Expression of erythropoietin in cats treated with a recombinant adeno-associated viral vector

American Journal of Veterinary Research

Volumn 66, Issue 3, 2005, Pages 450-456

  Walker, Mark C ^a,b,f   Mandell, Tamara C ^b   Crawford, P Cynda ^a   Simon, Greg G ^c   Cahill, Kevin S ^c   Fernandes, Peter J ^d,g   MacLeod, James N ^e,h   Byrne, Barry J ^c   Levy, Julie K ^a  

^a University of Florida   (United States)

^b APPLIED GENETIC TECHNOLOGIES CORPORATION   (United States)

^c UNIVERSITY OF FLORIDA   (United States)

^d Cytovet Diagnostics   (United States)

^e North Florida Veterinary Specialists ^*  (United States)

^f Florida Veterinary Specialists   (United States)

^g IDEXX Veterinary Services Inc   (United States)

^h UNIVERSITY OF KENTUCKY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GALACTOSIDASE; COMPLEMENTARY DNA; ERYTHROPOIETIN; PARVOVIRUS VECTOR;

ANIMAL TISSUE; ANTIBODY TITER; APLASIA; ARTICLE; CAT; CONCENTRATION RESPONSE; CONTROLLED STUDY; ERYTHROCYTE; ERYTHROCYTOSIS; FEMALE; GENE EXPRESSION; HEMATOCRIT; HEMATOLOGICAL PARAMETERS; MALE; NONHUMAN; NUCLEOTIDE SEQUENCE; VIRAL GENE DELIVERY SYSTEM;

ACTINS; ANEMIA; ANIMALS; ANTIBODIES, VIRAL; CAT DISEASES; CATS; DEPENDOVIRUS; DNA, COMPLEMENTARY; ENZYME-LINKED IMMUNOSORBENT ASSAY; ERYTHROPOIETIN; GENE THERAPY; GENETIC VECTORS; NEUTRALIZATION TESTS; PROMOTER REGIONS (GENETICS);

ADENO-ASSOCIATED VIRUS; ANIMALIA; FELIDAE; FELIS CATUS; PARVOVIRUS;

EID: 17544378467     PISSN: 00029645     EISSN: None     Source Type: Journal    
DOI: 10.2460/ajvr.2005.66.450     Document Type: Article

References (47)

1. Fisher JW, Koury S, Ducey T, et al. Erythropoietin production by interstitial cells of hypoxic monkey kidneys. Br J Haematol 1996;95:27-32.
2. Koury MJ, Bondurant MC. Control of red cell production: the roles of programmed cell death (apoptosis) and erythropoietin. Transfusion 1990;30:673-674.
3. Sato T, Maekawa T, Watanabe S, et al. Erythroid progenitors differentiate and mature in response to endogenous erythropoietin. J Clin Invest 2000;106:263-270.
4. Cowgill LD, James KM, Levy JK, et al. Use of recombinant human erythropoietin for management of anemia in dogs and cats with renal failure. J Am Vet Med Assoc 1998;212:521-528.
5. Arai M, Darman J, Lewis A, et al. The use of human hemopoietic growth factors (rhGM-CSF and rhEPO) as a supportive therapy for FIV-infected cats. Vet Immunol Immunopathol 2000;77:71-92.
6. Randolph JF, Scarlett JM, Stokol T, et al. Expression, bioactivity, and clinical assessment of recombinant feline erythropoietin. Am J Vet Res 2004;65:1355-1366.
7. Carter BJ. Adeno-associated virus vectors. Curr Opin Biotechnol 1992;3:533-539.
8. Muzyczka N. Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr Top Microbiol Immunol 1992;158:97-129.
9. Blacklow NR, Hoggan MD, Kapikian AZ, et al. Epidemiology of adenovirus-associated virus infection in a nursery population. Am J Epidemiol 1968;88:368-378.
10. Kearns WG, Afione SA, Fulmer SB, et al. Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Ther 1996;3:748-755.
11. Ponnazhagan S, Erikson D, Kearns WG, et al. Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells. Hum Gene Ther 1997;8:275-284.
12. Kessler PD, Podsakoff GM, Che X, et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci U S A 1996;93:14082-14087.
13. Zolotukhin S, Byrne BJ, Mason E, et al. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther 1999;6:973-985.
14. Wen D, Boissel JP, Tracy TE, et al. Erythropoietin structure-function relationships: high degree of sequence homology among mammals. Blood 1993;82:1507-1516.
15. Smith LM, Sanders JZ, Kaiser RJ, et al. Fluorescence detection in automated DNA sequence analysis. Nature 1986;321:674-679.
16. Daly TM, Okuyama T, Vogler C, et al. Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice. Hum Gene Ther 1999;10:85-94.
17. Hennighausen L, Fleckenstein B. Nuclear factor 1 interacts with five DNA elements in the promoter region of the human cytomegalovirus major immediate early gene. EMBO J 1986;5:1367-1371.
18. Miyazaki J, Takaki S, Araki K, et al. Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5. Gene 1989;79:269-277.
19. Niwa H, Yamamura K, Miyazaki J. Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 1991;108:193-199.
20. Xu L, Daly T, Gao C, et al. CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice. Hum Gene Ther 2001;12:563-573.
21. Zolotukhin S, Potter M, Zolotukhin I, et al. Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods 2002;28:158-167.
22. Grimm D, Kern A, Rittner K, et al. Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum Gene Ther 1998;9:2745-2760.
23. Williams LS, Levy JK, Robertson SA, et al. Use of the anesthetic combination of tiletamine, zolazepam, ketamine, and xylazine for neutering feral cats. J Am Vet Med Assoc 2002;220:1491-1495.
24. Naffakh N, Pinset C, Montarras D, et al. Long-term secretion of therapeutic proteins from genetically modified skeletal muscles. Hum Gene Ther 1996;7:11-21.
25. Hasler AH, Giger U. Serum erythropoietin values in polycythemic cats. J Am Anim Hosp Assoc 1996;32:294-301.
26. Jain NC. The cat: normal hematology with comments of response to disease. In: Schalm's veterinary hematology. 4th ed. Philadelphia: Lea & Febiger, 1986;126-139.
27. Meyer DJ, Harvey JW. Hematopoiesis and the evaluation of bone marrow. In: Meyer DJ, Harvey JW, eds. Veterinary laboratory medicine interpretation and diagnosis. 2nd ed. Philadelphia: WB Saunders Co, 1998;23-42.
28. Beall CJ, Phipps AJ, Mathes LE, et al. Transfer of the feline erythropoietin gene to cats using a recombinant adeno-associated virus vector. Gene Ther 2000;7:534-539.
29. Mandel RJ, Spratt SK, Snyder RO, et al. Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats. Proc Natl Acad Sci U S A 1997;94:14083-14088.
30. McCarty DM, Monahan PE, Samulski RJ. Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 2001;8:1248-1254.
31. Xiao W, Chirmule N, Schnell MA, et al. Route of administration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors. Mol Ther 2000;1:323-329.
32. Chirmule N, Propert K, Magosin S, et al. Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 1999;6:1574-1583
33. Snyder RO, Spratt SK, Lagarde C, et al. Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice. Hum Gene Ther 1997;8:1891-1900.
34. Chao H, Liu Y, Rabinowitz J, et al. Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol Ther 2000;2:619-623.
35. Rendahl KG, Quiroz D, Ladner M, et al. Tightly regulated long-term erythropoietin expression in vivo using tet-inducible recombinant adeno-associated viral vectors. Hum Gene Ther 2002;13:335-342.
36. Bohl D, Heard JM. Modulation of erythropoietin delivery from engineered muscles in mice. Hum Gene Ther 1997;8:195-204.
37. Fisher JW. Erythropoietin: physiologic and pharmacologic aspects. Proc Soc Exp Biol Med 1997;216:358-369.
38. Giger U. Regenerative anemias caused by blood loss or hemolysis. In: Ettinger SJ, Feldman EC, eds. Textbook of veterinary internal medicine. 5th ed. Philadelphia: WB Saunders Co, 2000;1784-1804.
39. Schellekens H. Bioequivalence and the immunogenicity of biopharmaceuticals. Nat Rev Drug Discov 2002;1:457-462.
40. Eckardt KU, Casadevall N. Pure red-cell aplasia due to antierythropoietin antibodies. Nephrol Dial Transplant 2003;18:865-869.
41. Conrad CK, Allen SS, Afione SA, et al. Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther 1996;3:658-668.
42. 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.
43. Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996;70:8098-8108.
44. Herzog RW, Yang EY, Couto LB, et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat Med 1999;5:56-63.
45. Favre D, Provost N, Blouin V, et al. Immediate and long-term safety of recombinant adeno-associated virus injection into the nonhuman primate muscle. Mol Ther 2001;4:559-566.
46. Ye X, Rivera V, Zoltick P, et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science 1999;283:88-91.
47. Kay MA, Landen CN, Rothenberg SR, et al. In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc Natl Acad Sci U S A 1994;91:2353-2357.