Feline immunodeficiency virus vectors. Gene transfer to mouse retina following intravitreal injection

Journal of Gene Medicine

Volumn 4, Issue 5, 2002, Pages 463-469

  Derksen, Todd A ^a   Sauter, Sybille L ^b   Davidson, Beverly L ^a,c  

^a Program in Gene Therapy   (United States)

^b NOVARTIS PHARMA AG   (Switzerland)

^c UNIVERSITY OF IOWA   (United States)

Author keywords

Cornea; FIV; Gene therapy; MPS; Trabecular meshwork

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; BETA GLUCURONIDASE; GREEN FLUORESCENT PROTEIN; VIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CORNEA ENDOTHELIUM; DISEASE MODEL; DRUG EFFICACY; ESCHERICHIA COLI; FELINE IMMUNODEFICIENCY VIRUS; GENE EXPRESSION; GENETIC TRANSDUCTION; HYPOTHESIS; IMMUNOHISTOCHEMISTRY; LYSOSOME STORAGE DISEASE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; PIGMENT EPITHELIUM; PRIORITY JOURNAL; RETINA DISEASE; TRANSGENE; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; VIRUS RECOMBINANT; ANIMAL; BAGG ALBINO MOUSE; CORNEA; GENE TRANSFER; GENE VECTOR; GENETICS; METABOLISM; RETINA; ULTRASTRUCTURE;

ADENOVIRIDAE; ANIMALIA; FELIDAE; FELINE IMMUNODEFICIENCY VIRUS; MURINAE;

ANIMALS; CORNEA; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GLUCURONIDASE; IMMUNODEFICIENCY VIRUS, FELINE; MICE; MICE, INBRED BALB C; MUCOPOLYSACCHARIDOSIS VII; RETINA;

EID: 0036727920     PISSN: 1099498X     EISSN: None     Source Type: Journal    
DOI: 10.1002/jgm.267     Document Type: Article

References (38)

1. Sly WS, Quinton BA, McAlister WH, et al. Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis. J Pediatr 1973; 82: 249-257.
2. Sands MS, Vogler C, Kyle JW, et al. Enzyme replacement therapy for murine mucopolysaccharidosis type VII. J Clin Invest 1994; 93: 2324-2331.
3. Sands MS, Vogler C, Torrey A, et al. Murine mucopolysaccharidosis type VII: long-term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation. J Clin Invest 1997; 99: 1596-1605.
4. Taylor RM, Wolfe JH. Cross-correction of β-glucuronidase deficiency by retroviral vector-mediated gene transfer. Exp Cell Res 1994, 214: 606-613.
5. Li T, Davidson BL. Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer. Proc Natl Acad Sci U S A 1995; 92: 7700-7704.
6. Birkenmeier EH, Davisson MT, Beamer WG, et al. Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency. J Clin Invest 1989; 83: 1258-1266.
7. Vogler C, Birkenmeier EH, Sly WS, et al. A murine model of mucopolysaccharidosis VII. Am J Pathol 1990; 136: 207-217.
8. Mollard RJ, Telegan P, Haskins M, et al. Corneal endothelium in mucopolysaccharide storage disorders. Cornea 1996; 15: 25-34.
9. Vogler C, Sands MS, Galvin N, et al. Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a routine model of lysosomal storage disease. J Inherit Metab Dis 1998; 21: 575-586.
10. Li T, Adamian M, Roof DJ, et al. In vivo transfer of a reporter gene to the retina mediated by an adenoviral vector. Invest Ophthalmol Vis Sci 1994; 35: 2543-2549.
11. Bennett J, Wilson J, Sun D, et al. Adenovirus vector-mediated in vivo gene transfer into adult murine retina. Invest Ophthalmol Vis Sci 1994; 35: 2535-2542.
12. Borrás T, Rowlette LL, Erzurum SC, et al. Adenoviral reporter gene transfer to the human trabecular meshwork does not alter aqueous humor outflow. Relevance for potential gene therapy of glaucoma. Gene Ther 1999; 6: 515-524.
13. Sakamoto T, Ueno H, Goto Y, et al. A vitrectomy improves the transfection efficiency of adenoviral vector-mediated gene transfer to Müller cells. Gene Ther 1998; 5: 1088-1097.
14. Grant CA, Ponnazhagan S, Wang X-S, et al. Evaluation of recombinant adeno-associated virus as a gene transfer vector for the retina. Curr Eye Res 1997; 16: 949-956.
15. Ali RR, Reichel MB, De Alwis M, et al. Adeno-associated virus gene transfer to mouse retina. Hum Gene Ther 1998; 9: 81-86.
16. Flannery JG, Zolotukhin S, Vaquero MI, et al. Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc Natl Acad Sci U S A 1997; 94: 6916-6921.
17. Jomary C, Vincent KA, Grist J, et al. Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration. Gene Ther 1997; 4: 683-690.
18. Rolling F, Shen W-Y, Tabarias H, et al. Evaluation of adeno-associated virus-mediated gene transfer into the rat retina by clinical fluorescence photography. Hum Gene Ther 1999; 10: 641-648.
19. Ali RR, Reichel MB, Thrasher AJ, et al. Gene transfer into the mouse retina mediated by an adeno-associated viral vector. Hum Mol Genet 1996; 5: 591-594.
20. Ali RR, Sarra G-M, Stephens C, et al. Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nature Genet 2000; 25: 306-310.
21. Dudus L, Anand V, Acland GM, et al. Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV. Vision Res 1999; 39: 2545-2553.
22. Auricchio A, Kobinger G, Anand V, 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.
23. Miyoshi H, Takahaski M, Gage FH, et al. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. Proc Natl Acad Sci U S A 1997; 94: 10319-10323.
24. Takahashi M, Miyoshi H, Verma IM, et al. Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer. J Virol 1999; 73: 7812-7816.
25. Wang X, Appukuttan B, Ott S, et al. Efficient and sustained transgene expression in human corneal cells mediated by a lentiviral vector. Gene Ther 2000; 7: 196-200.
26. Anderson RD, Haskell RE, Xia H, et al. A simple method for the rapid generation of recombinant adenovirus vectors. Gene Ther 2000; 7: 1034-1038.
27. Jones N, Shenk T. An adenovirus type 5 early gene function regulates expression of other early viral genes. Proc Natl Acad Sci U S A 1979; 76: 3665-3669.
28. Johnston JC, Gasmi M, Lim LE, et al. Minimal requirements for efficient transduction of dividing and nondividing cells by feline immunodeficiency virus vectors. J Virol 1999; 73: 4991-5000.
29. Yee JK, Miyanohara A, LaPorte P, et al. A general method for the generation of high-titer, pantropic retroviral vectors: highly efficient infection of primary hepatocytes. Proc Natl Acad Sci U S A 1994; 91: 9564-9568.
30. Bancroft JD. Enzyme histochemistry. in Theory and Practice of Histological Techniques, Bancroft JD, Stevens A (eds). Churchill Livingstone: New York, 1982: 387-388.
31. Ghodsi A, Stein C, Derksen T, et al. Extensive β-glucuronidase activity in murine CNS after adenovirus mediated gene transfer to brain. Hum Gene Ther 1998; 9: 2331-2340.
32. Roessler BJ, Davidson BL. Direct plasmid mediated transfection of adult murine brain cells in vivo using cationic liposomes. Neurosci Lett 1994; 167: 5-10.
33. Glaser JH, Sly WS. Beta-glucuronidase deficiency mucopolysaccharidosis: methods for enzymatic diagnosis. J Lab Clin Med 1973; 82: 969-977.
34. Hoffman LM, Maguire AM, Bennett J. Cell-mediated immune response and stability of intraocular transgene expression after adenovirus-mediated delivery. Invest Ophthalmol Vis Sci 1997; 38 2224-2233.
35. Reichel MB, Ali RR, Thrasher AJ, et al. Immune responses limit adenovirally mediated gene expression in the adult mouse eye. Gene Ther 1998; 5: 1038-1046.
36. Duan D, Yan Z, Yue Y, et al. Enhancement of muscle gene delivery with pseudotyped adeno-associated virus type 5 correlates with myoblast differentiation. J Virol 2001; 75: 7662-7671.
37. Bennett J, Maguire AM, Cideciyan AV, et al. Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natt Acad Sci U S A 1999; 96: 9920-9925.
38. Blömer U, Naldini L, Kafri T, et al. Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J Virol 1997; 71: 6641-6649.