Long-term retinal transgene expression with FIV versus adenoviral vectors

Molecular Vision

Volumn 10, Issue , 2004, Pages 272-280

  Loewen, Nils ^a   Leske, David A ^a   Cameron, J Douglas ^a   Chen, Yi ^a   Whitwam, Todd ^a   Simari, Robert D ^a   Teo, Wu Lin ^a   Fautsch, Michael P ^a   Poeschla, Eric M ^a   Holmes, Jonathan M ^a  

^a Mayo Clinic   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; GENE PRODUCT;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CHRONIC DISEASE; CONTROLLED STUDY; CYTOMEGALOVIRUS; DRUG ADMINISTRATION ROUTE; ENHANCER REGION; FELINE IMMUNODEFICIENCY VIRUS; FOLLOW UP; GENE CASSETTE; GENE DOSAGE; GENE EXPRESSION; GENE REPLICATION; GENETIC TRANSDUCTION; HISTOLOGY; LONG TERM CARE; MACROPHAGE; MICROSCOPY; NONHUMAN; OPTIMAL DRUG DOSE; PIGMENT EPITHELIUM; PRIORITY JOURNAL; PROMOTER REGION; RAT; RETINA DISEASE; SUBRETINAL DRUG ADMINISTRATION; TRANSCRIPTION REGULATION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VISUAL SYSTEM EXAMINATION; ADENOVIRUS; ANIMAL; COMPARATIVE STUDY; DEFECTIVE VIRUS; ENZYMOLOGY; GENE EXPRESSION REGULATION; GENE TRANSFER; GENE VECTOR; GENETICS; METABOLISM; PHYSIOLOGY; RETINA; SPRAGUE DAWLEY RAT; VIROLOGY;

ADENOVIRIDAE; ANIMALS; BETA-GALACTOSIDASE; DEFECTIVE VIRUSES; FOLLOW-UP STUDIES; GENE EXPRESSION REGULATION, ENZYMOLOGIC; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; IMMUNODEFICIENCY VIRUS, FELINE; RATS; RATS, SPRAGUE-DAWLEY; RETINA; TRANSGENES;

EID: 2342590133     PISSN: 10900535     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (58)

1. Mori K, Gehlbach P, Yamamoto S, Duh E, Zack DJ, Li Q, Berns KI, Raisler BJ, Hauswirth WW, Campochiaro PA. AAV-mediated gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization. Invest Ophthalmol Vis Sci 2002; 43:1994-2000.
2. Lai CC, Wu WC, Chen SL, Xiao X, Tsai TC, Huan SJ, Chen TL, Tsai RJ, Tsao YP. Suppression of choroidal neovascularization by adeno-associated virus vector expressing angiostatin. Invest Ophthalmol Vis Sci 2001; 42:2401-7.
3. Bainbridge JW, Stephens C, Parsley K, Demaison C, Halfyard A, Thrasher AJ, Ali RR. In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium. Gene Ther 2001; 8:1665-8.
4. Auricchio A, Behling KC, Maguire AM, O'Connor EM, Bennett J, Wilson JM, Tolentino MJ. Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents. Mol Ther 2002; 6:490-4.
5. Acland GM, Aguirre GD, Ray J, Zhang Q, Aleman TS, Cideciyan AV, Pearce-Kelling SE, Anand V, Zeng Y, Maguire AM, Jacobson SG, Hauswirth WW, Bennett J. Gene therapy restores vision in a canine model of childhood blindness. Nat Genet 2001; 28:92-5.
6. Liang FQ, Aleman TS, Dejneka NS, Dudus L, Fisher KJ, Maguire AM, Jacobson SG, Bennett J. Long-term protection of retinal structure but not function using RAAV.CNTF in animal models of retinitis pigmentosa. Mol Ther 2001; 4:461-72.
7. Vollrath D, Feng W, Duncan JL, Yasumura D, D'Cruz PM, Chappelow A, Matthes MT, Kay MA, LaVail MM. Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk. Proc Natl Acad Sci U S A 2001; 98:12584-9.
8. Lau D, McGee LH, Zhou S, Rendahl KG, Manning WC, Escobedo JA, Flannery JG. Retinal degeneration is slowed in transgenic rats by AAV-mediated delivery of FGF-2. Invest Ophthalmol Vis Sci 2000; 41:3622-33.
9. Takahashi M, Miyoshi H, Verma IM, Gage FH. Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer. J Virol 1999; 73:7812-6.
10. McNally N, Kenna P, Humphries MM, Hobson AH, Khan NW, Bush RA, Sieving PA, Humphries P, Farrar GJ. Structural and functional rescue of murine rod photoreceptors by human rhodopsin transgene. Hum Mol Genet 1999; 8:1309-12.
11. Ali RR, Sarra GM, Stephens C, Alwis MD, Bainbridge JW, Munro PM, Fauser S, Reichel MB, Kinnon C, Hunt DM, Bhattacharya SS, Thrasher AJ. Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat Genet 2000; 25:306-10.
12. Liang FQ, Dejneka NS, Cohen DR, Krasnoperova NV, Lem J, Maguire AM, Dudus L, Fisher KJ, Bennett J. AAV-mediated delivery of ciliary neurotrophic factor prolongs photoreceptor survival in the rhodopsin knockout mouse. Mol Ther 2001; 3:241-8.
13. Poeschla EM, Wong-Staal F, Looney DJ. Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors. Nat Med 1998; 4:354-7.
14. Loewen N, Barraza R, Whitwam T, Saenz D, Kemler I, Poeschla E. FIV Vectors. In: Federico M, editor. Lentivirus gene engineering protocols. Totowa (NJ): Humana Press; 2003. p. 251-71.
15. Saenz DT, Poeschla EM. FIV: from lentivirus to lentivector. J Gene Med 2004; 6:S95-104.
16. Tsui LV, Kelly M, Zayek N, Rojas V, Ho K, Ge Y, Moskalenko M, Mondesire J, Davis J, Roey MV, Dull T, McArthur JG. Production of human clotting Factor IX without toxicity in mice after vascular delivery of a lentiviral vector. Nat Biotechnol 2002; 20:53-7.
17. Pan D, Gunther R, Duan W, Wendell S, Kaemmerer W, Kafri T, Verma IM, Whitley CB. Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow. Mol Ther 2002; 6:19-29.
18. Loewen N, Bahler C, Teo WL, Whitwam T, Peretz M, Xu R, Fautsch MP, Johnson DH, Poeschla EM. Preservation of aqueous outflow facility after second-generation FIV vector-mediated expression of marker genes in anterior segments of human eyes. Invest Ophthalmol Vis Sci 2002; 43:3686-90.
19. Naldini L, Blomer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, Trono D. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272:263-7.
20. Loewen N, Leske DA, Chen Y, Teo WL, Saenz DT, Peretz M, Holmes JM, Poeschla EM. Comparison of wild-type and class I integrase mutant-FIV vectors in retina demonstrates sustained expression of integrated transgenes in retinal pigment epithelium. J Gene Med 2003; 5:1009-17.
21. Naldini L, Verma IM. Lentiviral vectors. Adv Virus Res 2000; 55:599-609.
22. Zhang Y, Chirmule N, Gao GP, Qian R, Croyle M, Joshi B, Tazelaar J, Wilson JM. Acute cytokine response to systemic adenoviral vectors in mice is mediated by dendritic cells and macrophages. Mol Ther 2001; 3:697-707.
23. Schnell MA, Zhang Y, Tazelaar J, Gao GP, Yu QC, Qian R, Chen SJ, Varnavski AN, LeClair C, Raper SE, Wilson JM. Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors. Mol Ther 2001; 3:708-22.
24. Li T, Adamian M, Roof DJ, Berson EL, Dryja TP, Roessler BJ, Davidson BL. In vivo transfer of a reporter gene to the retina mediated by an adenoviral vector. Invest Ophthalmol Vis Sci 1994; 35:2543-9.
25. Ali RR, Reichel MB, Byrnes AP, Stephens CJ, Thrasher AJ, Baker D, Hunt DM, Bhattacharya SS. Co-injection of adenovirus expressing CTLA4-Ig prolongs adenovirally mediated lacZ reporter gene expression in the mouse retina. Gene Ther 1998; 5:1561-5.
26. Holmes JM, Duffner LA. The effect of postnatal growth retardation on abnormal neovascularization in the oxygen exposed neonatal rat. Curr Eye Res 1996; 15:403-9.
27. Holmes JM, Zhang S, Leske DA, Lanier WL. Carbon dioxide-induced retinopathy in the neonatal rat. Curr Eye Res 1998; 17:608-16.
28. Holmes JM, Zhang S, Leske DA, Lanier WL. Metabolic acidosis-induced retinopathy in the neonatal rat. Invest Ophthalmol Vis Sci 1999; 40:804-9.
29. Penn JS, Tolman BL, Lowery LA. Variable oxygen exposure causes preretinal neovascularization in the newborn rat. Invest Ophthalmol Vis Sci 1993; 34:576-85.
30. Zhang S, Leske DA, Holmes JM. Neovascularization grading methods in a rat model of retinopathy of prematurity. Invest Ophthalmol Vis Sci 2000; 41:887-91.
31. Zhang S, Leske DA, Lanier WL, Berkowitz BA, Holmes JM. Preretinal neovascularization associated with acetazolamide-induced systemic acidosis in the neonatal rat. Invest Ophthalmol Vis Sci 2001; 42:1066-71.
32. Chen Y, Leske DA, Zhang S, Karger RA, Lanier WL, Holmes JM. Duration of acidosis and recovery determine preretinal neovascularization in the rat model of acidosis-induced retinopathy. Curr Eye Res 2002; 24:281-8.
33. Kemler I, Barraza R, Poeschla EM. Mapping the encapsidation determinants of feline immunodeficiency virus. J Virol 2002; 76:11889-903.
34. Engelhardt JF, Yang Y, Stratford-Perricaudet LD, Allen ED, Kozarsky K, Perricaudet M, Yankaskas JR, Wilson JM. Direct gene transfer of human CFTR into human bronchial epithelia of xenografts with E1-deleted adenoviruses. Nat Genet 1993; 4:27-34.
35. Yang Y, Raper SE, Cohn JA, Engelhardt JF, Wilson JM. An approach for treating the hepatobiliary disease of cystic fibrosis by somatic gene transfer. Proc Natl Acad Sci U S A 1993; 90:4601-5.
36. Khurana VG, Weiler DA, Witt TA, Smith LA, Kleppe LS, Parisi JE, Simari RD, O'Brien T, Russell SJ, Katusic ZS. A direct mechanical method for accurate and efficient adenoviral vector delivery to tissues. Gene Ther 2003; 10:443-52.
37. Chen AF, O'Brien T, Tsutsui M, Kinoshita H, Pompili VJ, Crotty TB, Spector DJ, Katusic ZS. Expression and function of recombinant endothelial nitric oxide synthase gene in canine basilar artery. Circ Res 1997; 80:327-35.
38. Loewen N, Fautsch MP, Peretz M, Bahler CK, Cameron JD, Johnson DH, Poeschla EM. Genetic modification of human trabecular meshwork with lentiviral vectors. Hum Gene Ther 2001; 12:2109-19.
39. Boshart M, Weber F, Jahn G, Dorsch-Hasler K, Fleckenstein B, Schaffner W. A very strong enhancer is located upstream of an immediate early gene of human cytomegalovirus. Cell 1985; 41:521-30.
40. Foecking MK, Hofstetter H. Powerful and versatile enhancer-promoter unit for mammalian expression vectors. Gene 1986; 45:101-5.
41. Whitwam T, Peretz M, Poeschla E. Identification of a central DNA flap in feline immunodeficiency virus. J Virol 2001; 75:9407-14.
42. Ohno T, Gordon D, San H, Pompili VJ, Imperiale MJ, Nabel GJ, Nabel EG. Gene therapy for vascular smooth muscle cell proliferation after arterial injury. Science 1994; 265:781-4.
43. Simari RD, San H, Rekhter M, Ohno T, Gordon D, Nabel GJ, Nabel EG. Regulation of cellular proliferation and intimal formation following balloon injury in atherosclerotic rabbit arteries. J Clin Invest 1996; 98:225-35.
44. Timmers AM, Nguyen TB, Saban DR, Grant MB. Viral vectors for efficient gene delivery to RPE cells. Invest Ophthalmol Vis Sci 2001; 42:S125.
45. Duisit G, Conrath H, Saleun S, Folliot S, Provost N, Cosset FL, Sandrin V, Moullier P, Rolling F. Five recombinant simian immunodeficiency virus pseudotypes lead to exclusive transduction of retinal pigmented epithelium in rat. Mol Ther 2002; 6:446-54.
46. Derksen TA, Sauter SL, Davidson BL. Feline immunodeficiency virus vectors. Gene transfer to mouse retina following intravitreal injection. J Gene Med 2002; 4:463-9.
47. Doi K, Hargitai J, Kong J, Tsang SH, Wheatley M, Chang S, Goff S, Gouras P. Lentiviral transduction of green fluorescent protein in retinal epithelium: evidence of rejection. Vision Res 2002; 42:551-8.
48. Bennett J, Wilson J, Sun D, Forbes B, Maguire A. Adenovirus vector-mediated in vivo gene transfer into adult murine retina. Invest Ophthalmol Vis Sci 1994; 35:2535-42.
49. Lai CM, Shen WY, Constable I, Rakoczy PE. The use of adenovirus-mediated gene transfer to develop a rat model for photoreceptor degeneration. Invest Ophthalmol Vis Sci 2000; 41:580-4.
50. Reichel MB, Ali RR, Thrasher AJ, Hunt DM, Bhattacharya SS, Baker D. Immune responses limit adenovirally mediated gene expression in the adult mouse eye. Gene Ther 1998; 5:1038-46.
51. Miyoshi H, Takahashi M, Gage FH, Verma IM. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. Proc Natl Acad Sci U S A 1997; 94:10319-23.
52. da Cruz L, Robertson T, Hall MO, Constable IJ, Rakoczy PE. Cell polarity, phagocytosis and viral gene transfer in cultured human retinal pigment epithelial cells. Curr Eye Res 1998; 17:668-72.
53. Anglade E, Csaky KG. Recombinant adenovirus-mediated gene transfer into the adult rat retina. Curr Eye Res 1998; 17:316-21.
54. Mitani K, Kubo S. Adenovirus as an integrating vector. Curr Gene Ther 2002; 2:135-44.
55. Harui A, Suzuki S, Kochanek S, Mitani K. Frequency and stability of chromosomal integration of adenovirus vectors. J Virol 1999; 73:6141-6.
56. Reichel MB, Bainbridge J, Baker D, Thrasher AJ, Bhattacharya SS, Ali RR. An immune response after intraocular administration of an adenoviral vector containing a beta galactosidase reporter gene slows retinal degeneration in the rd mouse. Br J Ophthalmol 2001; 85:341-4.
57. Muruve DA, Barnes MJ, Stillman IE, Libermann TA. Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo. Hum Gene Ther 1999; 10:965-76.
58. Kafri T, Morgan D, Krahl T, Sarvetnick N, Sherman L, Verma I. Cellular immune response to adenoviral vector infected cells does not require de novo viral gene expression: implications for gene therapy. Proc Natl Acad Sci U S A 1998; 95:11377-82.