Retinal degeneration and RPE transplantation in RPE65-/- mice

Investigative Ophthalmology and Visual Science

Volumn 43, Issue 10, 2002, Pages 3307-3311

  Gouras, Peter ^a   Kong, Jian ^a   Tsang, Stephen H ^a  

^a College of Physicians and Surgeons ^*  (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADOLESCENT; ANIMAL CELL; ARTICLE; CELL TRANSPLANTATION; CONTROLLED STUDY; ELECTRORETINOGRAPHY; HISTOLOGY; MOUSE; NONHUMAN; PHOTORECEPTOR; PIGMENT EPITHELIUM; PRIORITY JOURNAL; RETINA DEGENERATION;

ANIMALS; CARRIER PROTEINS; ELECTRORETINOGRAPHY; EYE PROTEINS; GENE DELETION; MICE; MICE, KNOCKOUT; PHOTORECEPTORS; PIGMENT EPITHELIUM OF EYE; PROTEINS; REFERENCE VALUES; RETINA; RETINAL DEGENERATION;

EID: 0036784952     PISSN: 01460404     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (32)

1. Gu SM, Thompson DA, Srikumari CRS, et al. Mutations in RPE65 cause autosomal recessive childhood-onset severe retinal dystrophy. Nat Genet. 1997;17:194-197.
2. Marhlens F, Griffoin JM, Bareil C, et al. Mutations in RPE65 cause Leber's congenital amaurosis. Nat Genetics, 1997; 17:139-141.
3. Marhlens F, Griffoin JM, Bareil C, Arnaud B, Claustres M, Hamel CP. Autosomal recessive retinal dystrophy associated with two novel mutations in the RPE65 gene. Eur J Hum Genet. 1998;6:527-531.
4. Morimura H, Fishman GA, Grover GA, Fulton AB, Berson EL, Dryja TP. Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber's congenital amaurosis. Proc Nat Acad Sci USA. 1998;95:3088-3093.
5. Perrault I, Rozet JM, Ghazi I, et al. Different functional outcome of RetGC1 and RPE65 gene mutations in Leber's congenital amaurosis. Am J Human Genet. 1999;64:1225-1228.
6. Lorenz B, Gyurus P, Preising M, et al. Early-onset, severe rod-cone dystrophy in young children with RPE65 mutations. Invest Ophthalmol Vis Sci. 2000;41:2735-2742.
7. Lotery AJ, Namperumalsamy P, Jacobson SG, et al. Mutation analysis of 3 genes in patients with Leber's congenital amaurosis. Arch Ophthalmol. 2000;118:538-543.
8. Thompson D, Gyurus P, Fleischer L, et al. Genetics and phenotypes of RPE65 mutations in inherited retinal degenerations. Invest Ophthalmol Vis Sci. 2000;41:4293-4299.
9. Hamel CP, Tsilou E, Pfeffer B, Hooks JJ, Detrick B, Redmond TM. Molecular cloning and expression of RPE65, a novel retinal pigment epithelium-specific microsomal protein that is post-transcriptionally regulated in vitro. J Biol Chem. 1993;268:15751-15757.
10. Nicoletti A, Wong DJ, Kawase K, et al. Molecular characterization of the human gene encoding an abundant 61 kDa protein specific to the retinal epithelium. Hum Mol Genet. 1995;4:641-649.
11. Redmond TM, Yu S, Lee E, et al. Rpe65 is necessary for production of 11-cis vitamin A in the retinal visual cycle. Nat Genetics. 1998; 20:344-353.
12. Veske A, Nilsson SD, Narfstrom K, Gal A. Retinal dystrophy of Swedish Briard/Briard-beagle dogs is due to a 4-bp deletion in RPE65. Genomics. 1999;57:57-61.
13. Narfstrom K. Retinal dystrophy or "congenital stationary night blindness" in the Briard dog. Vet Ophthalmol. 1999;2:75-76.
14. Wrigstad A, Nilsson SEG, Narfstrom K. Ultrastructural changes of the retina and the retinal pigment epithelium in Briard dogs with hereditary congenital night blindness and partial day blindness. Exp Eye Res. 1992 55;805-818.
15. Van Hooser JP, Aleman TS, He YG, et al. Rapid restoration of visual pigment and function with oral retinoid in a mouse model of childhood blindness. Proc Natl Acad Sci USA. 2000;97:8623-8628.
16. Ackland GM, Aguirre GD, Ray J, et al. Gene therapy restores vision in a canine model of childhood blindness. Nat Genet. 2001;28:92-95.
17. Berglin L, Gouras P, Sheng Y, et al. Tolerance of human fetal pigment epithelium xenografts in monkey retina. Graefes Arch Clin Exp Ophthalmol. 1997 235:103-110.
18. LaVail MM, Li L, Turner JE, Yasumura D. Retinal pigment epithelial cell transplantation in RCS rats: normal metabolism in rescued photoreceptors. Exp Eye Res. 1992;55:555-562.
19. Yamamoto SDJ, Gouras P, Kjeldbye H. Retinal pigment epithelial transplants and retinal function in RCS rats. Invest Ophthalmol Vis Sci. 1993;34:3068-3075.
20. Coffey PJ, Girman S. Wang SM, et al. Long term preservation of cortically dependent visual function in RCS rats by transplantation. Nat Neurosci. 2002;5:53-56.
21. Algvere PV, Gouras P, Dafgard Kopp E. Long-term outcome of RPE allografts in non-immunosuppressed patients with AMD. Eur J Ophthalmol. 1999;9:217-230.
22. Wenkel H, Streilein JW. Evidence that retinal pigment epithelium functions as an immune privileged tissue. Invest Ophthalmol Vis Sci. 2000;41:3467-3473.
23. He S, Wang HM, Ogden TE, Ryan SJ. Transplantation of cultured human retinal pigment epithelium into rabbit subretina. Graefes Arch Clin Exp Ophthalmol. 1993;231:737-742.
24. Sheng Y, Gouras P, Cao H, et al. Patch transplants of human fetal retinal pigment epithelium in rabbit and monkey retina. Invest Ophthalmol Vis Sci. 1995;36:381-390.
25. Crafoord S, Algvere P, Seregard S, Kopp ED. Long-term outcome of RPE allografts to the subretinal space of rabbits. Acta Ophthalmol Scand. 1999;77:247-254.
26. Crafoord S, Algvere PV, Kopp ED, Seregard S. Cyclosporine treatment of RPE allografts in the rabbit subretinal space. Acta Ophthalmol Scand. 2000;78:122-129.
27. Wang H, Leonard DS, Castellarin AA, et al. Short-term study of allogeneic retinal pigment epithelium transplants onto debrided Bruch's membrane. Invest Ophthalmol Vis Sci. 2001;42:2990-2999.
28. Del Priore L, Tezel TH. Reattachment rate of human retinal pigment epithelium to layers of human Bruch's membrane. Arch Ophthalmol. 1999;116:335-341.
29. Zhang X, Bok D. Transplantation of retinal pigment epithelial cells and immune response in the subretinal space. Invest Ophthalmol Vis Sci. 1998;39:1021-1027.
30. Seeliger M, Grimm C, Stahlberg F, et al. New views on RPE65 deficiency: the rod system is the source of vision in a mouse model of Leber's amaurosis. Nat Genet. 2001;29:70-74.
31. -/- mice. Vision Res. 2001;41:2425-2433.
32. Wrigstad A. Hereditary dystrophy of the retina and the retinal pigment epithelium in a strain of Briard dogs: Linkoping, Sweden; Linkoping University Medical Dissertations No. 423; 1994.