD34+ Cells. J. Virol. 74: 1286-1295 [Abstract] [Full Text]  
  • LaVail, M. M., Yasumura, D., Matthes, M. T., Drenser, K. A., Flannery, J. G., Lewin, A. S., Hauswirth, W. W. (2000). Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy. Proc. Natl. Acad. Sci. USA 97: 11488-11493 [Abstract] [Full Text]  
  • Vollrath, D., Feng, W., Duncan, J. L., Yasumura, D., D'Cruz, P. M., Chappelow, A., Matthes, M. T., Kay, M. A., LaVail, M. M. (2001). Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk. Proc. Natl. Acad. Sci. USA 98: 12584-12589 [Abstract] [Full Text]  


Copyright © 2009 by the American Society for Microbiology.   For an alternate route to Journals.ASM.org, visit: http://intl-journals.asm.org | More Info»