Simian lentiviral vector-mediated retinal gene transfer of pigment epithelium-derived factor protects retinal degeneration and electrical defect in Royal College of Surgeons rats

Gene Therapy

Volumn 10, Issue 17, 2003, Pages 1503-1511

  Miyazaki, M ^a,b   Ikeda, Y ^a,b   Yonemitsu, Y ^a   Goto, Y ^b   Sakamoto, T ^c   Tabata, T ^d   Ueda, Y ^d   Hasegawa, M ^d   Tobimatsu, S ^b   Ishibashi, T ^b   Sueishi, K ^a  

^a KYUSHU UNIVERSITY   (Japan)

^b KYUSHU UNIVERSITY   (Japan)

^c KAGOSHIMA UNIVERSITY   (Japan)

^d DNAVEC Research Inc   (Japan)

Author keywords

Apoptosis; Pigment epithelium derived factor; Retinitis pigmentosa; Royal College of Surgeons rats; Simain immunodeficiency virus

Indexed keywords

BETA GALACTOSIDASE; LENTIVIRUS VECTOR; NEUROTROPHIC FACTOR; PIGMENT EPITHELIUM DERIVED FACTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; APOPTOSIS; CONTROLLED STUDY; DRUG MECHANISM; GENE EXPRESSION REGULATION; GENE TARGETING; GENE THERAPY; HISTOLOGY; NEUROPROTECTION; NICK END LABELING; NONHUMAN; PHOTORECEPTOR CELL; PHOTOSENSITIVITY; PIGMENT EPITHELIUM; PRIORITY JOURNAL; RAT; RETINA DEGENERATION; RETINITIS PIGMENTOSA; REVIEW; SIMIAN IMMUNODEFICIENCY VIRUS; TISSUE SPECIFICITY; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; APOPTOSIS; ELECTRORETINOGRAPHY; EYE PROTEINS; GENE THERAPY; GENETIC VECTORS; IMMUNOHISTOCHEMISTRY; IN SITU NICK-END LABELING; NERVE GROWTH FACTORS; PROTEINS; RATS; RATS, INBRED STRAINS; RETINA; RETINITIS PIGMENTOSA; SERPINS; SIMIAN IMMUNODEFICIENCY VIRUS; TRANSDUCTION, GENETIC;

ANIMALIA; LENTIVIRUS; RNA VIRUSES; SIMIAE; SIMIAN IMMUNODEFICIENCY VIRUS;

EID: 0042025129     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302028     Document Type: Review

References (48)

1. Pagon RA. Retinitis pigmentosa. Surv Ophthalmol 1988; 33: 137-177.
2. Wong P. Apoptosis, retinitis pigmentosa, and degeneration. Biochem Cell Biol 1994; 72: 489-498.
3. Dryja TP et al. A point mutation of the rhodopsin gene in one form of retinitis pigmentosa. Nature 1990; 343: 364-366.
4. Kajiwara K et al. Mutations in the human retinal degeneration slow gene in autosomal dominant retinitis pigmentosa. Nature 1991; 354: 480-483.
5. Cotran PR et al. Genetic analysis of patients with retinitis pigmentosa using a cloned cDNA probe for the human gamma subunit of cyclic GMP phosphodiesterase. Exp Eye Res 1991; 53: 557-564.
6. Bennet J et al. Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy. Nat Med 1996; 2: 649-654.
7. Cayouette M, Gravel C. Adenovirus-mediated gene transfer of ciliary neurotrophic factor can prevent photoreceptor degeneration in the retinal degeneration (rd) mouse. Hum Gene Ther 1997; 8: 423-430.
8. Lewin AS et al. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Nat Med 1998; 4: 967-971.
9. Takahashi M et al. Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer. J Virol 1999; 73: 7812-7816.
10. Ali RR et al. Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat Genet 2000; 25: 306-310.
11. Liang FQ et al. Long-term protection of retinal structure but not function using RAAV. CNTF in animal models of retinitis pigmentosa. Mol Ther 2001; 4: 461-472.
12. Naldini L et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-267.
13. Miyoshi H et al. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. Proc Natl Acad Sci USA 1997; 94: 10319-10323.
14. Benette J et al. Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natl Acad Sci USA 1999; 96: 9920-9925.
15. Vollrath D et al. Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk. Proc Natl Acad Sci USA 2001; 98: 12584-12589.
16. Jomary C et al. Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration. Gene Therapy 1997; 4: 683-690.
17. Sarra GM et al. Gene replacement therapy in the retinal degeneration slow (rds) mouse: the effect on retinal degeneration following partial transduction of the retina. Hum Mol Genet 2001; 10: 2353-2361.
18. Lavail MM et al. Ribozyme rescue of photoreceptor cells in P23H transgenic rats: long-term survival and late-stage therapy. Proc Natl Acad Sci USA 2000; 97: 11488-11493.
19. Nir I et al. Expression of Bcl-2 protects against photoreceptor degeneration in retinal degeneration slow (rds) mice. J Neurosci 2000; 20: 2150-2154.
20. Akimoto M et al. Adenovirally expressed basic fibroblast growth factor rescues photoreceptor cells in RCS rats. Invest Ophthalmol Vis Sci 1999; 40: 273-279.
21. Nakajima T et al. Development of novel simian immunodeficiency virus vectors carrying a dual gene expression system. Hum Gene Ther 2000; 11: 1863-1874.
22. Ikeda Y et al. Long-term histological and functional analysis for Simian Immunodeficiency virus (SIV)-based lentiviral vector-mediated intraocular gene transfer in adult rats. Gene Therapy 2003 (in press).
23. Tombran-Tink J, Johnson LV. Neuronal differentiation of retinoblastoma cells induced by medium conditioned by human RPE cells. Invest Ophthalmol Vis Sci 1989; 30: 1700-1707.
24. Ortego J et al. Gene expression of the neurotrophic pigment epithelium-derived factor in the human ciliary epithelium. Synthesis and secretion into the aqueous humor. Invest Ophthalmol Vis Sci 1996; 37: 2759-2767.
25. Tombran-Tink J et al. Expression, secretion, and age-related downregulation of pigment epithelium-derived factor, a serpin with neurotrophic activity. J Neurosci 1995; 15: 4992-5003.
26. Alberdi E et al. Pigment epithelium-derived factor (PEDF) binds to glycosaminoglycans: analysis of the binding site. Biochemistry 1998; 37:10643-10652.
27. Taniwaki T et al. Pigment epithelium-derived factor is a survival factor for cerebellar granule cells in culture. J Neurochem 1995; 64: 2509-2517.
28. Taniwaki T et al. Pigment epithelium-derived factor protects cultured cerebellar granule cells against glutamate-induced neurotoxicity. J Neurochem 1997; 68: 26-32.
29. Araki T et al. Pigment epithelium-derived factor (PEDF) differentially protects immature but not mature cerebellar granule cells against apoptotic cell death. J Neurosci Res 1998; 53: 7-15.
30. Houenou LJ, et al. Pigment epithelium-derived factor promotes the survival and differentiation of developing spinal motor neurons. J Comp Neurol 1999; 412: 506-514.
31. Bilak MM et al. Pigment epithelium-derived factor (PEDF) protects motor neurons from chronic glutamate-mediated neurodegeneration. J Neuropathol Exp Neurol 1999; 58: 719-728.
32. Decoster MA et al. Neuroprotection by pigment epithelial-derived factor against glutamate toxicity in developing primary hippocampal neurons. J Neurosci Res 1999; 56: 604-610.
33. Cayouette M et al. Pigment epithelium-derived factor delays the death of photoreceptors in mouse models of inherited retinal degenerations. Neurobiol Dis 1999; 6: 523-532.
34. Dawson DW et al. Pigment epithelium-derived factor: a potent inhibitor of angiogenesis. Science 1999; 285: 245-248.
35. Stellmach V et al. Prevention of ischemia-induced retinopathy by the natural ocular antiangiogenic agent pigment epithelium-derived factor. Proc Natl Acad Sci USA 2001; 98: 2593-2597.
36. Volpert OV et al. Inducer-stimulated Fas targets activated endothelium for destruction by anti-angiogenic thrombospondin-1 and pigment epithelium-derived factor. Nat Med 2002; 8: 349-357.
37. D'cruz PM et al. Mutation of the receptor tyrosine kinase gene Mertk in the retinal dystrophic RCS rat. Hum Mol Genet 2000; 9: 645-651.
38. Gal A et al. Mutations in MERTK, the human orthologue of the RCS rat retinal dystrophy gene, cause retinitis pigmentosa. Nat Genet 2002; 26: 270-271.
39. Lau D et al. Retinal degeneration is slowed in transgenic rats by AAV-mediated delivery of FGF-2. Invest Ophthalmol Vis Sci 2000; 41: 3622-3633.
40. Cayouette M et al. Intraocular gene transfer of ciliary neurotrophic factor prevents death and increases responsiveness of rod photoreceptors in the retinal degeneration slow mouse. J Neurosci 1998; 18: 9282-9293.
41. Liang FQ et al. AAV-mediated delivery of ciliary neurotrophic factor prolongs photoreceptor survival in the rhodopsin knockout mouse. Mol Ther 2001; 3: 241-248.
42. LaVail MM et al. Multiple growth factors, cytokines, and neurotrophins rescue photoreceptors from the damaging effects of constant light. Proc Natl Acad Sci USA 1992; 89:11249-11253.
43. Wahlin KJ et al. Neurotrophic factors cause activation of intracellular signaling pathways in Muller cells and other cells of the inner retina, but not photoreceptors. Invest Ophthalmol Vis Sci 2000; 41: 927-936.
44. Aymerich MS et al. Evidence for pigment epithelium-derived factor receptors in the neural retina. Invest Ophthalmol Vis Sci 2001; 42: 3287-3293.
45. Candille SI et al. Localization of the mouse nob (no b-wave) gene to the centromeric region of the X chromosome. Invest Ophthalmol Vis Sci 1999; 40: 2748-2751.
46. Hsu SM, Soban E. Color modification of diaminobenzidine (DAB) precipitation by metallic ions and its application for double immunohistochemistry. J Histochem Cytochem 1982; 30: 1079-1082.
47. Goto Y et al. Magnesium deficiency differentially affects the retina and visual cortex of intact rats. J Nutr 2001; 131: 2378-2381.
48. Hisatomi T et al. Critical role of photoreceptor apoptosis in functional damage after retinal detachment. Curr Eye Res 2002; 24: 161-172.