Simian immunodeficiency virus-based lentivirus vector for retinal gene transfer: A preclinical safety study in adult rats

Gene Therapy

Volumn 10, Issue 14, 2003, Pages 1161-1169

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

^a KYUSHU UNIVERSITY   (Japan)

^b KAGOSHIMA UNIVERSITY   (Japan)

^c DNAVEC Research Inc   (Japan)

Author keywords

Electroretinogram; Lentivirus; Retina; Simian immunodeficiency virus

Indexed keywords

BETA GALACTOSIDASE; BUFFER; GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CELL INFILTRATION; CONTROLLED STUDY; DARK ADAPTATION; ELECTRORETINOGRAPHY; FUNCTIONAL ASSESSMENT; GENE EXPRESSION; GENE LOCATION; GENE TRANSFER; LIGHT ADAPTATION; MALE; MONONUCLEAR CELL; NONHUMAN; PIGMENT EPITHELIUM; PRIORITY JOURNAL; RAT; RETINA CELL; RETINA DEGENERATION; RETINA TUMOR; RETINITIS; RETINITIS PIGMENTOSA; SAFETY; SIMIAN IMMUNODEFICIENCY VIRUS; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS LOAD;

ANIMALS; ELECTRORETINOGRAPHY; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; INJECTIONS; LUMINESCENT PROTEINS; MALE; MODELS, ANIMAL; NECROSIS; RATS; RATS, WISTAR; RETINA; RETINAL DISEASES; SIMIAN IMMUNODEFICIENCY VIRUS; TIME FACTORS; TRANSGENES;

ANIMALIA; CERCOPITHECUS AETHIOPS; HUMAN IMMUNODEFICIENCY VIRUS; LENTIVIRUS; RNA VIRUSES; SIMIAE; SIMIAN IMMUNODEFICIENCY VIRUS;

EID: 0042810885     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3301973     Document Type: Article

References (32)

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. Bennett 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. Bennett J et al. Adenovirus-mediated delivery of rhodopsin-promoted bcl-2 results in a delay in photoreceptor cell death in the rd/rd mouse. Gene Therapy 1998; 5: 1156-1164.
9. Sakamoto T et al. Retinal functional change caused by adenoviral vector-mediated transfection of LacZ gene. Hum Gene Ther 1998; 9: 789-799.
10. Ali RR et al. Gene transfer into the mouse retina mediated by an adeno-associated viral vector. Hum Mol Genet 1996; 5: 591-594.
11. Flannery JG et al. Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc Natl Acad Sci USA 1997; 94: 6916-6921.
12. Bennett J et al. Real-time, noninvasive in vivo assessment of adeno-associated virus-mediated retinal transduction. Invest Ophthalmol Vis Sci 1997; 38: 2857-2863.
13. 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.
14. Bennett 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. 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.
16. 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.
17. Ali RR et al. Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat Genet 2000; 25: 306-310.
18. Naldini L et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-267.
19. Nakajima T et al. Development of novel simian immunodeficiency virus vectors carrying a dual gene expression system. Hum Gene Ther 2000; 11: 1863-1874.
20. Auricchio A et al. Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum Mol Genet 2001; 10: 3075-3081.
21. Bainbridge JW et al. 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 Therapy 2001; 8: 1665-1668.
22. Duisit G et al. Five recombinant simian immunodeficiency virus pseudotypes lead to exclusive transduction of retinal pigmented epithelium in rat. Mol Ther 2002; 6: 446-454.
23. Sakamoto T et al. A vitrectomy improves the transfection efficiency of adenoviral vector-mediated gene transfer to Muller cells. Gene Therapy 1998; 5: 1088-1097.
24. Reichel MB et al. Immune responses limit adenovirally mediated gene expression in the adult mouse eye. Gene Therapy 1998; 5: 1038-1046.
25. Van Ginkel FW et al. Intratracheal gene delivery with adenoviral vector induces elevated systemic IgG and mucosal IgA antibodies to adenovirus and beta-galactosidase. Hum Gene Ther 1995; 6: 895-903.
26. Van Ginkel FW et al. Adenoviral gene delivery elicits distinct pulmonary-associated T helper cell responses to the vector and to its transgene. J Immunol 1997; 159: 685-693.
27. Stripecke R et al. Immune response to green fluorescent protein: implications for gene therapy. Gene Therapy 1999; 6: 1305-1312.
28. Doi K et al. Lentiviral transduction of green fluorescent protein in retinal epithelium: evidence of rejection. Vision Res 2002; 42: 551-558.
29. Johnson PR et al. Molecular clones of SIVsm and SIVagm: experimental infection of macaques and African green monkeys. J Med Primatol 1990; 19: 279-286.
30. Ikeda Y et al. Recombinant sendai virus-mediated gene transfer into the retinal tissue of adult rats: efficient gene transfer by brief exposure. Exp Eye Res 2002; 75: 39-48.
31. Goto Y et al. Functional abnormalities in transgenic mice expressing a mutant rhodopsin gene. Invest Ophthalmol Vis Sci 1995; 36: 62-71.
32. Goto Y. An electrode to record the mouse cornea electroretinogram. Doc Ophthalmol 1996; 91: 147-154.