Long-term reversal of chronic anemia using a hypoxia-regulated erythropoietin gene therapy

Blood

Volumn 100, Issue 7, 2002, Pages 2406-2413

  Binley, Katie ^a,b,c,d   Askham, Zoe ^a,b,c,d   Iqball, Sharifah ^a,b,c,d   Spearman, Hayley ^a,b,c,d   Martin, Leigh ^a,b,c,d   De Alwis, Mahesh ^a,b,c,d   Thrasher, Adrian J ^a,b,c,d   Ali, Robin R ^a,b,c,d   Maxwell, Patrick H ^a,b,c,d   Kingsman, Susan ^a,b,c,d   Naylor, Stuart ^a,b,c,d  

^a OXFORD BIOMEDICA UK LTD   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

^d IMPERIAL COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ERYTHROPOIETIN; VIRUS LARGE T ANTIGEN;

ADENO ASSOCIATED VIRUS; ANEMIA; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CHRONIC DISEASE; CONTROLLED STUDY; CYTOMEGALOVIRUS; GENE CONTROL; GENE THERAPY; LIVER; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN EXPRESSION; SPLEEN;

EID: 0036785428     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2002-02-0605     Document Type: Article

References (40)

1. Jelkmann W. Erythropoietin: structure, control of production, and function. Physiol Rev. 1992;72:449-489.
2. Eschbach JW. The anemia of chronic renal failure: pathophysiology and the effects of recombinant erythropoietin. Kidney Int. 1989;35:134-148.
3. Goodnough LT, Skikne B, Brugnara C. Erythropoietin, iron, and erythropoiesis. Blood. 2000;96:823-833.
4. Bron D, Meuleman N, Mascaux C. Biological basis of anemia. Semin Oncol. 2001;28:1-6.
5. Rudich SM, Zhou S, Srivastava R, Escobedo JA, Perez RV, Manning WC. Dose response to a single intramuscular injection of recombinant adeno-associated virus-erythropoietin in monkeys. J Surg Res. 2000;90:102-108.
6. Beall CJ, Phipps AJ, Mathes LE, Stromberg P, Johnson PR. Transfer of the feline erythropoietin gene to cats using a recombinant adeno-associated virus vector. Gene Ther. 2000;7:534-539.
7. Serguera C, Bohl D, Rolland E, Prevost P, Heard JM. Control of erythropoietin secretion by doxycycline or mifepristone in mice bearing polymer-encapsulated engineered cells. Hum Gene Ther. 1999;10:375-383.
8. Bohl D, Salvetti A, Moullier P, Heard JM. Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector. Blood. 1998;92:1512-1517.
9. Seppen J, Barry SC, Harder B, Osborne WR. Lentivirus administration to rat muscle provides efficient sustained expression of erythropoietin. Blood. 2001;98:594-596.
10. Bartholomew A, Patil S, Mackay A, et al. Baboon mesenchymal stem cells can be genetically modified to secrete human erythropoietin in vivo. Hum Gene Ther. 2001;12:1527-1541.
11. Bohl D, Bosch A, Cardona A, Salvetti A, Heard JM. Improvement of erythropoiesis in beta-thalassemic mice by continuous erythropoietin delivery from muscle. Blood. 2000;95:2793-2798.
12. Dalle B, Payen E, Regulier E, et al. Improvement of mouse beta-thalassemia upon erythropoietin delivery by encapsulated myoblasts. Gene Ther. 1999;6:157-161.
13. Hamamori Y, Samal B, Tian J, Kedes L. Myoblast transfer of human erythropoietin gene in a mouse model of renal failure. J Clin Invest. 1995;95:1808-1813.
14. Maxwell PH, Osmond MK, Pugh CW, et al. Identification of the renal erythropoietin-producing cells using transgenic mice. Kidney Int. 1993;44:1149-1162.
15. Middleton RJ, Parfrey PS, Foley RN. Left ventricular hypertrophy in the renal patient. J Am Soc Nephrol. 2001;12:1079-1084.
16. Zhou S, Murphy JE, Escobedo JA, Dwarki VJ. Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates. Gene Ther. 1998;5:665-670.
17. Polycythaemia due to hypoxaemia: advantage or disadvantage [review]? Lancet. 1989;2:20-22.
18. Ye X, Rivera VM, Zoltick P, et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science. 1999;283:88-91.
19. Rendahl K, Leff S, Otten G, et al. Regulation of gene expression in vivo following transduction by two separate rAAV vectors. Nat Biotechnol. 1998;16:757-761.
20. Bohl D, Naffakh N, Heard JM. Long-term control of erythropoietin secretion by doxycycline in mice transplanted with engineered primary myoblasts. Nat Med. 1997;3:299-305.
21. Bachmann S, Le Hir M, Eckardt KU. Co-localization of erythropoietin mRNA and ecto-5′-nucleotidase immunoreactivity in peritubular cells of rat renal cortex indicates that fibroblasts produce erythropoietin. J Histochem Cytochem. 1993;41:335-341.
22. 2 tension. Proc Natl Acad Sci U S A. 1995;92:5510-5514.
23. Maxwell PH, Pugh CW, Ratcliffe PJ. Inducible operation of the erythropoietin 3′ enhancer in multiple cell lines: evidence for a widespread oxygen-sensing mechanism. Proc Natl Acad Sci U S A. 1993;90:2423-2427.
24. Wang GL, Semenza GL. General involvement of hypoxia-inducible factor 1 in transcriptional response to hypoxia. Proc Natl Acad Sci U S A. 1993;90:4304-4308.
25. Semenza GL. HIF-1 and human disease: one highly involved factor. Genes Dev. 2000;14:1983-1991.
26. Binley K, Iqball S, Kingsman A, Kingsman S, Naylor S. An adenoviral vector regulated by hypoxia for the treatment of ischaemic disease and cancer. Gene Ther. 1999;6:1721-1727.
27. Boast K, Binley K, Iqball S, et al. Characterization of physiologically regulated vectors for the treatment of ischemic disease. Hum Gene Ther. 1999;10:2197-2208.
28. Griffiths L, Binley K, Iqball S, et al. The macrophage: a novel system to deliver gene therapy to pathological hypoxia. Gene Ther. 2000;7:255-262.
29. Post D, Van Meir E. Generation of bidirectional hypoxia/HIF-responsive expression vectors to target gene expression to hypoxic cells. Gene Ther. 20001;8:1801-1807.
30. Lynch CM. Gene therapy for hemophilia. Curr Opin Mol Ther. 1999;1:493-499.
31. Zhang X, De Alwis M, Hart SL, et al. High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H. Hum Gene Ther. 1999;10:2527-2537.
32. 3H-thymidine incorporation into spleen cells from phenylhydrazine treated mice. Exp Hematol. 1983;11:649-660.
33. Kina T, Ikuta K, Takayama E, et al. The monoclonal antibody TER-119 recognizes a molecule associated with glycophorin A and specifically marks the late stages of murine erythroid lineage. Br J Haematol. 2000;109:280-287.
34. Raja KB, Maxwell PH, Ratcliffe PJ, Salisbury JR, Simpson RJ, Peters TJ. Iron metabolism in transgenic mice with hypoplastic anaemia due to incomplete deficiency of erythropoietin. Br J Haematol. 1997;96:248-253.
35. Setoguchi Y, Danel C, Crystal RG. Stimulation of erythropoiesis by in vivo gene therapy: physiologic consequences of transfer of the human erythropoietin gene to experimental animals using an adenovirus vector. Blood. 1994;84:2946-2953.
36. Rinsch C, Regulier E, Deglon N, Dalle B, Beuzard Y, Aebischer P. A gene therapy approach to regulated delivery of erythropoietin as a function of oxygen tension. Hum Gene Ther. 1997;8:1881-1889.
37. Erslev AJ, Caro J, Besarab A. Why the kidney? Nephron. 1985;41:213-216.
38. Foley RN, Parfrey PS, Harnett JD, Kent GM, Murray DC, Barre PE. The prognostic importance of left ventricular geometry in uremic cardiomyopathy. J Am Soc Nephrol. 1995;5:2024-2031.
39. Villeval JL, Rouyer-Fessard P, Blumenfeld N, Henri A, Vainchenker W, Beuzard Y. Retrovirus-mediated transfer of the erythropoietin gene in hematopoietic cells improves the erythrocyte phenotype in murine beta-thalassemia. Blood. 1994;84:928-933.
40. 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.