GENE TRANSFER TO HEMATOPOIETIC STEM CELLS: Implications for gene therapy of human disease

Annual Review of Medicine

Volumn 47, Issue , 1996, Pages 11-20

  Dunbar, Cynthia E ^a  

^a NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

Author keywords

Adeno associated virus; Gene expression; Retrovirus; Transplantation

Indexed keywords

GENE PRODUCT;

ADENOVIRUS; ANIMAL CELL; EXPRESSION VECTOR; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; NONHUMAN; PRIORITY JOURNAL; RETROVIRUS; REVIEW;

ADENOVIRIDAE; ANIMALS; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; HUMANS; PHENOTYPE; RETROVIRIDAE;

EID: 0029887997     PISSN: 00664219     EISSN: None     Source Type: Book Series    
DOI: 10.1146/annurev.med.47.1.11     Document Type: Review

References (43)

1. Karlsson S. 1991. Treatment of genetic defects in hematopoietic cell function by gene transfer. Blood 78:2481-92
2. Yu M, Poeschla E, Wong-Staal F. 1994. Progress towards gene therapy for HIV infection. Gene Ther. 1:13-26
3. Kessler DA, Siegel JP, Noguchi PD, et al, 1993. Regulation of somatic-cell therapy and gene therapy by the Food and Drug Administration. N. Engl. J. Med. 329: 1169-73
4. Miller AD. 1992. Human gene therapy comes of age. Nature 357:455-60
5. Miller AD. 1990. Retrovirus packaging cell lines. Hum. Gene Ther. 1:5-9
6. Miller DG, Adam MA, Miller AD. 1990. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol. Cell. Biol. 10: 4239-43
7. Donahue RE, Kessler SW, Bodine D, et al. 1992. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J. Exp. Med. 176:1125-35
8. Markowitz D, Goff S, Bank A. 1988. A safe packaging line for gene transfer: separating viral genes on two different plasmids. J. Virol. 62:1120-24
9. Cornetta K, Morgan RA, Anderson WF. 1991. Safety issues related to retroviral-mediated gene transfer to humans. Hum. Gene Ther. 2:5-14
10. Luskey BD, Rosenblatt M, Zsebo K, Williams DA. 1992. Stem cell factor promotes retroviral-mediated gene transfer into murine hematopoietic stem cells. Blood 80: 396-402
11. Fraser C, Eaves CJ, Szilvassy SJ, Humphries RK. 1990. Expansion in vitro of retrovirally marked totipotent hematopoietic stem cells. Blood 76:1071-76
12. Bodine DM, McDonagh KT, Seidel NE, Nienhuis AW. 1991. Survival and retrovirus infection of murine hematopoietic stem cells in vitro: effects of 5-FU and method of infection. Exp. Hematol. 19: 206-12
13. Wilson JM, Danos O, Grossman M, et al. 1990. Expression of human adenosine deaminase in mice reconstituted with retrovirus-transduced hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 87: 439-45
14. Correll PH, Colilla S, Dave HP, Karlsson S. 1992. High levels of human glucocerebrosidase activity in macrophages of long-term reconstituted mice after retroviral infection of hematopoietic stem cells. Blood 80:331-36
15. Van Beusechem VW, Kukler A, Heidt PJ, Valerio D. 1992. Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone-marrow cells. Proc. Natl. Acad. Sci. USA 89:7640-44
16. Bodine DM, Moritz T, Donahue RE, et al. 1993. Long term expression of a murine adenosine deaminase (ADA) gene in rhesus hematopoietic cells of multiple line-ages following retroviral mediated gene transfer into CD34+ bone marrow cells. Blood 82:1975-80
17. Schuening FG, Kawahara K, Miller AD, et al. 1991. Retrovirus-mediated gene transduction into long-term repopulating marrow cells of dogs. Blood 78:2568-76
18. Nolta JA, Kohn DB. 1990. Comparison of the effects of growth factors on retroviral vector-mediated gene transfer and the proliferative status of human hematopoietic progenitor cells. Human Gene Ther. 1:257-68
19. Hughes PFD, Thacker JD, Hogge D, et al. 1992. Retroviral gene transfer to primitive normal and leukemic hematopoietic cells using clinically-applicable procedures. J. Clin. Invest. 89:1817-24
20. Moore KA, Deisseroth AB, Reading CL, et al. 1992. Stromal support enhances cell-free retroviral vector transduction of human bone marrow long-term culture initiating cells. Blood 79:1393-99
21. Cassel A, Cottler-Fox M, Doren S, Dunbar CE. 1993. Retroviral-mediated gene transfer into CD34-enriched human peripheral blood stem cells. Exp. Hematol 21:585-91
22. Lu L, Xiao M, Clapp DW, et al. 1993. High efficiency retroviral mediated gene transduction into single isolated immature and replatable CD34+ hematopoietic stem/progenitor cells from human umbilical cord blood. J. Exp. Med. 178:2089-96
23. von Kalle C, Kiem HP, Goehle S, et al. 1995. Increased gene transfer into human hematopoietic progenitor cells by extended in vitro exposure to a pseudotyped retroviral vector. Blood 84:2890-97
24. Brenner MK, Rill DR, Moen RC, et al. 1993. Gene-marking to trace origin of relapse after autologous bone marrow transplantation. Lancet 341:85-86
25. Brenner MK, Rill DR, Holladay MS, et al. 1993. Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients. Lancet 342:1134-37
26. Dunbar CE, Cottler-Fox M, O'Shaughnessy JA, et al. 1995. Retrovirally-marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85:3048-57
27. Blaese RM, Anderson WF, Culver KW, Rosenberg SA. 1990. The ADA human gene therapy clinical protocol. Hum. Gene Ther. 1:327-62
28. Blaese RM. 1993. Development of gene therapy for immunodeficiency: adenosine deaminase deficiency. Pediatr. Res. 33: S49-53
29. Walsh CE, Grompe M, Vanin E, et al. 1994. A functionally active retrovirus vector for gene therapy in Fanconi anemia group C. Blood 84:453-59
30. Stewart FM, Crittenden RB, Lowry PA, et al. 1993. Long-term engraftment of normal and post-5-fluorouracil murine marrow into normal nonmyeloablated mice. Blood 81:2544-71
31. Barquinero J, Kiem HP, von Kalle C, et al. 1995. Myelosuppressive conditioning improves autologous engraftment of genetically marked hematopoietic repopulating cells in dogs. Blood 85:1195-1201
32. Muzyczka N. 1992. Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr. Top. Microbiol. Immunol. 158:97-105
33. Samulski RJ, Zhu X, Xiao X, et al. 1991. Targeted integration of adeno-associated virus (AAV) into chromosome 19. EMBO J. 10:3941-50
34. Russell DW, Miller AD, Alexander IE. 1994. Adeno-associated virus vectors preferentially transduce cells in S phase. Proc. Natl. Acad. Sci. USA 91:8915-19
35. Podsakoff G, Wong JKK, Chatterjee S. 1994. Efficient gene transfer into nondividing cells by adeno-associated virus-based vectors. J. Virol. 68:5656-66
36. Samulski RJ, Chang LS, Shenk T. 1989. Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression. J. Virol. 63:3822-27
37. Miller JL, Donahue RE, Sellers SE, et al. 1994. Recombinant adeno-associated virus (rAAV) mediated expression of a human γ-globin gene in human progenitor derived erythroid cells. Proc. Natl. Acad. Sci. USA 92:10183-87
38. Goodman S, Xiao X, Donahue RE, et al. 1994. Recombinant adeno-associated virus mediated gene transfer into hematopoietic progenitor cells. Blood 84:1492-1500
39. Sorrentino BP, Brandt SJ, Bodine D, et al. 1992. Selection of drug resistant bone marrow cells in vivo after retroviral transfer of human MDR1. Science 257:99-103
40. Hanania EG, Deisseroth AB. 1994. Serial transplantation shows that early hematopoietic precursor cells are transduced by MDR-1 retroviral vector in a mouse gene therapy model. Cancer Gene Ther. 1:21-25
41. Aran JM, Gottesman MM, Pastan I. 1994. Drug-selected coexpression of human glucocerebrosidase and p-glycoprotein using a bicistronic vector. Proc. Natl. Acad. Sci. USA 91:3176-80
42. Russell SJ, Hawkins RE, Winter G. 1993. Retroviral vectors displaying functional antibody fragments. Nucleic Acids Res. 21: 1081-85
43. Kasahara N, Dozy AM, Kan YW. 1994. Tissue-specific targeting of retroviral vectors through ligand-receptor interactions. Science 266:1373-76