Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy

Nature Genetics

Volumn 25, Issue 3, 2000, Pages 306-310

  Ali, Robin R ^a   Sarra, Gian Marco ^a   Stephens, Clare ^a   De Alwis, Mahesh ^a   Bainbridge, James W B ^a   Munro, Peter M ^a   Fauser, Sascha ^c   Reichell, Martin B ^d   Kinnon, Christine ^b   Hunt, David M ^a   Bhattacharya, Shomi S ^a   Thrasher, Adrian J ^b  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c UNIVERSITY HOSPITAL TÜBINGEN   (Germany)

^d UNIVERSITY HOSPITAL LEIPZIG   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

PERIPHERIN; RHODOPSIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL FUNCTION; CELL INTERACTION; CELL ULTRASTRUCTURE; CONTROLLED STUDY; GENE THERAPY; GENE TRANSFER; MOUSE; NONHUMAN; PHOTORECEPTOR; PHOTOTRANSDUCTION; PRIORITY JOURNAL; PROTEIN EXPRESSION; RETINA DEGENERATION;

ANIMALS; CELL LINE; CONES (RETINA); CRICETINAE; DISEASE MODELS, ANIMAL; GENE THERAPY; INTERMEDIATE FILAMENT PROTEINS; MEMBRANE GLYCOPROTEINS; MICE; MICE, INBRED CBA; MICE, TRANSGENIC; NERVE TISSUE PROTEINS; RETINITIS PIGMENTOSA; RHODOPSIN; RODS (RETINA);

ADENO-ASSOCIATED VIRUS; ANIMALIA;

EID: 0033947697     PISSN: 10614036     EISSN: None     Source Type: Journal    
DOI: 10.1038/77068     Document Type: Article

References (18)

1. Travis, G.H., Brennan, M.B., Danielson, P.E., Kozak, C.A. & Sutcliffe, J.G. Identification of a photoreceptor-specific mRNA encoded by the gene responsible for retinal degeneration slow (rds). Nature 338, 70-73 (1989).
2. Bascom, R.A. et al. Cloning of the cDNA for a novel photoreceptor membrane protein (rom-1) identifies a disk rim protein family implicated in human retinopathies. Neuron 8, 1171-1184 (1992).
3. Molday, R.S. Peripherin/rds and rom-1: Molecular properties and role in photoreceptor cell degeneration. Prog. Ret. Eye Res. 13, 271-299 (1994).
4. Gregory-Evans, K. & Bhattacharya, S.S. Genetic blindness: Current concepts in the pathogenesis of human outer retinal dystophies. Trends Genet. 14, 103-108 (1998).
5. Sanyal, S. & Jansen, H. Absence of receptor outer segments in the retina of rds mutant mice. Neurosci. Lett. 21, 23-26 (1981).
6. Shalken, J.J., Janssen, J.J., DeGrip, W.J., Hawkins, R.K. & Sanyal, S. Immunoassay of rod visual pigment (opsin) in the eyes of rds mutant mice lacking photoreceptor outer segments. Biochim. Biophys. Acta 839, 122-126 (1985).
7. Nir, I., Agrawal, N. & Papermaster, D.S. Opsin gene expression during early and late phases of retinal degeneration in rds mice. Exp. Eye Res. 51, 257-267 (1983).
8. Chang, G.-Q., Hao, Y. & Wong, F. Apoptosis: Final common pathway of photoreceptor death in rd, rds, and rhodopsin mutant mice. Neuron 11, 595-605 (1993).
9. Portera-Cailliau, C., Sung, C.H., Nathans, J. & Adler, R. Apoptotic photoreceptor cell death in mouse models of retinitis pigmentosa. Proc. Natl Acad. Sci. USA 91, 974-978 (1994).
10. Reuter, J.H. & Sanyal, S. Development and degeneration of retina in rds mutant mice: The electroretinogram. Neurosci. Lett. 48, 231-237 (1984).
11. Travis, G.H., Groshan, K.R., Lloyd, M. & Bok, D. Complete rescue of photoreceptor dysplasia and degeneration in transgenic retinal degeneration slow (rds) mice. Neuron 9, 113-119 (1992).
12. Takahashi, M., Miyoshi, H., Verma, IM & Gage, F.H. Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer. J. Virol. 73, 7812-7816 (1999).
13. Lewin, A.S. et al. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal retinitis pigmentosa. Nature Med. 4, 967-971 (1998).
14. Boursnell, M.E. et al. A genetically inactivated herpes simplex virus type 2 (HSV-2) vaccine provides effective protection against primary and recurrent HSV-2 disease. J. Infect. Dis. 175, 16-25 (1997).
15. Zhang, X., O'Shea, H., Boursnell, M., Efstathiou, S. & Inglis, S. An efficient selection system for packaging herpes simplex virus amplicons. J. Gen. Virol. 79, 125-131 (1998).
16. Hill, T.J., Field, H.J. & Blyth, W.A. Acute and recurrent infection with herpes simplex virus in the mouse: A model for studying latency and recurrent disease. J. Gen. Virol. 28, 341-353 (1975).
17. Zhang, X. et al. High titre recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H. Hum. Gene Ther. 10, 2527-2537 (1999).
18. Ali, R.R. et al. Gene transfer into the mouse retina mediated by an adeno-associated viral vector. Hum. Mol. Genet. 5, 591-594 (1996).