Retroviral vector-mediated gene transfer into umbilical cord blood CD34(br)CD38-CD33- cells

Experimental Hematology

Volumn 27, Issue 8, 1999, Pages 1244-1254

  Gentry, Tracy ^a   Smith, Clay ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

Cord blood; Differentiation; Hematopoietic stem cells; Retroviral vector

Indexed keywords

CD33 ANTIGEN; CD34 ANTIGEN; CD38 ANTIGEN; VIRUS VECTOR;

ANTIGEN EXPRESSION; ARTICLE; CELL PROLIFERATION; CELL RENEWAL; CONTROLLED STUDY; FLUORESCENCE ACTIVATED CELL SORTER; GENE TRANSFER; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; PRIORITY JOURNAL; RETROVIRUS; UMBILICAL CORD BLOOD;

ADP-RIBOSYL CYCLASE; ANTIGENS, CD; ANTIGENS, CD34; ANTIGENS, CD38; ANTIGENS, DIFFERENTIATION; ANTIGENS, DIFFERENTIATION, MYELOMONOCYTIC; CELL CULTURE TECHNIQUES; CELL SEPARATION; CELLS, CULTURED; FETAL BLOOD; FLOW CYTOMETRY; GENE THERAPY; GENES, REPORTER; GENETIC VECTORS; HEMATOPOIETIC CELL GROWTH FACTORS; HEMATOPOIETIC STEM CELLS; HUMANS; MEMBRANE GLYCOPROTEINS; NAD+ NUCLEOSIDASE; RECEPTORS, NERVE GROWTH FACTOR; RECOMBINANT FUSION PROTEINS; RETROVIRIDAE; TRANSFECTION;

EID: 0032783275     PISSN: 0301472X     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0301-472X(99)00065-X     Document Type: Article

References (42)

1. Karlsson S. Treatment of genetic defects in hematopoietic cell function by gene transfer. Blood. 78:1991;2481.
2. Brenner M. Gene transfer to hematopoietic cells. N Engl J Med. 335:1996;337.
3. Miller A.D. Retroviral vectors. Curr Top Microbiol Immunol. 158:1992;1.
4. Allay J., Persons D., Galipeau J., Riberdy J., Ashmun R., Blakely R., Sorrentino B. In vivo selection of retrovirally transduced hematopoietic cells. Nature Med. 4:1998;1136.
5. Dunbar C.E., Emmons R.V. Gene transfer into hematopoietic progenitor and stem cells. progress and problems Stem Cells. 12:1994;563.
6. Nolta J.A., Crooks G.M., Overell R.W., Williams D.E., Kohn D.B. Retroviral vector-mediated gene transfer into primitive human hematopoietic progenitor cells. effects of mast cell growth factor (MGF) combined with other cytokines Exp Hematol. 20:1992;1065.
7. +++ hematopoietic stem/progenitor cells from human umbilical cord blood. J Exp Med. 178:1993;2089.
8. + enriched human peripheral blood stem cells. Exp Hematol. 21:1993;585.
9. Nolta J.A., Smogorzewska E.M., Kohn D.B. Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells. Blood. 86:1995;101.
10. + cells from Gaucher patients. Blood. 86:1995;141.
11. Bordignon C., Notarangelo L.D., Nobili N., Ferrari G., Casorati G., Panina P., Mazzolari E., Maggioni D., Rossi C., Servida P., Ugazio A.G., Mauilio F. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science. 270:1995;470.
12. Dunbar C.E., Cottler-Fox M., O'Shaughnessy J.A., Doren S., Carter C., Berenson R., Brown S., Moen R.C., Greenblatt J., Stewart F.M., Leitman S., Wilson W., Cowan R., Young N.S., Nientuis A.W. Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood. 85:1995;3048.
13. Kohn D.B., Weinberg K.I., Nolta J.A., Heiss L.N., Lenarsky C., Crooks G.M., Hanley M.E., Annett G., Brooks J.S., el-Khoureiy A., Lawrence R., Wells S., Moen R.C., Bastian J., Williams-Herman D., Elder M., Wara D., Bowen T., Hershfield M.S., Mullen C.A., Blaese R.M., Parkman R. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nature Med. 1:1995;1017.
14. + hematopoietic progenitor cells measured in vitro and in vivo. Gene Ther. 2:1995;245.
15. Larochelle A., Vormoor H., Hanenberg J., Wang M., Bhatia T., Lapidot T., Moritz B., Murdoch L., Xiao I. Identification of primitive hematopoietic cells capable of repopulating NOD/SCID mice. implications for gene therapy Nature Med. 2:1996;1329.
16. Bhatia M., Bonnet D., Kapp U., Wang J.C., Murdoch B., Dick J.E. Quantitative analysis reveals expansion of human hematopoietic repopulating cells after short ex-vivo culture. J Exp Med. 186:1997;619.
17. Orlic D., Girard L.J., Jordan C.T., Anderson S.M., Cline A.P., Bodine D.M. The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction. Proc Natl Acad Sci U S A. 93:1996;11097.
18. Riddell S.R., Elliott M., Lewinsohn D.A., Gilbert M.J., Wilson L., Manley S.A., Lupton S.D., Overell R.W., Reynolds T.C., Corey L., Greenberg P.D. T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. Nature Med. 2:1996;216.
19. Peters S.O., Kittler E.L.W., Ramshaw H.S., Quesenberry P.J. Ex vivo expansion of murine marrow cells with interleukin-3 (IL-3), IL-6, IL-11, and stem cell factor leads to impaired engraftment in irradiated hosts. Blood. 87:1996;30.
20. Williams D.A., Wells H.B., Whitcomb J. Ex vivo expansion of hematopoietic stem and progenitor cells-robbing peter to pay paul. Blood. 81:1993;3169.
21. McCowage G., Phillips K., Gentry T., Hull S., Kurtzberg J., Gilboa E., Smith C. Multiparameter FACS analysis of retroviral vector gene transfer into primitive umbilical cord blood cells. Exp Hematol. 26:1998;1.
22. Storms, R (1998) Stem cell based gene therapies in the treatment of AIDS. In: C. Smith (ed) Gene therapy for HIV Infection. RG Landes Co. p 117.
23. Moritz T., Parmesh D., Xiao X., Carstanjen D., Vik T., Hanenberg H., Williams D.A. Fibronectin improves transduction of reconstituting hematopoietic stem cells by retroviral vectors. evidence of direct viral binding to chymotryptic carboxy-terminal fragments Blood. 88:1996;855.
24. + hematopoietic cells. Hum Gene Ther. 8:1997;2193.
25. - progenitor cells in long term cultures and semisolid assays. Blood. 81:1993;2916.
26. - cell production in vitro. Br J Hematol. 98:1997;528.
27. Andrews R., Singer J., Berstein I. Precursors of colony forming cells can be distinguished from colony forming cells by the expression of CD33 and CD34 antigens and light scatter properties. J Exp Med. 169:1989;1721.
28. + T-lymphocytes. Gene Ther. 3:1996;695.
29. Phillips K., Gentry T., McCowage G., Gilboa E., Smith C. Cell-surface markers for assessing gene transfer into human hematopoietic cells. Nature Med. 2:1996;1154.
30. Hao Q., Thiemann F.T., Peterson D., Smorgorzewska E.M., Crooks G.M. Extended long-term culture reveals a highly quiescent and primitive human hematopoietic progenitor population. Blood. 87:1996;3306.
31. Conneally E., Eaves C.J., Humphries R.K. Efficient retroviral-mediated gene transfer to human cord blood stem cells with in vivo repopulating potential. Blood. 91:1998;3487.
32. Miller D.G., Adam M.A., Miller A.D. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol. 10:1990;4239.
33. Roe T.R., Reynolds T.C., Yu G., Brown P.O. Integration of murine leukemia virus DNA depends on mitosis. EMBO J. 12:1993;2099.
34. + cells. Blood. 89:1997;3903.
35. Holyoake T.L., Freshney M.G., McNair L., Parker A.N., McKay P.J., Steward W.P., Fitzsimons E., Graham G.J., Pragnell I.B. Ex vivo expansion with stem cell factor and interleukin-11 augments both short-term recovery posttransplant and the ability to serially transplant marrow. Blood. 87:1996;4589.
36. - cells and maintain progenitor cells in vitro. Blood. 87:1996;3563.
37. Elwood N.J., Zogos H., Willson T., Begley C.G. Retroviral transduction of human progenitor cells. use of granulocyte colony-stimulating factor plus stem cell factor to mobilize progenitor cells in-vivo and stimulation by Flt3/Flk-2 ligand in vitro Blood. 88:1996;4452.
38. Moore K.A., Ema H., Lemischka I.R. In-vitro maintenance of highly purified, transplantable hematopoietic stem cells. Blood. 89:1997;4337.
39. Naldini L., Blomer U., Gallay P., Ory D., Mulligan R., Gage F.H., Verma I.M., Trono D. In-vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 272:1996;263.
40. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science. 283:1999;682.
41. van Hennik P.B., Verstegen M.M.A., Bierhuizen M.F.A., Limón A., Wognum A.W., Cancelas J.A., Barquinero J., Ploemacher R.E., Wagemaker G. Highly efficient transduction of the green fluorescent protein gene in human umbilical cord blood stem cells capable of cobblestone formation in long-term cultures and multilineage engraftment of imminodeficient mice. Blood. 92:1998;4013.
42. low primitive cells capable of reconstituting long-term cultures in-vitro and nonobese diabetic-severe combined immunodeficiency mice in-vivo. Hum Gene Ther. 9:1998;1497.