Restriction of transgene expression to the B-lymphoid progeny of human lentivirally transduced CD34+ cells

Molecular Therapy

Volumn 10, Issue 1, 2004, Pages 45-56

  Moreau, Thomas ^a   Bardin, Florence ^a   Imbert, Jean ^b   Chabannon, Christian ^a   Tonnelle, Cécile ^a  

^a INSTITUT PAOLI CALMETTES   (France)

^b INSERM   (France)

Author keywords

B specificity; CD19 promoter; Gene transfer; Hematopoietic stem cells; Lentiviral vectors; Transcriptional regulation

Indexed keywords

CD19 ANTIGEN; CD34 ANTIGEN; ERYTHROPOIETIN RECEPTOR; GREEN FLUORESCENT PROTEIN; IMMUNOGLOBULIN M; LENTIVIRUS VECTOR;

ANIMAL CELL; ARTICLE; B LYMPHOCYTE; BONE MARROW CELL; CELL DIFFERENTIATION; CELL MATURATION; CONTROLLED STUDY; ERYTHROID CELL; GENE EXPRESSION; GENE INSERTION; GENE SEQUENCE; GENE TARGETING; GENE TRANSFER; GENETIC CONSERVATION; GENETIC TRANSCRIPTION; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; MOUSE; NATURAL KILLER CELL; NONHUMAN; NUCLEOTIDE SEQUENCE; PROMOTER REGION; REAL TIME POLYMERASE CHAIN REACTION; SEQUENCE HOMOLOGY; STEM CELL GENE THERAPY; T LYMPHOCYTE; TRANSGENE; UMBILICAL CORD BLOOD;

ANIMALS; ANTIGENS, CD19; ANTIGENS, CD34; B-LYMPHOCYTES; CELL LINE; FETAL BLOOD; GENE EXPRESSION; GENES, REPORTER; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC STEM CELLS; HUMANS; LENTIVIRUS; MICE; MICE, INBRED NOD; MICE, SCID; REGULATORY SEQUENCES, NUCLEIC ACID; TRANSDUCTION, GENETIC; TRANSGENES; TRANSPLANTATION, HETEROLOGOUS;

INSERTION SEQUENCES;

EID: 3042856076     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.04.005     Document Type: Article

References (51)

1. Baum C. et al. Side effects of retroviral gene transfer into hematopoietic stem cells Blood 101 2003 2099-2114
2. Naldini L. et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector Science 272 1996 263-267
3. Reiser J. et al. Transduction of nondividing cells using pseudotyped defective high-titer HIV type 1 particles Proc. Natl. Acad. Sci. USA 93 1996 15266-15271
4. + NOD/SCID-repopulating cells Blood 99 2002 709-712
5. Sirven A. et al. Enhanced transgene expression in cord blood CD34(+)-derived hematopoietic cells, including developing T cells and NOD/SCID mouse repopulating cells, following transduction with modified trip lentiviral vectors Mol. Ther. 3 2001 438-448
6. Piacibello W. et al. Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice: nonobese diabetic/severe combined immunodeficient Blood 100 2002 4391-4400
7. Ailles L. et al. Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells Mol. Ther. 6 2002 615-626
8. Woods N.B. et al. Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells Blood 96 2000 3725-3733
9. - bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors Mol. Ther. 1 2000 566-573
10. Indraccolo S. et al. Effects of CD2 locus control region sequences on gene expression by retroviral and lentiviral vectors Blood 98 2001 3607-3617
11. + cells using lentiviral vectors with regulatory sequences from the CD4 gene Blood 101 2003 3416-3423
12. Cui Y. et al. Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells Blood 99 2002 399-408
13. Gough P.J. Raines E.W. Gene therapy of apolipoprotein E-deficient mice using a novel macrophage-specific retroviral vector Blood 101 2003 485-491
14. Imren S. et al. Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells Proc. Natl. Acad. Sci. USA 99 2002 14380-14385
15. May C. Rivella S. Chadburn A. Sadelain M. Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene Blood 99 2002 1902-1908
16. Pawliuk R. et al. Correction of sickle cell disease in transgenic mouse models by gene therapy Science 294 2001 2368-2371
17. Kozmik Z. Wang S. Dorfler P. Adams B. Busslinger M. The promoter of the CD19 gene is a target for the B-cell-specific transcription factor BSAP Mol. Cell. Biol. 12 1992 2662-2672
18. Tedder T.F. Inaoki M. Sato S. The CD19-CD21 complex regulates signal transduction thresholds governing humoral immunity and autoimmunity Immunity 6 1997 107-118
19. Engel P. et al. Abnormal B lymphocyte development, activation, and differentiation in mice that lack or overexpress the CD19 signal transduction molecule Immunity 3 1995 39-50
20. Kleindienst P. Chretien I. Winkler T. Brocker T. Functional comparison of thymic B cells and dendritic cells in vivo Blood 95 2000 2610-2616
21. Maas A. Dingjan G.M. Grosveld F. Hendriks R.W. Early arrest in B cell development in transgenic mice that express the E41K Bruton's tyrosine kinase mutant under the control of the CD19 promoter region J. Immunol. 162 1999 6526-6533
22. Zhou L.J. et al. Tissue-specific expression of the human CD19 gene in transgenic mice inhibits antigen-independent B-lymphocyte development Mol. Cell. Biol. 14 1994 3884-3894
23. Riva A. Wilson G.L. Kehrl J.H. In vivo footprinting and mutational analysis of the proximal CD19 promoter reveal important roles for an SP1/Egr-1 binding site and a novel site termed the PyG box J. Immunol. 159 1997 1284-1292
24. Pennacchio L.A. Rubin E.M. Genomic strategies to identify mammalian regulatory sequences Nat. Rev. Genet. 2 2001 100-109
25. Quandt K. Frech K. Karas H. Wingender E. Werner T. MatInd and MatInspector: new fast and versatile tools for detection of consensus matches in nucleotide sequence data Nucleic Acids Res. 23 1995 4878-4884
26. Kustikova O.S. et al. Dose finding with retroviral vectors: correlation of retroviral vector copy numbers in single cells with gene transfer efficiency in a cell population Blood 102 2003 3934-3937
27. Woods N.B. et al. Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis Blood 101 2003 1284-1289
28. Janssens W. et al. Efficiency of onco-retroviral and lentiviral gene transfer into primary mouse and human B-lymphocytes is pseudotype dependent Hum. Gene Ther. 14 2003 263-276
29. Bovia F. et al. Efficient transduction of primary human B lymphocytes and nondividing myeloma B cells with HIV-1-derived lentiviral vectors Blood 101 2003 1727-1733
30. Kikuchi K. et al. Appearance of human plasma cells following differentiation of human B cells in NOD/SCID mouse spleen Clin. Dev. Immunol. 10 2003 197-202
31. Fluckiger A.C. et al. In vitro reconstitution of human B-cell ontogeny: from CD34(+) multipotent progenitors to Ig-secreting cells Blood 92 1998 4509-4520
32. Tarte K. et al. Generation of polyclonal plasmablasts from peripheral blood B cells: a normal counterpart of malignant plasmablasts Blood 100 2002 1113-1122
33. Lutzko C. Senadheera D. Skelton D. Petersen D. Kohn D.B. Lentivirus vectors incorporating the immunoglobulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes J. Virol. 77 2003 7341-7351
34. Gisler R. Akerblad P. Sigvardsson M. A human early B-cell factor-like protein participates in the regulation of the human CD19 promoter Mol. Immunol. 36 1999 1067-1077
35. Dittmer J. The biology of the Ets1 proto-oncogene Mol. Cancer 2 2003 29
36. Maier H. Colbert J. Fitzsimmons D. Clark D.R. Hagman J. Activation of the early B-cell-specific mb-1 (Ig-alpha) gene by Pax-5 is dependent on an unmethylated Ets binding site Mol. Cell. Biol. 23 2003 1946-1960
37. Ballow M. Primary immunodeficiency disorders: antibody deficiency J. Allergy Clin. Immunol. 109 2002 581-591
38. Kohn D.B. Sadelain M. Glorioso J.C. Occurrence of leukaemia following gene therapy of X-linked SCID Nat. Rev. Cancer 3 2003 477-488
39. Goodman P.A. Wood C.M. Vassilev A.O. Mao C. Uckun F.M. Defective expression of Bruton's tyrosine kinase in acute lymphoblastic leukemia Leuk. Lymphoma 44 2003 1011-1018
40. Kersseboom R. et al. Bruton's tyrosine kinase cooperates with the B cell linker protein SLP-65 as a tumor suppressor in pre-B cells J. Exp. Med. 198 2003 91-98
41. - B cell subsets activated through their antigen receptors or CD40 antigens Eur. J. Immunol. 22 1992 2831-2839
42. Hubbard T. et al. The Ensembl genome database project Nucleic Acids Res. 30 2002 38-41
43. Schwartz S. et al. PipMaker - a Web server for aligning two genomic DNA sequences Genome Res. 10 2000 577-586
44. Rocancourt D. Bonnerot C. Jouin H. Emerman M. Nicolas J.F. Activation of a beta-galactosidase recombinant provirus: application to titration of human immunodeficiency virus (HIV) and HIV-infected cells J. Virol. 64 1990 2660-2668
45. Kuhlcke K. et al. Highly efficient retroviral gene transfer based on centrifugation-mediated vector preloading of tissue culture vessels Mol. Ther. 5 2002 473-478
46. Metcalf D. Techniques for the clonal culture of hematopoietic cells Techniques and Applications 1984 Elsevier New York
47. Hacein-Bey S. et al. Optimization of retroviral gene transfer protocol to maintain the lymphoid potential of progenitor cells Hum. Gene Ther. 12 2001 291-301
48. Tonnelle C. et al. Forced expression of the Ikaros 6 isoform in human placental blood CD34(+) cells impairs their ability to differentiate toward the B-lymphoid lineage Blood 98 2001 2673-2680
49. Shultz L.D. et al. Multiple defects in innate and adaptive immunologic function in NOD/LtSz-scid mice J. Immunol. 154 1995 180-191
50. Kerre T.C. et al. Adapted NOD/SCID model supports development of phenotypically and functionally mature T cells from human umbilical cord blood CD34(+) cells Blood 99 2002 1620-1626
51. Tanaka T. et al. A novel monoclonal antibody against murine IL-2 receptor beta-chain: characterization of receptor expression in normal lymphoid cells and EL-4 cells J. Immunol. 147 1991 2222-2228