Gene therapy for canine leukocyte adhesion deficiency with lentiviral vectors using the murine stem cell virus and human phosphoglycerate kinase promoters

Human Gene Therapy

Volumn 22, Issue 6, 2011, Pages 689-696

  Hunter, Michael J ^a   Zhao, Huifen ^b   Tuschong, Laura M ^a   Bauer Jr , Thomas R ^a   Burkholder, Tanya H ^c   Persons, Derek A ^b   Hickstein, Dennis D ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

^b ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^c NATIONAL INSTITUTES OF HEALTH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD18 ANTIGEN; CD34 ANTIGEN; LENTIVIRUS VECTOR; PHOSPHOGLYCERATE KINASE; RETROVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIGEN EXPRESSION; ARTICLE; BONE MARROW CELL; CD3+ T LYMPHOCYTE; CONTROLLED STUDY; ENHANCER REGION; EXPERIMENTAL DOG; FLOW CYTOMETRY; GENE DOSAGE; HEMATOPOIETIC STEM CELL; HUMAN IMMUNODEFICIENCY VIRUS 1; LEUKOCYTE ADHESION DEFICIENCY; LYMPHOCYTE PROLIFERATION; MURINE STEM CELL VIRUS; NEUTROPHIL; NONHUMAN; NONMYELOABLATIVE CONDITIONING; PHENOTYPE; PROMOTER REGION; RADIATION DOSE; RETROVIRUS; STEM CELL GENE THERAPY; TREATMENT OUTCOME; WHOLE BODY RADIATION;

ANIMALS; ANTIGENS, CD18; DOG DISEASES; DOGS; GENE THERAPY; GENETIC VECTORS; HIV-1; HUMANS; LENTIVIRUS; LEUKOCYTE-ADHESION DEFICIENCY SYNDROME; MICE; PHOSPHOGLYCERATE KINASE; PROMOTER REGIONS, GENETIC; STEM CELLS; TRANSDUCTION, GENETIC; WHOLE-BODY IRRADIATION;

ANIMALIA; BACTERIA (MICROORGANISMS); CANIS FAMILIARIS; HUMAN IMMUNODEFICIENCY VIRUS 1; MURINAE;

EID: 79958231379     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2010.130     Document Type: Article

References (26)

1. Aiuti, A., Slavin, S., Aker, M., et al. (2002). Correction of ADA-SCID by stem cell gene therapy combined with non-myeloablative conditioning. Science 296, 2410-2413. (Pubitemid 34734219)
2. Anderson, D.C., and Springer, T.A. (1987). Leukocyte adhesion deficiency: an inherited defect in the Mac-1, LFA-1, and p150,95 glycoproteins. Annu. Rev. Med. 38, 175-194.
3. Anderson, D.C., Schmalsteig, F.C., Finegold, M.J., et al. (1985). The severe and moderate phenotypes of heritable Mac-1, LFA-1 deficiency: their quantitative definition and relation to leukocyte dysfunction and clinical features. J. Infect. Dis. 152, 668-689. (Pubitemid 16215942)
4. Bauer, T.R., Jr., Allen, J.M., Hai, M., et al. (2008). Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors. Nat. Med. 14, 93-97.
5. Bauer, T.R., Jr., Gu, Y.C., Tuschong, L.M., et al. (2005). Non-myeloablative hematopoietic stem cell transplantation corrects the disease phenotype in the canine model of leukocyte adhesion deficiency. Exp. Hematol. 33, 706-712. (Pubitemid 40725837)
6. Bauer, T.R., Jr., Hai, M., Tuschong, L.M., et al. (2006). Correction of the disease phenotype in canine leukocyte adhesion deficiency using ex-vivo hematopoietic stem cell gene therapy. Blood 108, 1767-1769. (Pubitemid 44316148)
7. Cavazzana-Calvo, M., Hacein-Bey, S., de Saint Basile, G., et al. (2000). Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669-672. (Pubitemid 30241569)
8. Dull, T., Zufferey, R., Kelly, M., et al. (1998). A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72, 8463-8471. (Pubitemid 28471781)
9. Hacein-Bey-Abina, S., Garrigue, A., Wang, G.P., et al. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118, 3132-3142.
10. Hawley, R.G., Hawley, T.S., Fong, A.Z., et al. (1996). Thrombo-poietic potential and serial repopulating ability of murine hematopoietic stem cells constitutively expressing interleukin 11. Proc. Natl. Acad. Sci. U. S. A. 93, 10297-302. (Pubitemid 26314616)
11. Kijas, J.M.H., Bauer, T.R., Jr., Gäfvert, S., et al. (1999). A missense mutation in the b-2 integrin gene (ITGB2) causes canine leukocyte adhesion deficiency. Genomics 61, 101-107. (Pubitemid 29487626)
12. Kim, Y.J., Kim, Y.S., Larochelle, A., et al. (2009). Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34\+ cells. Blood 113, 5434-5443.
13. Kishimoto, T.K., Hollander, N., Roberts, T.M., et al. (1987). Heterogenous mutations in the b subunit common to the LFA-1, Mac-1, and p150,95 glycoproteins cause leukocyte adhesion deficiency. Cell 50, 193-202.
14. Lewis, P.F., and Emerman, M. (1994). Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J. Virol. 68, 510-516.
15. Lim, B., Apperley, J.F., Orkin, S.H., and Williams, D.A. (1989). Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells. Proc. Natl. Acad. Sci. U. S. A. 86, 8892-8896. (Pubitemid 20010331)
16. Modlich, U., Navarro, S., Zychlinski, D., et al. (2009). Insertional transformation of hematopoietic cells by self-inactivating len-tiviral and gammaretroviral vectors. Mol. Ther. 17, 1919-1928.
17. Montini, E., Cesana, D., Schmidt, M., et al. (2006). Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat. Biotechnol. 24, 687-696. (Pubitemid 43882120)
18. Naldini, L., Blomer, U., Gallay, P., et al. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263-267. (Pubitemid 26119138)
19. Nelson, E.J., Tuschong, L.M., Hunter, M.J., et al. (2010). Lentiviral vectors incorporating a human elongation factor 1alpha promoter for the treatment of canine leukocyte adhesion deficiency. Gene Ther. 17:672-677.
20. Ott, M.G., Schmidt, M., Schwarzwaelder, K., et al. (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 12, 401-409.
21. Ramezani, A., Hawley, T.S., and Hawley, R.G. (2000). Lentiviral vectors for enhanced gene expression in human hematopoietic cells. Mol. Ther. 2, 458-469.
22. Salmon, P., Kindler, V., Ducrey, O., et al. (2000). High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. Blood 96, 3392-3398.
23. Trowald-Wigh, G., Ekman, S., Hansson, K., et al. (2000). Clinical, radiological and pathological features of 12 Irish setters with canine leucocyte adhesion deficiency. J. Small Anim. Pract. 41, 211-217.
24. Wu, X., Li, Y., Crise, B., and Burgess, S.M. (2003). Transcription start regions in the human genome are favored targets for MLV integration. Science 300, 1749-1751. (Pubitemid 36712571)
25. Zhao, H., Pestina, T.I., Nasimuzzaman, M., et al. (2009). Amelioration of murine beta-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both gamma-globin and the MGMT drug-resistance gene. Blood 113, 5747-5756.
26. Zychlinski, D., Schambach, A., Modlich, U., et al. (2008). Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol. Ther. 16, 718-725. (Pubitemid 351426173)