Recombinant AAV-transduced iris pigment epithelial cell transplantation may transfer vector to native RPE but suppress systemic dissemination

Investigative Ophthalmology and Visual Science

Volumn 47, Issue 2, 2006, Pages 745-752

  Yoshioka, Yuki ^a   Abe, Toshiaki ^a   Wakusawa, Ryosuke ^a   Moriya, Takuya ^a   Mochizuki, Shizue ^a   Saigo, Yoko ^a   Saito, Takae ^a   Murata, Hiromi ^a   Tokita, Yumi ^a   Iseya, Takashi ^a   Sugano, Eriko ^a   Tomita, Hiroshi ^a   Sagara, Yoshiko ^a   Tamai, Makoto ^a  

^a TOHOKU UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

VIRUS VECTOR; BETA GALACTOSIDASE; DEXTRAN; FLUORESCEIN DERIVATIVE; FLUORESCEIN DEXTRAN; FLUORESCEIN-DEXTRAN; VIRUS DNA;

ADENOVIRUS; ANIMAL CELL; ANIMAL TISSUE; ARTICLE; CELL TRANSPLANTATION; CONTROLLED STUDY; ELECTRON MICROSCOPY; ELECTRORETINOGRAPHY; IMMUNOHISTOCHEMISTRY; IRIS; NONHUMAN; PIGMENT EPITHELIUM; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; RAT; RETINA; VIRAL GENE DELIVERY SYSTEM; VIRUS GENOME; VIRUS RECOMBINANT; ADENO ASSOCIATED VIRUS; ANIMAL; CELL CULTURE; CELL SURVIVAL; CYTOLOGY; EXTRACELLULAR SPACE; FLUORESCENCE MICROSCOPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; LACTOSE OPERON; LONG EVANS RAT; METABOLISM; PHYSIOLOGY; TRANSPLANTATION; VIROLOGY;

ANIMALS; BETA-GALACTOSIDASE; CELL SURVIVAL; CELL TRANSPLANTATION; CELLS, CULTURED; DEPENDOVIRUS; DEXTRANS; DNA, VIRAL; ELECTRORETINOGRAPHY; EXTRACELLULAR SPACE; FLUORESCEINS; GENETIC VECTORS; GENOME, VIRAL; IMMUNOHISTOCHEMISTRY; IRIS; LAC OPERON; MICROSCOPY, FLUORESCENCE; PIGMENT EPITHELIUM OF EYE; POLYMERASE CHAIN REACTION; RATS; RATS, LONG-EVANS; RETINA; TRANSDUCTION, GENETIC;

EID: 33644849963     PISSN: 01460404     EISSN: None     Source Type: Journal    
DOI: 10.1167/iovs.05-0398     Document Type: Article

References (45)

1. Rabinowitz JE, Samulski J. Adeno-associated virus expression systems for gene transfer. Curr Opin Biotechnol. 1998;9:470-475.
2. Guy J, Qi X, Muzyczka N, Hauswirth WW. Reporter expression persists 1 year after adeno-associated virus-mediated gene transfer to the optic nerve. Arch Ophthalmol. 1999;117:929-937.
3. Shen Y, Muramatsu SI, Ikeguchi K, et al. Triple transduction with adeno-associated virus vectors expressing tyrosine hydroxylase, aromatic-L-amino-acid decarboxylase, and GTP cyclohydrolase I for gene therapy of Parkinson's disease. Hum Gene Ther. 2000;11:1509-1519.
4. Harvey AR, Kamphuis W, Eggers R, et al. Intravitreal injection of adeno-associated viral vectors results in the transduction of different types of retinal neurons in neonatal and adult rats: a comparison with lentiviral vectors. Mol Cell Neurosci. 2002;21:141-157.
5. Wu WC, Lai CC, Chen SL, et al. Gene therapy for detached retina by adeno-associated virus vector expressing glial cell line-derived neurotrophic factor. Invest Ophthalmol Vis Sci. 2002;43:3480-3488.
6. Lau D, McGee LH, Zhou S, et al. Retinal degeneration is slowed in transgenic rats by AAV-mediated delivery of FGF-2. Invest Ophthalmol Vis Sci. 2000;41:3622-3633.
7. Adamus G, Sugden B, Shiraga S, Timmers AM, Hauswirth WW. Anti-apoptotic effects of CNTF gene transfer on photoreceptor degeneration in experimental antibody-induced retinopathy. J Autoimmun. 2003;21:121-129.
8. Petrin D, Baker A, Coupland SG, et al. Structural and functional protection of photoreceptors from MNU-induced retinal degeneration by the X-linked inhibitor of apoptosis. Invest Ophthalmol Vis Sci. 2003;44:2757-2763.
9. Hojo M, Abe T, Sugano E, et al. Photoreceptor protection by iris pigment epithelial transplantation transduced with AAV-mediated brain-derived neurotrophic factor gene. Invest Ophthalmol Vis Sci. 2004;45:3721-3726.
10. Narfstrom K, Katz ML, Bragadottir R, et al. Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog. Invest Ophthalmol Vis Sci. 2003;44:1663-1672.
11. Monahan PE, Samulski RJ, Tazelaar J, et al. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther. 1998;5:40-49.
12. Brockstedt DG, Podsakoff GM, Fong L, et al. Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration. Clin Immunol. 1999;92:67-75.
13. Zhang Y, Chirmule N, Gao G, Wilson J. CD40 ligand-dependent activation of cytotoxic T lymphocytes by adeno-associated virus vectors in vivo: role of immature dendritic cells. J Virol. 2000;74:8003-8010.
14. Yuasa K, Sakamoto M, Miyagoe-Suzuki Y, et al. Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product. Gene Ther. 2002;9:1576-1588.
15. Tenenbaum L, Lehtonen E, Monahan PE. Evaluation of risks related to the use of adeno-associated virus-based vectors. Curr Gene Ther. 2003;3:545-565.
16. Weber M, Rabinowitz J, Provost N, et al. Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery. Mol Ther. 2003;7:774-781.
17. Mehrle S, Rohde V, Schlehofer JR. Evidence of chromosomal integration of AAV DNA in human testis tissue. Virus Genes. 2004;28:61-69.
18. Erles K, Sebokova P, Schlehofer JR. Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV). J Med Virol. 1999;59:406-411.
19. Auricchio A, Behling KC, Maguire AM, et al. Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents. Mol Ther. 2002;6:490-494.
20. Grimm D, Kern A, Pawlita M, et al. Titration of AAV-2 particles via a novel capsid ELISA: packaging of genomes can limit production of recombinant AAV-2. Gene Ther. 1999;6:1322-1330.
21. Abe T, Tomita H, Ohashi T, et al. Characterization of iris pigment epithelial cell for auto cell transplantation. Cell Transplant. 1999;8:501-510.
22. Hiratsuka M, Kishikawa Y, Narahara K, et al. Detection of angiotensin-converting enzyme insertion/deletion polymorphisms using real-time polymerase chain reaction and melting curve analysis with SYBR Green I on a GeneAmp 5700. Anal Biochem. 2001;289:300-303.
23. Kim DW. Real time quantitative PCR [in Korean]. Exp Mol Med. 2001;33:101-109.
24. Anglade E, Csaky KG. Recombinant adenovirus-mediated gene transfer into the adult rat retina. Curr Eye Res. 1998;17:316-321.
25. Mizota A, Adachi-Usami E. Effect of body temperature on electro-retinogram of mice. Invest Ophthalmol Vis Sci. 2002;43:3754-3757.
26. Feeney SA, Simpson DA, Gardiner TA, et al. Role of vascular endothelial growth factor and placental growth factors during retinal vascular development and hyaloid regression. Invest Ophthalmol Vis Sci. 2003;44:839-847.
27. Reich SJ, Fosnot J, Kuroki A, et al. Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model. Mol Vis. 2003;9:210-216.
28. Clark KR, Voulgaropoulou F, Johnson PR. A stable cell line carrying adenovirus-inducible rep and cap genes allows for infectivity titration of adeno-associated virus vectors. Gene Ther. 1996;3:1124-1132.
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. Nakai H, Montini E, Fuess S, et al. AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet. 2003;34:297-302.
31. Russell DW. AAV loves an active genome. Nat Genet. 2003;34:241-242.
32. Hansen KA, Sugino IK, Yagi F, et al. Adeno-associated virus encoding green fluorescent protein as a label for retinal pigment epithelium. Invest Ophthalmol Vis Sci. 2003;44:772-780.
33. Del Priore LV, Ishida O, Johnson EW, et al. Triple immune suppression increases short-term survival of porcine fetal retinal pigment epithelium xenografts. Invest Ophthalmol Vis Sci. 2003;44:4044-4053.
34. 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.
35. Crafoord S, Dafgard Kopp E, Seregard S, Algvere PV. Cellular migration into neural retina following implantation of melanin granules in the subretinal space. Graefes Arch Clin Exp Ophthalmol. 2000;238:682-689.
36. Braun M, Kage A, Heimann K, Schraermeyer U. Retinal pigment epithelial cells from Royal College of Surgeons dystrophic rats can take up melanin granules. Graefes Arch Clin Exp Ophthalmol. 1999;237:67-71.
37. Tezel TH, Del Priore LV. Reattachment to a substrate prevents apoptosis of human retinal pigment epithelium. Graefes Arch Clin Exp Ophthalmol. 1997;235:41-47.
38. Abe T, Saigo Y, Hojo M, et al. Protection of photoreceptor cells from phototoxicity by transplanted retinal pigment epithelial cells expressing different neurotrophic factors. Cell Transplant. In press.
39. Saigo Y, Abe T, Hojo M, et al. Transplantation of transduced retinal pigment epithelium in rats. Invest Ophthalmol Vis Sci. 2004;45:1996-2004.
40. Lawrence JM, Sauve Y, Keegan DJ, et al. Schwann cell grafting into the retina of the dystrophic RCS rat limits functional deterioration. Royal College of Surgeons. Invest Ophthalmol Vis Sci. 2000;41:518-528.
41. Bennett J, Duan D, Engelhardt JF, Maguire AM. Real-time, noninvasive in vivo assessment of adeno-associated virus-mediated retinal transduction. Invest Ophthalmol Vis Sci. 1997;38:2857-2863.
42. Abe T, Takeda Y, Yamada K, et al. Cytokine gene expression after subretinal transplantation. Tohoku J Exp Med. 1999;189:179-189.
43. Couto L, Parker A, Gordon JW. Direct exposure of mouse spermatozoa to very high concentrations of a serotype-2 adeno-associated virus gene therapy vector fails to lead to germ cell transduction. Hum Gene Ther. 2004;15:287-291.
44. Kano T, Abe T, Tomita H, et al. Protective effect against ischemia and light damage of iris pigment epithelial cells transfected with the BDNF gene. Invest Ophthalmol Vis Sci. 2002;43:3744-3753.
45. Lawrence JM, Keegan DJ, Muir EM, et al. Transplantation of Schwann cell line clones secreting GDNF or BDNF into the retinas of dystrophic Royal College of Surgeons rats. Invest Ophthalmol Vis Sci. 2004;45:267-274.