Transgene expression in various organs post BM-HSC transplantation

Stem Cell Research

Volumn 12, Issue 1, 2014, Pages 209-221

  Wang, Nan ^a   Rajasekaran, Narendiran ^a   Hou, Tieying ^a   Mellins, Elizabeth D ^a  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD11B ANTIGEN; GLYCOPROTEIN P 15095; LENTIVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; B LYMPHOCYTE; BLOOD; BONE MARROW; BONE MARROW DERIVED HEMATOPOIETIC STEM CELL; CELL ISOLATION; CELL MATURATION; CELL PERMEABILIZATION; CONTROLLED STUDY; DENDRITIC CELL; FLOW CYTOMETRY; GENE EXPRESSION; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; INSULIN DEPENDENT DIABETES MELLITUS; LENTIVIRINAE; LYMPH NODE; MACROPHAGE; MONOCYTE; MOUSE; NONHUMAN; POINT MUTATION; PRIORITY JOURNAL; SPLEEN CELL; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

AA; AB; ADL; ADRENOLEUKODYSTROPHY; AMINO ACID; ANTIBODY; ANTIGEN PRESENTING CELLS; APC; BM; BONE MARROW; DC; DENDRITIC CELLS; EF1A; ELONGATION FACTOR 1A; GFP; GM-CSF; GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR; GREEN FLUORESCENT PROTEIN; HEMATOPOIETIC STEM CELLS; HSC; II; INTERNAL RIBOSOME ENTRY SITES; INVARIANT CHAIN; IRES; LIN; LINAGE; MAJOR HISTOCOMPATIBILITY COMPLEX CLASS II; MEAN FLUORESCENCE INTENSITIES; MFI; MHCII; MOBILIZED PERIPHERAL BLOOD; MOI; MPB; MSCV; MULTIPLICITY OF INFECTION; MURINE STEM CELL VIRUS; PANCREATIC LYMPH NODES; PLN; T2A; THOSEAASIGNA VIRUS 2A;

ANIMALS; ANTIGEN-PRESENTING CELLS; ANTIGENS, DIFFERENTIATION, B-LYMPHOCYTE; BONE MARROW CELLS; CELLS, CULTURED; GENE EXPRESSION REGULATION; GENES, REPORTER; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HISTOCOMPATIBILITY ANTIGENS CLASS II; LENTIVIRUS; LYMPH NODES; MICE; PROMOTER REGIONS, GENETIC; SPLEEN; TRANSGENES;

EID: 84888073495     PISSN: 18735061     EISSN: 18767753     Source Type: Journal    
DOI: 10.1016/j.scr.2013.10.010     Document Type: Article

References (64)

1. Aguilar S., Scotton C.J., McNulty K., Nye E., Stamp G., Laurent G., Janes S.M. Bone marrow stem cells expressing keratinocyte growth factor via an inducible lentivirus protects against bleomycin-induced pulmonary fibrosis. PLoS One 2009, 4(11):e8013. 10.1371/journal.pone.0008013.
2. Aiuti A., Biasco L., Scaramuzza S., Ferrua F., Cicalese M.P., Baricordi C., Naldini L. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 2013, 341(6148):1233151. 10.1126/science.1233151.
3. Alam C., Valkonen S., Palagani V., Jalava J., Eerola E., Hanninen A. Inflammatory tendencies and overproduction of IL-17 in the colon of young NOD mice are counteracted with diet change. Diabetes 2010, 59(9):2237-2246. 10.2337/db10-0147.
4. Amsellem S., Ravet E., Fichelson S., Pflumio F., Dubart-Kupperschmitt A. Maximal lentivirus-mediated gene transfer and sustained transgene expression in human hematopoietic primitive cells and their progeny. Mol. Ther. 2002, 6(5):673-677.
5. Astrakhan A., Sather B.D., Ryu B.Y., Khim S., Singh S., Humblet-Baron S., Rawlings D.J. Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome. Blood 2012, 119(19):4395-4407. 10.1182/blood-2011-03-340711.
6. Bank A. Hematopoietic stem cell gene therapy: selecting only the best. J. Clin. Invest. 2003, 112(10):1478-1480. 10.1172/JCI20336.
7. Barrette S., Douglas J.L., Seidel N.E., Bodine D.M. Lentivirus-based vectors transduce mouse hematopoietic stem cells with similar efficiency to moloney murine leukemia virus-based vectors. Blood 2000, 96(10):3385-3391.
8. Biffi A., Montini E., Lorioli L., Cesani M., Fumagalli F., Plati T., Naldini L. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 2013, 341(6148):1233158. 10.1126/science.1233158.
9. Buchschacher G.L., Wong-Staal F. Development of lentiviral vectors for gene therapy for human diseases. Blood 2000, 95(8):2499-2504.
10. Cartier N., Hacein-Bey-Abina S., Bartholomae C.C., Veres G., Schmidt M., Kutschera I., Aubourg P. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009, 326(5954):818-823. 10.1126/science.1171242.
11. Chicurel M. Virology. Probing HIV's elusive activities within the host cell. Science 2000, 290(5498):1876-1879.
12. Choi J.K., Hoang N., Vilardi A.M., Conrad P., Emerson S.G., Gewirtz A.M. Hybrid HIV/MSCV LTR enhances transgene expression of lentiviral vectors in human CD34(+) hematopoietic cells. Stem Cells 2001, 19(3):236-246. 10.1634/stemcells.19-3-236.
13. Cui Y., Golob J., Kelleher E., Ye Z., Pardoll D., Cheng L. Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells. Blood 2002, 99(2):399-408.
14. Curtin J.A., Dane A.P., Swanson A., Alexander I.E., Ginn S.L. Bidirectional promoter interference between two widely used internal heterologous promoters in a late-generation lentiviral construct. Gene Ther. 2008, 15(5):384-390. 10.1038/sj.gt.3303105.
15. Davoust N., Vuaillat C., Cavillon G., Domenget C., Hatterer E., Bernard A., Nataf S. Bone marrow CD34+/B220+ progenitors target the inflamed brain and display in vitro differentiation potential toward microglia. FASEB J. 2006, 20(12):2081-2092. 10.1096/fj.05-5593com.
16. Drutman S.B., Kendall J.C., Trombetta E.S. Inflammatory spleen monocytes can upregulate CD11c expression without converting into dendritic cells. J. Immunol. 2012, 188(8):3603-3610. 10.4049/jimmunol.1102741.
17. Ellis J. Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum. Gene Ther. 2005, 16(11):1241-1246. 10.1089/hum.2005.16.1241.
18. Engering A.J., Richters C.D., Fluitsma D.M., van Pelt A.M., Kamperdijk E.W., Hoefsmit E.C., Pieters J. MHC class II and invariant chain biosynthesis and transport during maturation of human precursor dendritic cells. Int. Immunol. 1998, 10(11):1713-1723.
19. Frimpong K., Spector S.A. Cotransduction of nondividing cells using lentiviral vectors. Gene Ther. 2000, 7(18):1562-1569. 10.1038/sj.gt.3301283.
20. Gordon S., Taylor P.R. Monocyte and macrophage heterogeneity. Nat. Rev. Immunol. 2005, 5(12):953-964. 10.1038/nri1733.
21. Hausmann D.H., Yu B., Hausmann S., Wucherpfennig K.W. pH-dependent peptide binding properties of the type I diabetes-associated I-Ag7 molecule: rapid release of CLIP at an endosomal pH. J. Exp. Med. 1999, 189(11):1723-1734.
22. Heckl D., Wicke D.C., Brugman M.H., Meyer J., Schambach A., Busche G., Modlich U. Lentiviral gene transfer regenerates hematopoietic stem cells in a mouse model for Mpl-deficient aplastic anemia. Blood 2011, 117(14):3737-3747. 10.1182/blood-2010-09-308262.
23. Hu B., Tai A., Wang P. Immunization delivered by lentiviral vectors for cancer and infectious diseases. Immunol. Rev. 2011, 239(1):45-61. 10.1111/j.1600-065X.2010.00967.x.
24. Iwata Y., Furuichi K., Kitagawa K., Hara A., Okumura T., Kokubo S., Wada T. Involvement of CD11b+GR-1 low cells in autoimmune disorder in MRL-Fas lpr mouse. Clin. Exp. Nephrol. 2010, 14(5):411-417. 10.1007/s10157-010-0309-9.
25. Kahn J., Byk T., Jansson-Sjostrand L., Petit I., Shivtiel S., Nagler A., Lapidot T. Overexpression of CXCR4 on human CD34+ progenitors increases their proliferation, migration, and NOD/SCID repopulation. Blood 2004, 103(8):2942-2949. 10.1182/blood-2003-07-2607.
26. Kaminitz A., Yolcu E.S., Mizrahi K., Shirwan H., Askenasy N. Killer Treg cells ameliorate inflammatory insulitis in non-obese diabetic mice through local and systemic immunomodulation. Int. Immunol. 2013, 25(8):485-494. 10.1093/intimm/dxt016.
27. Kim Y.J., Kim Y.S., Larochelle A., Renaud G., Wolfsberg T.G., Adler R., Persons D.A. Sustained high-level polyclonal hematopoietic marking and transgene expression 4years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34+ cells. Blood 2009, 113(22):5434-5443. 10.1182/blood-2008-10-185199.
28. Klagge I., Kopp U., Koch N. Granulocyte-macrophage colony-stimulating factor elevates invariant chain expression in immature myelomonocytic cell lines. Immunology 1997, 91(1):114-120.
29. Klug C.A., Cheshier S., Weissman I.L. Inactivation of a GFP retrovirus occurs at multiple levels in long-term repopulating stem cells and their differentiated progeny. Blood 2000, 96(3):894-901.
30. Kutner R.H., Zhang X.Y., Reiser J. Production, concentration and titration of pseudotyped HIV-1-based lentiviral vectors. Nat. Protoc. 2009, 4(4):495-505. 10.1038/nprot.2009.22.
31. LeBien T.W., Tedder T.F. B lymphocytes: how they develop and function. Blood 2008, 112(5):1570-1580. 10.1182/blood-2008-02-078071.
32. Levasseur D.N., Ryan T.M., Pawlik K.M., Townes T.M. Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells. Blood 2003, 102(13):4312-4319. 10.1182/blood-2003-04-1251.
33. Lu R., Neff N.F., Quake S.R., Weissman I.L. Tracking single hematopoietic stem cells in vivo using high-throughput sequencing in conjunction with viral genetic barcoding. Nat. Biotechnol. 2011, 29(10):928-933. 10.1038/nbt.1977.
34. Marodon G., Mouly E., Blair E.J., Frisen C., Lemoine F.M., Klatzmann D. Specific transgene expression in human and mouse CD4+ cells using lentiviral vectors with regulatory sequences from the CD4 gene. Blood 2003, 101(9):3416-3423. 10.1182/blood-2002-02-0578.
35. Martin P., del Hoyo G.M., Anjuere F., Ruiz S.R., Arias C.F., Marin A.R., Ardavin C. Concept of lymphoid versus myeloid dendritic cell lineages revisited: both CD8alpha(-) and CD8alpha(+) dendritic cells are generated from CD4(low) lymphoid-committed precursors. Blood 2000, 96(7):2511-2519.
36. Matza D., Lantner F., Bogoch Y., Flaishon L., Hershkoviz R., Shachar I. Invariant chain induces B cell maturation in a process that is independent of its chaperonic activity. Proc. Natl. Acad. Sci. U. S. A. 2002, 99(5):3018-3023. 10.1073/pnas.052703299.
37. Moreno-Carranza B., Gentsch M., Stein S., Schambach A., Santilli G., Rudolf E., Grez M. Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells. Gene Ther. 2009, 16(1):111-118. 10.1038/gt.2008.143.
38. Mostoslavsky G., Kotton D.N., Fabian A.J., Gray J.T., Lee J.S., Mulligan R.C. Efficiency of transduction of highly purified murine hematopoietic stem cells by lentiviral and oncoretroviral vectors under conditions of minimal in vitro manipulation. Mol. Ther. 2005, 11(6):932-940. 10.1016/j.ymthe.2005.01.005.
39. Mostoslavsky G., Fabian A.J., Rooney S., Alt F.W., Mulligan R.C. Complete correction of murine Artemis immunodeficiency by lentiviral vector-mediated gene transfer. Proc. Natl. Acad. Sci. U. S. A. 2006, 103(44):16406-16411. 10.1073/pnas.0608130103.
40. Nagasawa T. Microenvironmental niches in the bone marrow required for B-cell development. Nat. Rev. Immunol. 2006, 6(2):107-116. 10.1038/nri1780.
41. Nikolic T., Bouma G., Drexhage H.A., Leenen P.J. Diabetes-prone NOD mice show an expanded subpopulation of mature circulating monocytes, which preferentially develop into macrophage-like cells in vitro. J. Leukoc. Biol. 2005, 78(1):70-79. 10.1189/jlb.1104662.
42. Perumbeti A., Higashimoto T., Urbinati F., Franco R., Meiselman H.J., Witte D., Malik P. A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction. Blood 2009, 114(6):1174-1185. 10.1182/blood-2009-01-201863.
43. Pierre P., Mellman I. Developmental regulation of invariant chain proteolysis controls MHC class II trafficking in mouse dendritic cells. Cell 1998, 93(7):1135-1145.
44. Rajasekaran N., Wang N., Truong P., Rinderknecht C., Macaubas C., Beilhack G.F., Mellins E.D. Host-derived CD4+ T cells attenuate stem cell-mediated transfer of autoimmune arthritis in lethally irradiated C57BL/6.g7 mice. Arthritis Rheum. 2013, 65(3):681-692. 10.1002/art.37800.
45. Rinderknecht C.H., Lu N., Crespo O., Truong P., Hou T., Wang N., Mellins E.D. I-Ag7 is subject to post-translational chaperoning by CLIP. Int. Immunol. 2010, 22(8):705-716. 10.1093/intimm/dxq056.
46. Rovere P., Zimmermann V.S., Forquet F., Demandolx D., Trucy J., Ricciardi-Castagnoli P., Davoust J. Dendritic cell maturation and antigen presentation in the absence of invariant chain. Proc. Natl. Acad. Sci. U. S. A. 1998, 95(3):1067-1072.
47. Seita J., Sahoo D., Rossi D.J., Bhattacharya D., Serwold T., Inlay M.A., Weissman I.L. Gene expression commons: an open platform for absolute gene expression profiling. PLoS One 2012, 7(7):e40321. 10.1371/journal.pone.0040321.
48. Shizuru J.A., Negrin R.S., Weissman I.L. Hematopoietic stem and progenitor cells: clinical and preclinical regeneration of the hematolymphoid system. Annu. Rev. Med. 2005, 56:509-538. 10.1146/annurev.med.54.101601.152334.
49. Sirven A., Ravet E., Charneau P., Zennou V., Coulombel L., Guetard D., Dubart-Kupperschmitt A. 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. 2001, 3(4):438-448. 10.1006/mthe.2001.0282.
50. Sunderkotter C., Nikolic T., Dillon M.J., Van Rooijen N., Stehling M., Drevets D.A., Leenen P.J. Subpopulations of mouse blood monocytes differ in maturation stage and inflammatory response. J. Immunol. 2004, 172(7):4410-4417.
51. Swindle C.S., Kim H.G., Klug C.A. Mutation of CpGs in the murine stem cell virus retroviral vector long terminal repeat represses silencing in embryonic stem cells. J. Biol. Chem. 2004, 279(1):34-41. 10.1074/jbc.M309128200.
52. Trobridge G.D., Beard B.C., Gooch C., Wohlfahrt M., Olsen P., Fletcher J., Kiem H.P. Efficient transduction of pigtailed macaque hematopoietic repopulating cells with HIV-based lentiviral vectors. Blood 2008, 111(12):5537-5543. 10.1182/blood-2007-09-115022.
53. van Til N.P., Stok M., Aerts Kaya F.S., de Waard M.C., Farahbakhshian E., Visser T.P., Wagemaker G. Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype. Blood 2010, 115(26):5329-5337. 10.1182/blood-2009-11-252874.
54. Verhoeyen E., Wiznerowicz M., Olivier D., Izac B., Trono D., Dubart-Kupperschmitt A., Cosset F.L. Novel lentiviral vectors displaying "early-acting cytokines" selectively promote survival and transduction of NOD/SCID repopulating human hematopoietic stem cells. Blood 2005, 106(10):3386-3395. 10.1182/blood-2004-12-4736.
55. Visigalli I., Delai S., Politi L.S., Di Domenico C., Cerri F., Mrak E., Biffi A. Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood 2010, 116(24):5130-5139. 10.1182/blood-2010-04-278234.
56. Wang N., Rajasekaran N., Hou T., Lisowski L., Mellins E.D. Comparison of transduction efficiency among various lentiviruses containing GFP reporter in bone marrow hematopoietic stem cell transplantation. Exp. Hematol 2013, 10.1016/j.exphem.2013.07.002.
57. Wanisch K., Yanez-Munoz R.J. Integration-deficient lentiviral vectors: a slow coming of age. Mol. Ther. 2009, 17(8):1316-1332. 10.1038/mt.2009.122.
58. Wentworth J.M., Naselli G., Brown W.A., Doyle L., Phipson B., Smyth G.K., Harrison L.C. Pro-inflammatory CD11c+CD206+ adipose tissue macrophages are associated with insulin resistance in human obesity. Diabetes 2010, 59(7):1648-1656. 10.2337/db09-0287.
59. Woods N.B., Ooka A., Karlsson S. Development of gene therapy for hematopoietic stem cells using lentiviral vectors. Leukemia 2002, 16(4):563-569. 10.1038/sj.leu.2402447.
60. Xue M., Dervish S., Harrison L.C., Fulcher G., Jackson C.J. Activated protein C inhibits pancreatic islet inflammation, stimulates T regulatory cells, and prevents diabetes in non-obese diabetic (NOD) mice. J. Biol. Chem. 2012, 287(20):16356-16364. 10.1074/jbc.M111.325951.
61. Yu X., Zhan X., D'Costa J., Tanavde V.M., Ye Z., Peng T., Cheng L. Lentiviral vectors with two independent internal promoters transfer high-level expression of multiple transgenes to human hematopoietic stem-progenitor cells. Mol. Ther. 2003, 7(6):827-838.
62. Zhang J.L., Cai J., Jackson J.D., Kuszynski C.A., Walls S., McIvor R.S., Fox I.J. Long-term transgene expression and survival of transgene-expressing grafts following lentivirus transduction of bone marrow side population cells. Transplantation 2005, 79(8):882-888.
63. Zhang F., Thornhill S.I., Howe S.J., Ulaganathan M., Schambach A., Sinclair J., Thrasher A.J. Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells. Blood 2007, 110(5):1448-1457. 10.1182/blood-2006-12-060814.
64. Ziegler-Heitbrock L., Ancuta P., Crowe S., Dalod M., Grau V., Hart D.N., Lutz M.B. Nomenclature of monocytes and dendritic cells in blood. Blood 2010, 116(16):e74-e80. 10.1182/blood-2010-02-258558.