Nonviral ocular gene transfer

Gene Therapy

Volumn 12, Issue 10, 2005, Pages 843-851

  Kachi, S ^a   Oshima, Y ^a   Esumi, N ^a   Kachi, M ^a   Rogers, B ^a   Zack, D J ^a   Campochiaro, P A ^a  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Electroporation; Eye; Lipofection; Ocular gene therapy; Retinal pigmented epithelium

Indexed keywords

BETA GALACTOSIDASE; LIPOFECTAMINE; LIPOPLEX; PLASMID DNA;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CHOROID DISEASE; CONTROLLED STUDY; CULTURE MEDIUM; CYTOMEGALOVIRUS; DATA ANALYSIS; ELECTROPORATION; ELECTRORETINOGRAPHY; EPITHELIUM CELL; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; HISTOCHEMISTRY; IN VIVO STUDY; MORPHOLOGY; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PHOTORECEPTOR; PIGMENT EPITHELIUM; PLASMID; PRIORITY JOURNAL; PROMOTER REGION; RETINA DISEASE; RETINA INJURY; RETINA MACULA VITELLIFORM DEGENERATION; TISSUE SPECIFICITY; TRANSGENE; VISUAL SYSTEM;

ANIMALS; ELECTROPORATION; ELECTRORETINOGRAPHY; EYE DISEASES; GENE EXPRESSION; GENE THERAPY; HISTOCYTOCHEMISTRY; INJECTIONS; LAC OPERON; LIPIDS; MICE; MICE, INBRED BALB C; OCULOMOTOR MUSCLES; PIGMENT EPITHELIUM OF EYE; PLASMIDS; RETINAL GANGLION CELLS; TIME FACTORS;

CUCUMBER MOSAIC VIRUS;

EID: 19944373097     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302475     Document Type: Article

References (50)

1. Okamoto N et al. Transgenic mice with increased expression of vascular endothelial growth factor in the retina: a new model of intraretinal and subretinal neovascularization. Am J Pathol 1997; 151:281-291.
2. Tobe T et al. Targeted disruption of the FGF2 gene does not prevent choroidal neovascularization in a murine model. Am J Pathol 1998; 153: 1641-1646.
3. Weng J et al. Insights into the function of rim protein in photoreceptors and etiology of Stargardt's disease from the phenotype in abcr knockout mice. Cell 1999; 98: 13-23.
4. Seo M-S et al. Photoreceptor-specific expression of PDGF-B results in traction retinal detachment. Am J Pathol 2000; 157: 995-1005.
5. Yamada H et al. Platelet-derived growth factor-A-induced retinal gliosis protects against ischemic retinopathy. Am J Pathol 2000; 156: 477-487.
6. Yamada H et al. FGF2 decreases hyperoxia-induced cell death in mice. J Am Pathlol 2001; 159: 1113-1120.
7. Ando A et al. Nitric oxide is proangiogenic in retina and choroid. J Cell Physiol 2001; 191: 116-124.
8. Ando A, Yang A, Nambu H, Campochiaro PA. Blockade of nitric-oxide synthase reduces choroidal neovascularization. Mol Pharmacol 2002; 62: 539-544.
9. Okoye G et al. Increased expression of BDNF preserves retinal function and slows cell death from rhodopsin mutation or oxidative damage. J Neuosci 2003; 23: 4164-4172.
10. Bennett J et al. Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy. Nat Med 1996; 2: 649-654.
11. Mori K et al. Pigment epithelium-derived factor inhibits retinal and choroidal neovascularization. J Cell Physiol 2001; 188: 253-263.
12. Mori K et al. Inhibition of choroidal neovascularization by intravenous injection of adenoviral vectors expressing secretable endostatin. Am J Pathol 2001; 159: 313-320.
13. Lai C-C et al. Suppression of choroidal neovascularization by adeno-associated virus vector expressing angiostatin. Invest Ophthalmol Vis Sci 2001; 42: 2401-2407.
14. Acland GM et al. Gene therapy restores vision in a canine model of childhood blindness. Nat Genet 2001; 28: 92-95.
15. Sanftner LHM, Abel H, Hauswirth WW, Flannery JG. Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa. Mol Ther 2001; 4: 622-629.
16. Bainbridge J et al. Inhibition of retinal neovascularization by gene transfer of soluble VEGF receptor sFlt-1. Gene Therapy 2002; 9: 320-326.
17. Takahashi K et al. Intraocular expression of endostatin reduces VEGF-induced retinal vascular permeability, neovascularization, and retinal detachment. FASEB J 2003; 17: 896-898 (Epub 2003 Mar 28).
18. Rasmussen HS et al. Clinical protocol. An open-label, phase I, single administration, dose-escalation study of ADGVPEDF.11D (ADPEDF) in neovascular age-related macular degeneration (AMD). Hum Gene Ther 2001; 12: 2029-2032.
19. Somiari S et al. Theory and in vivo application of electroporative gene delivery. Mol Ther 2000; 2: 178-187.
20. Coster HGA. Quantitative analysis of the voltage-current relationships of fixed charge membranes and the associated property of 'punch-through'. Biophys J 1965; 5: 669-686.
21. Titomirov AV, Sukharev S, Kistanova E. In vivo electorporation and stable transformation of skin cells of newborn mice by plasmid DNA. Biochim Biophys Acta 1991; 1088: 131-134.
22. Zhang L, Li L, Hoffmann GA, Hoffmann RM. Depth-targeted efficient gene delivery and expression in the skin by pulsed electric fields: an approach to gene therapy of skin aging and other diseases. Biochem Biophys Res Commun 1996; 220: 633-636.
23. Heller R et al. In vivo gene electroinjection and expression in rat liver. FEBS Lett 1996; 389: 225-228.
24. Oshima Y et al. Targeted gene transfer to corneal endothelium in vivo by electric pulse. Gene Therapy 1998; 5: 1347-1354.
25. Oshima Y et al. Targeted gene transfer to corneal stroma in vivo by electric pulses. Exp Eye Res 2002; 74: 191-198.
26. Dezawa M et al. Gene trasfer into retinal ganglion cells by in vivo electroporation: a new approach. Micron 2002; 33: 1-6.
27. Mo X et al. Rescue of axotomized retinal ganglion cells by BDNF gene electroporation in adult rats. Invest Ophthalmol Vis Sci 2002; 43: 2401-2405.
28. Matsuda T, Cepko CL. Electroporation and RNA interference in the rodent retina in vivo and in vitro. Proc Natl Acad Sci USA 2004; 101: 16-22.
29. Felgner PL et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987; 84: 7413-7417.
30. Brigham KS et al. In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle. Am J Med Sci 1989; 298: 278-281.
31. Ono T, Fujino Y, Tsuchiya T, Tsuda M. Plasmid DNAs directly injected into mouse brain with lipofectin can be incorporated and expressed by brain cells. Neurosci Lett 1990; 117: 259-263.
32. Zhu N, Liggitt D, Liu Y, Debs R. Systemic gene expression after intravenous DNA delivery into adult mice. Science 1993; 261: 209-211.
33. Liu F, Song YK, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Therapy 1999; 6: 1258-1266.
34. Zack DJ et al. Unusual topography of bovine rhodopsin promoter-lacZ fusion gene expression in transgenic mouse retinas. Neuron 1991; 6: 187-199.
35. Esumi N et al. Analysis of the VMD2 promoter and implication of E-Box binding factors in Its regulation. J Biol Chem 2004; 279: 19064-19073.
36. Petrukhin K et al. Identification of the gene responsible for Best macular dystrophy. Nat Genet 1998; 19: 241-247.
37. Cepko CL et al. Lineage analysis with retroviral vectors. Methods Enzymol 2000; 327: 118-145.
38. Oshima Y et al. Angiopoietin-2 enhances retinal vessel sensitivity to vascular endothelial growth factor. J Cell Physiol 2004; 199: 412-417.
39. Oshima Y et al. Intraocular gutless adenoviral vectored VEGF stimulates anterior segment but not retinal neovascularization. J Cell Physiol 2004; 199: 399-411.
40. 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.
41. Ali RR et al. Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat Genet 2000; 25: 306-310.
42. 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.
43. Lai C-M et al. Inhibition of angiogenesis by adenovirus-mediated sFlt-1 expression in a rat model of corneal neovascularization. Hum Gene Ther 2001; 12: 1299-1310.
44. 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.
45. 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.
46. Liang F-Q et al. Long-term protection of retinal structure but not function using RAAVCNTF in animal models of retinitis pigmentosa. Mol Ther 2001; 4: 461-472.
47. Campochiaro PA, Hackett SF. Corneal endothelial cell matrix promotes expression of differentiated features of retinal pigmented epithelial cells: implication of laminin and basic fibroblast growth factor as active components. Exp Eye Res 1993; 57: 539-547.
48. Gehlbach P et al. Periocular injection of an adenoviral vector encoding pigment epithelium-derived factor inhibits choroidal neovascularization. Gene Therapy 2003; 10: 637-646.
49. Gehlbach P et al. Periocular gene transfer of sFlt-1 suppresses ocular neovascularization and VEGF-induced breakdown of the blood-retinal barrier. Hum Gene Ther 2003; 14: 129-141.
50. Mori K et al. Intraocular adenoviral vector-mediated gene transfer is increased in proliferative retinopathies. Invest Ophthalmol Vis Sci 2002; 43: 1610-1615.