Restoration of lymphoid populations in a murine model of X-linked severe combined immunodeficiency by a gene-therapy approach

Blood

Volumn 94, Issue 9, 1999, Pages 3027-3036

  Lo, Mindy ^b   Bloom, Michael L ^b   Imada, Kazunori ^b   Berg, Maria ^b   Bollenbacher, Julie M ^b   Bloom, Eda T ^b   Kelsall, Brian L ^b   Leonard, Warren J ^a  

^a Building 10   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

CYTOKINE RECEPTOR; INTERLEUKIN 2 RECEPTOR; INTERLEUKIN 7 RECEPTOR;

ANIMAL CELL; ANIMAL MODEL; ARTICLE; COMBINED IMMUNODEFICIENCY; CONTROLLED STUDY; GENE EXPRESSION SYSTEM; GENE THERAPY; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; IMMUNOGLOBULIN BLOOD LEVEL; LEUKOCYTE COUNT; LYMPHOCYTE PROLIFERATION; LYMPHOID CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; X CHROMOSOME LINKED DISORDER;

EID: 0033229710     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v94.9.3027     Document Type: Article

References (40)

1. Conley ME: Molecular approaches to analysis of X-linked immunodeficiencies. Annu Rev Immunol 10:215, 1992
2. Leonard WJ: The molecular basis of X-linked combined immunodeficiency: Defective cytokine receptor signaling. Annu Rev Med 47:229, 1996
3. Buckley RH, Schiff RI, Schiff SE, Markert ML, Williams LW, Harville TO, Roberts JL, Puck JM: Human severe combined immunodeficiency: Genetic, phenotypic, and functional diversity in 108 infants. J Pediatr 130:378, 1997
4. Noguchi M, Yi H, Rosenblatt HM, Filipovich AH, Adelstein S, Modi WS, McBride OW, Leonard WJ: Interleukin-2 receptor γ chain mutation results in X-linked severe combined immunodeficiency in humans. Cell 73:147, 1993
5. Kondo M, Takeshita T, Ishii N, Nakamura M, Watanabe S, Arai K, Sugamura K: Sharing of the interleukin-2 (IL-2) receptor γ chain between receptors for IL-2 and IL-4. Science 262:1874, 1993
6. Noguchi M, Nakamura Y, Russell SM, Ziegler SF, Tsang M, Cao X, Leonard WJ: Interleukin-2 receptor γ chain: A functional component of the interleukin-7 receptor. Science 262:1877, 1993
7. Russell SM, Keegan AD, Harada N, Nakamura Y, Noguchi M, Leland P, Friedmann MC, Miyajima A, Puri RK, Paul WE, Leonard WJ: Interleukin-2 receptor γ chain: A functional component of the interleukin-4 receptor. Science 262:1880, 1993
8. Kondo M, Takeshita T, Higuchi M, Nakamura M, Sudo T, Nishikawa S, Sugamura K: Functional participation of the IL-2 receptor γ chain in IL-7 receptor complexes. Science 263:1453, 1994
9. c chains with Jak1 and Jak3: Implications for XSCID and XCID. Science 266:1042, 1994
10. Kimura Y, Takeshita T, Kondo M, Ishii N, Nakamura M, Van Snick J, Sugamura K: Sharing of the IL-2 receptor γ chain with the functional IL-9 receptor complex. Int Immunol 7:115, 1995
11. Giri JG, Ahdieh M, Eisenman J, Shanebeck K, Grabstein K, Kumatki S, Namen A, Park LS, Cosman D, Anderson D: Utilization of the β and γ chains of the IL-2 receptor by the novel cytokine IL-15. EMBO J 13:2822, 1994
12. + severe combined immunodeficiency. Nat Genet 20:394, 1998
13. Lodolce JP, Boone DL, Chai S, Swain RE, Dassopoulos T, Trettin S, Ma A: IL-15 receptor maintains lymphoid homeostasis by supporting lymphocyte homing and proliferation. Immunity 9:669, 1998
14. Dror Y, Gallagher R, Wara DW, Colombe BW, Merino A, Benkerrou M, Cowan MJ: Immune reconstitution in severe combined immunodeficiency disease after lectin-treated haplocompatible bone marrow transplantation. Blood 81:2021, 1993
15. van Leeuwen JE, van Tol MJ, Joosten AM, Schellekens PT, van den Bergh RL, Waaijer JL, Oudeman-Gruber NJ, van der Weijden-Ragas CP, Roos MT, Gerritsen EJ: Relationship between patterns of engraftment in peripheral blood and immune reconstitution after allogenic bone marrow transplantation for severe combined immunodeficiency. Blood 84:3936, 1994
16. Qazilbash MH, Walsh CE, Russell SM, Noguchi M, Mann MM, Leonard WJ, Liu JM: Retroviral vector for gene therapy of X-linked severe combined immunodeficiency syndrome. J Hematother 4:91, 1995
17. Candotti F, Johnston JA, Puck JM, Sugamura K, O'Shea JJ, Blaese RM: Retroviral-mediated gene correction for X-linked severe combined immunodeficiency. Blood 87:3097, 1996
18. c gene. Blood 87:3103, 1996
19. c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function. Blood 87:3108, 1996
20. Whitman T, Haskins ME, Henthorn PS, Kraszewski JN, Kleiman SE, Seidel NE, Bodine DM, Puck JM: Retroviral marking of canine bone marrow: Long-term, high-level expression of human interleukin-2 receptor common gamma chain in canine lymphocytes. Blood 92:1565, 1998
21. c(-) X-linked severe combined immunodeficiency hematopoietic progenitor cells in murine fetal thymic organ cultures. Blood 92:4090, 1998
22. Bunting KD, Sangster MY, Ihle JN, Sorrentino BP: Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer. Nat Med 4:58, 1998
23. Macchi P, Villa A, Gillani S, Sacco MG, Frattini A, Porta F, Ugazio AG, Johnston JA, Candotti F, O'Shea JJ, Vezzoni P, Notarangelo LD: Mutations of Jak-3 gene in patients with autosomal severe combined immune deficiency (SCID). Nature 377:65, 1995
24. Russell SM, Tayebi N, Nakajima H, Riedy MC, Roberts JL, Aman MJ, Migone TS, Noguchi M, Markert ML, Buckley RH, O'Shea JJ, Leonard WJ: Mutation of Jak3 in a patient with SCID: Essential role of Jak3 in lymphoid development. Science 270:797, 1995
25. DiSanto JP, Muller W, Guy-Grand D, Fischer A, Rajewsky K: Lymphoid development in mice with a targeted deletion of the interleukin-2 receptor γ chain. Proc Natl Acad Sci USA 92:377, 1995
26. Cao X, Shores EW, Hu-Li J, Anver MR, Kelsall BL, Russell SM, Drago J, Noguchi M, Grinberg A, Bloom ET, Paul WE, Katz SI, Love PE, Leonard WJ: Defective lymphoid development in mice lacking expression of the common cytokine receptor γ chain. Immunity 2:223, 1995
27. Ohbo K, Suda T, Hashiyama M, Mantani A, Ikebe M, Miyakawa K, Moriyama M, Nakamura M, Katsuki M, Takahashi K, Yamamura K, Sugamura K: Modulation of hematopoiesis in mice with a truncated mutant of the interleukin-2 receptor γ chain. Blood 87:956, 1996
28. Riviere I, Brose K, Mulligan RC: Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells. Proc Natl Acad Sci USA 92:6733, 1995
29. Nakajima H, Shores EW, Noguchi M, Leonard WJ: The common cytokine receptor γ chain plays an essential role in regulating lymphoid homeostasis. J Exp Med 185:189, 1997
30. Nakajima H, Leonard WJ: Impaired peripheral deletion of activated T cells in mice lacking the common cytokine receptor γ chain. J Immunol 159:4737, 1997
31. Takebe Y, Seiki M, Fujisawa J-i, Hoy P, Yokota K, Aral K-i, Yoshida M, Arai N. SRα promoter: An efficient and versatile mammalian cDNA expression system composed of the Simian virus 40 early promoter and the R-U5 segment of human T-cell leukemia virus type 1 long terminal repeat. Mol Cell Biol 8:466, 1988
32. Goodwin RG, Friend D, Ziegler SF, Jerzy R, Falk BA, Gimpel S, Cosman D, Dower SK, March CJ, Namen AE: Cloning of the human and murine interleukin-7 receptors: Demonstration of a soluble form and homology to a new receptor superfamily. Cell 60:941, 1990
33. Nakajima H, Liu X-W, Wynshaw-Boris A, Rosenthal LA, Imada K, Finbloom DS, Hennighausen L, Leonard WJ: An indirect effect of Stat5 in IL-2-induced proliferation: A critical role for Stat5a in IL-2 mediated IL-2 receptor a chain gene expression. Immunity 7:691, 1997
34. Lin J-X, Mietz J, Modi WS, John S, Leonard WJ: Cloning of human Stat5B: Reconstitution of interleukin-2-induced Stat5A and Stat5B DNA binding activity in COS-7 cells. J Biol Chem 271:10738, 1996
35. Taniguchi T: Cytokine signaling through nonreceptor protein tyrosine kinases. Science 268:251, 1995
36. Otani H, Siegel JP, Erdos M, Gnarra JR, Toledano MB, Sharon M, Mostowski H, Feinberg MB, Pierce JH, Leonard WJ: Interleukin (IL)-2 and IL-3 induce distinct but overlapping responses in murine IL-3-dependent 32D cells transduced with human IL-2 receptor β chain: Involvement of tyrosine kinase(s) other than p561ck. Proc Natl Acad Sci USA 89:2789, 1992
37. Zhu M-h, Berry JA, Russell SM, Leonard WJ: Delineation of the regions of interleukin-2 (IL-2) receptor β chain important for association of Jak1 and Jak3: Jak1-independent functional recruitment of Jak3 to IL-2Rβ. J Biol Chem 273:10719, 1998
38. John S, Robbins CM, Leonard WJ: An IL-2 response element in the human IL-2 receptor a chain promoter is a composite element that binds Stat5, Elf-1, HMG-I(Y), and a GATA family protein. EMBO J 15:5627, 1996
39. Mardiney M, Malech HL: Enhanced engraftment of hematopoietic progenitor cells in mice treated with granulocyte colony-stimulating factor before low-dose irradiation: Implications for gene therapy. Blood 87:4049, 1996
40. Leonard WJ: Type I cytokines and interferons and their receptors, in Paul WE (ed): Fundamental Immunology (ed 4). Philadelphia, PA, Lippincott Raven, 1999, p 741