Prospects for gene therapy

Novartis Foundation Symposium

Volumn 255, Issue , 2003, Pages 165-172

  Ali, Robin R ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 33751568670     PISSN: 15282511     EISSN: None     Source Type: Book Series    
DOI: None     Document Type: Article

References (21)

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2. Ali RR, Sarra GM, Stephens C et al 2000 Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat Genet 25:306-310
3. Apfelstedt-Sylla E, Theischen M, Ruther K et al 1995 Extensive intrafamilial and interfamilial phenotypic variation among patients with autosomal dominant retinal dystrophy and mutations in the human RDS/peripherin gene. Br J Ophthalmol 79:28-34
4. Bainbridge JWB, Mistry A, Schlichtenbrede F et al 2003 Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina. Gene Ther 10:1336-1344
5. Bennett J, Maguire AM, Cideciyan AV et al 1999 Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natl Acad Sci USA 96:9920-9925
6. Bessant DA, Ali RR, Bhattacharya SS 2001 Molecular genetics and prospects for therapy of the inherited retinal dystrophies. Curr Opin Genet Dev 11:307-316
7. Bok D, Yasumura D, Matthes MT et al 2002 Effects of adeno-associated virus-vectored ciliary neurotrophic factor on retinal structure and function in mice with a p216l rds/peripherin mutation. Exp Eye Res 74:719-735
8. Caffe AR, Soderpalm AK, Holmqvist I, van Veen T 2001 A combination of CNTF and BDNF rescues rd photoreceptors but changes rod differentiation in the presence of RPE in retinal explants. Invest Ophthalmol Vis Sci 42:275-282
9. D'Cruz PM, Yasumura D, Weir J et al 2000 Mutation of the receptor tyrosine kinase gene Mertk in the retinal dystrophic RCS rat. Hum Mol Genet 9:645-651
10. Dudus L, Anand V, Acland GM et al 1999 Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV. Vision Res 39:2545-2553
11. Gal A, Li Y, Thompson D et al 2000 Mutations in MERTK, the human orthologue of the RCS rat retinal dystrophy gene, cause retinitis pigmentosa. Nat Genet 26:270-271
12. Liang FQ, Aleman TS, Dejneka NS et al 2001 Long-term protection of retinal structure but not function using RAAV.CNTF in animal models of retinitis pigmentosa. Mol Ther 4:461-472
13. McGee Sanftner LH, Abel H, Hauswirth WW, Flannery JG 2001 Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa. Mol Ther 4:622-629
14. Sarra GM, Stephens C, de Alwis M et al 2001 Gene replacement therapy in the retinal degeneration slow (rds) mouse: the effect on retinal degeneration following partial transduction of the retina. Hum Mol Genet 10:2353-2361
15. Sarra GM, Stephens C, Schlichtenbrede FC et al 2002 Kinetics of transgene expression in mouse retina following sub-retinal injection of recombinant adeno-associated virus. Vision Res 42:541-549
16. Schlichtenbrede FC, MacNeil A, Bainbridge JWB et al 2003a Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2Rd2/Rd2 model of retinal degeneration. Gene Ther 10:523-527
17. Schlichtenbrede FC, da Cruz L, Stephens C et al 2003b Long-term evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapy. J Gene Med 5:757-764
18. Smith AJ, Schlichtenbrede FC, Tschernutter M, Bainbridge JW, Thrasher AJ, Ali RR 2003 AAV-mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa. Mol Ther 8:188-195
19. Travis GH, Sutcliffe JG, Bok D 1991 The retinal degeneration slow (rds) gene product is a photoreceptor disc membrane-associated glycoprotein. Neuron 6:61-70
20. Vollrath D, Feng W, Duncan JL et al 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
21. Wahlin KJ, Adler R, Zack DJ, Campochiaro PA 2001 Neurotrophic signaling in normal and degenerating rodent retinas. Exp Eye Res 73:693-701