Neonatal gene therapytransfer and expression of exogenous genes in neonatal sheep following direct injection of retroviral vectors into the bone marrow space

Experimental Hematology

Volumn 28, Issue 6, 2000, Pages 642-650

  Porada, Christopher D ^a   Tran, Nam D ^a   Zhao, Yi ^b   Anderson, W French ^b   Zanjani, Esmail D ^a  

^a VA Sierra Nevada Health Care System   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

Gene therapy; Intraosseous injection; Neonatal sheep; Retroviral vectors

Indexed keywords

VIRUS VECTOR; BETA GALACTOSIDASE; HYBRID PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BONE MARROW; CONTROLLED STUDY; FEMALE; GENE THERAPY; GENE TRANSFER; HEMATOPOIETIC CELL; NEWBORN; NONHUMAN; PRIORITY JOURNAL; RETROVIRUS; SHEEP; TRANSGENE; ANIMAL; BIOSYNTHESIS; CELL SURVIVAL; FLOW CYTOMETRY; FLUORESCENCE MICROSCOPY; GENE EXPRESSION; GENE VECTOR; GENETIC TRANSFECTION; GENETICS; HEMATOPOIETIC STEM CELL; INJECTION; ISOLATION AND PURIFICATION; LACTOSE OPERON; METHODOLOGY; POLYMERASE CHAIN REACTION; REPORTER GENE; SOUTHERN BLOTTING; TIME; VIROLOGY;

ANIMALIA; OVIS ARIES;

ANIMALS; ANIMALS, NEWBORN; BETA-GALACTOSIDASE; BLOTTING, SOUTHERN; BONE MARROW; CELL SURVIVAL; FLOW CYTOMETRY; GENE EXPRESSION; GENE THERAPY; GENES, REPORTER; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; INJECTIONS; LAC OPERON; MICROSCOPY, FLUORESCENCE; POLYMERASE CHAIN REACTION; RECOMBINANT FUSION PROTEINS; RETROVIRIDAE; SHEEP; TIME FACTORS; TRANSFECTION;

EID: 0033624048     PISSN: 0301472X     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0301-472X(00)00158-2     Document Type: Article

References (51)

1. Anderson W.F. Human gene therapy. Science. 256:1992;808.
2. Karlsson S. Treatment of genetic defects in hematopoietic cell function by gene transfer. Blood. 78:1991;2481.
3. Miller A.D. Human gene therapy comes of age. Nature. 357:1992;455.
4. Correll P.H., Calilla S., Dave H.P.G., Karlsson S. High levels of human glucocerebrosidase activity in macrophages of long-term reconstituted mice after retroviral infection of hematopoietic stem cells. Blood. 80:1992;331.
5. Eglitis M.A., Kantoff P., Gilboa E., Anderson W.F. Gene expression in mice after high efficiency retroviral-mediated gene transfer. Science. 230:1985;1395.
6. Ohashi T., Boggs S., Robbins P.et al. Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector. Proc Natl Acad Sci U S A. 89:1992;11332.
7. Plavec I., Papayannopoulou T., Maury C., Meyer F. A human β-globin gene fused to the human β-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells. Blood. 81:1993;1384.
8. Schiffmann R., Medin J.A., Ward J.M., Stahl S., Cottler-Fox M., Karlsson S. Transfer of the human glucocerebrosidase gene into hematopoietic stem cells of nonablated recipients. successful engraftment and long-term expression of the transgene Blood. 86:1995;1218.
9. Bank A. Human somatic cell gene therapy. Bioessays. 18:1996;999.
10. Kohn D.B. Gene therapy for haematopoietic and lymphoid disorders. Clin Exp Immunol. 107:1997;54.
11. Bienzle D., Abrams-Ogg A.C., Kruth S.A.et al. Gene transfer into hematopoietic stem cells. long-term maintenance of in vitro activated progenitors without marrow ablation Proc Natl Acad Sci U S A. 91:1994;350.
12. + bone marrow cells. Blood. 82:1993;1975.
13. Ekhterae D., Crumbleholme T., Karson E., Harrison M.R., Anderson W.F., Zanjani E.D. Retroviral vector-mediated transfer of the bacterial neomycin resistance gene into fetal and adult sheep and human hematopoietic progenitors in vitro. Blood. 75:1990;365.
14. Van Beusechem V.W., Bakx T.A., Kaptein L.C.et al. Retrovirus-meditated gene transfer into rhesus monkey hematopoietic stem cells. the effect of viral titers on transduction efficiency Hum Gene Ther. 4:1993;239.
15. von Kalle C., Kiem H.P., Goehle S.et al. Increased gene transfer into human hematopoietic progenitor cells by extended exposure to a pseudotyped retroviral vector. Blood. 84:1994;2890.
16. + cells. Blood. 84:1994;1408.
17. Xu L., Stahl S.K., Dave H.P.G.et al. Correction of the enzyme deficiency in hematopoietic cells of Gaucher patients using a clinically acceptable retroviral supernatant protocol. Exp Hematol. 22:1994;223.
18. - human hematopoietic cells by HIV-1-based lentiviral vectors. Proc Natl Acad Sci U S A. 96:1999;2988.
19. Takiyama N., Mohney T., Swaney W.et al. Comparison of methods for retroviral mediated transfer of glucocerebrosidase gene to CD34+ hematopoietic progenitor cells. Eur J Haematol. 61:1998;1.
20. Breems D.A., Van Driel E.M., Hawley R.G., Siebel K.E., Ploemacher R.E. Stroma-conditioned medium and sufficient prestimulation improve fibronectin fragment-mediated retroviral gene transfer into human primitive mobilized peripheral blood stem cells through effects on their recovery and transduction efficiency. Leukemia. 12:1998;951.
21. Dao M.A., Hashino K., Kato I., Nolta J.A. Adhesion to fibronectin maintains regenerative capacity during ex vivo culture and transduction of human hematopoietic stem and progenitor cells. Blood. 92:1998;4612.
22. Dutt P., Hanenberg H., Vik T., Williams D.A., Yoder M.C. A recombinant human fibronectin fragment facilitates retroviral mediated gene transfer into human hematopoietic progenitor cells. Biochem Mol Biol Int. 42:1997;909.
23. Hanenberg H., Hashino K., Konishi H., Hock R.A., Kato I., Williams D.A. Optimization of fibronectin-assisted retroviral gene transfer into human CD34+ hematopoietic cells. Hum Gene Ther. 8:1997;2193.
24. Hanenberg H., Xiao X.L., Dilloo D., Hashino K., Kato I., Williams D.A. Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells. Nat Med. 2:1996;876.
25. Kiem H.P., Andrews R.G., Morris J.et al. Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor. Blood. 92:1998;878.
26. Moritz T., Dutt P., Xiao X.et al. 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.
27. Bertolini F., Battaglia M., Corsini C.et al. Engineered stromal layers and continuous flow culture enhance multidrug resistance gene transfer in hematopoietic progenitors. Cancer Res. 56:1996;2566.
28. Asch J., Weinberg R.S., Mueller L.et al. Retroviral gene transfer into cord blood stem/progenitor cells using purified vector stocks. Am J Hematol. 57:1998;16.
29. Clapp D.W., Dumenco L.L., Hatzoglou M., Gerson S.L. Fetal liver hematopoietic stem cells as a target for in utero retroviral gene transfer. Blood. 78:1991;1132.
30. Clapp D.W., Freie B., Lee W.H., Zhang Y.Y. Molecular evidence that in situ-transduced fetal liver hematopoietic stem/progenitor cells give rise to medullary hematopoiesis in adult rats. Blood. 86:1995;2113. a.
31. Clapp D.W., Freie B., Srour E., Yoder M.C., Fortney K., Gerson S.L. Myeoloproliferative sarcoma virus directed expression of β-galactosidase following retroviral transduction of murine hematopoietic cells. Exp Hematol. 23:1995;630. b.
32. 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.
33. Haneline L.S., Marshall K.P., Clapp D.W. The highest concentration of primitive hematopoietic progenitor cells in cord blood is found in extremely premature infants. Pediatr Res. 39:1996;820.
34. Kantoff P.W., Flake A.W., Eglitis M.A.et al. In utero gene transfer and expression. a sheep transplantation model Blood. 73:1989;1066.
35. Movassagh M., Desmyter C., Baillou C.et al. High-level gene transfer to cord blood progenitors using gibbon ape leukemia virus pseudotype retroviral vectors and an improved clinically applicable protocol. Hum Gene Ther. 9:1998;225.
36. Ohashi T., Iizuka S., Sly W.S., Machiki K., Eto Y. Efficient and persistent expression of beta-glucuronidase gene in CD34+ cells from human umbilical cord blood by retroviral vector. Eur J Haematol. 61:1998;235.
37. Porada C.D., Tran N., Eglitis M.et al. In utero gene therapy. transfer and long-term expression of the bacterial neo(r) gene in sheep after direct injection of retroviral vectors into preimmune fetuses Hum Gene Ther. 9:1998;1571.
38. Verma S., Woffendin C., Bahner I.et al. Gene transfer into human umbilical cord blood-derived CD34+ cells by particle-mediated gene transfer. Gene Ther. 5:1998;692.
39. Kohn D.B., Weinberg K.I., Nolta J.A.et al. Engraftment of gene-modified cells from umbilical cord blood in neonates with adenosine deaminase deficiency. Nat Med. 1:1995;1017.
40. Cornetta K., Morgan R.A., Anderson W.F. Safety issues related to retroviral-mediated gene transfer in humans. Hum Gene Ther. 2:1991;5.
41. Goodman S., Xiao X., Donahue R.E.et al. Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells. Blood. 84:1994;1492.
42. Sambrook J., Fritsch E.F., Maniatis T. Molecular cloning. A laboratory manual. 2nd ed:1989;Cold Spring Harbor Laboratory, Cold Spring Harbor, NY.
43. Ye X., Rivera V.M., Zoltick P.et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science. 283:1999;8.
44. Rother R.P., Squinto S.P., Mason J.M., Rollins S.A. Protection of retroviral particles in human blood through complement inhibition. Hum Gene Ther. 6:1995;429.
45. Shimizu K., Miyao Y., Tamura M.et al. Infectious retrovirus is inactivated by serum but not cerebrospinal fluid or fluid from the tumor bed in patients with malignant glioma. Jpn J Cancer Res. 86:1995;1010.
46. Takeuchi Y., Cosset F.L., Lachmann P.J., Okada H., Weiss R.A., Collins M.K. Type C retrovirus inactivation by human complement is determined by both the viral genome and the producer cell. J Virol. 68:1994;8001.
47. Nelson D.M., Metzger M.E., Donahue R.E., Morgan R.A. In vivo retrovirus-mediated gene transfer into multiple hematopoietic lineages in rabbits without preconditioning. Hum Gene Ther. 8:1997;747.
48. Foley R., Ellis R., Walker I.et al. Intramarrow cytokine gene transfer by adenoviral vectors in dogs. Hum Gene Ther. 8:1997;545.
49. Lutzko C., Kruth S., Abrams-Ogg A.C.et al. Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency. Blood. 93:1999;1895.
50. Zepeda M., Wilson J.M. Neonatal cotton rats do not exhibit destructive immune responses to adenoviral vectors. Gene Ther. 3:1996;973.
51. Lemischka I.R., Raulet D.H., Mulligan R.C. Developmental potential and dynamic behavior of hematopoietic stem cells. Cell. 45:1986;917.