A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34+ cells from the bone marrow of human immunodeficiency virus-1-infected children

Blood

Volumn 94, Issue 1, 1999, Pages 368-371

  Kohn, Donald B ^a   Bauer, Gerhard ^b   Rice, C Robert ^b   Rothschild, J C ^b   Carbonaro, Denise A ^b   Valdez, Penelope ^b   Hao, Qian Lin ^b   Zhou, Chen ^b   Bahner, Ingrid ^b   Kearns, Karen ^b   Brody, Kate ^b   Fox, Sarah ^b   Haden, Elizabeth ^b   Wilson, Kathy ^b   Salata, Cathy ^b   Dolan, Cathy ^b   Wetter, Charles ^b   Aguilar Cordova, Estuardo ^b   Church, Joseph ^b  

^a CHILDREN S HOSPITAL LOS ANGELES   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; VIRUS VECTOR;

ADOLESCENT; ARTICLE; CASE REPORT; GENE TARGETING; GENE TRANSLOCATION; HEMATOPOIETIC STEM CELL; HOST RESISTANCE; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; IMMUNOREGULATION; INFECTION RESISTANCE; LYMPHOCYTE PROLIFERATION; PRIORITY JOURNAL; RETROVIRUS; SCHOOL CHILD; T LYMPHOCYTE ACTIVATION;

EID: 0033168686     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v94.1.368.413a47_368_371     Document Type: Article

References (23)

1. Baltimore D: Intracellular immunization. Nature 335:395, 1988
2. Gilboa E, Smith C: Gene therapy for infectious diseases: The AIDS model. Trends Genet 10:139, 1994
3. + hematopoietic progenitor cells by a retroviral vector expressing an RRE decoy inhibits HIV-1 replication in the myelomonocytic cells produced in long-term culture. J Virol 70:4352, 1996
4. + cells from HIV-1-infected donors using retroviral vectors containing anti-HIV-1 genes. Blood 89:2259, 1997
5. Woffendin C, Ranga U, Yang Z-Y, Nabel GJ: Expression of a protective gene prolongs survival of T cells in human immunodeficiency virus-infected patients. Proc Natl Acad Sci USA 93:2889, 1996
6. Morgan RA, Walker R, Carter C, Bechtel C, Bunnell B, Fellowes V, Muul L, Leitman S, Lane C, Blaese RM: Preferential survival of CD4 T-cells engineered with anti-HIV genes in patients enrolled in a gene therapy trial in HIV-1 discordant identical twins. American Society of Gene Therapy, Seattle, WA, 1998, p 44a (abstr)
7. Miller AD, Buttimore C: Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Mol Cell Biol 6:2895, 1986
8. Wells S, Malik P, Pensiero M, Kohn DB, Nolta JA: The presence of an autologous marrow stromal cell layer increases glucocerebrosidase gene transduction of long term culture initiating cells (LTCIC) from the bone marrow of a patient with Gaucher disease. Gene Ther 2:512, 1995
9. Kearns K, Bahner I, Bauer G, Wei SF, Valdez P, Wheeler S, Woods L, Miller R, Casciato D, Galpin J, Church J, Kohn DB: Suitability of bone marrow from HIV-1-infected donors for retroviral-mediated gene transfer. Human Gene Ther 8:310, 1997
10. Kohn DB, Weinberg KI, Nolta JA, Heiss LN, Lenarsky C, Crooks GM, Hanley ME, Annett G, Brooks JS, El-Khoureiy A, Lawrence K, Wells S, Shaw K, Moen RC, Bastian J, Williams-Herman DE, Elder M, Wara D, Bowen T, Hershfield MS, Mullen CA, Blaese RM, Parkman R: Engraftment of gene-modified cells from umbilical cord blood in neonates with adenosine deaminase deficiency. Nat Med 1:1017, 1995
11. Kohn DB, Hershfield MS, Carbonaro D, Shigeoka A, Brooks J, Smogorzewska EM, Barsky LW, Chan R, Burotto F, Annett G, Nolta JA, Crooks G, Kapoor N, Elder M, Wara D, Bowen T, Madsen E, Snyder FF, Bastian J, Muul L, Blaese RM, Weinberg K, Parkman R: T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nat Med 4:775, 1998
12. - cells in cord blood and bone marrow. Blood 86:3745, 1995
13. Kiem HP, Heyward S, Winkler A, Potter J, Allen JM, Miller AD, Andrews RG: Gene transfer into marrow repopulating cells: Comparison between amphotropic and gibbon ape leukemia virus pseudotyped retroviral vectors in a competitive repopulation assay in baboons. Blood 90:4638, 1997
14. Dunbar CE, Seidel NE, Doren S, Sellers S, Cline AP, Metzger ME, Agricola BA, Donahue RE, Bodine DM: Improved retroviral gene transfer into murine and rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor. Proc Natl Acad Sci USA 93:11871, 1996
15. Hanenberg H, Xiao XL, Dilloo D, Hashino K, Kato I, Williams DA: Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells. Nat Med 2:876, 1996
16. - cells and maintains progenitor cells in vitro. Blood 87:3563, 1996
17. +progenitors to sustain long-term hematopoiesis in immune-deficient mice afer ex vivo retroviral-mediated transduction. Blood 89:446, 1997
18. kip-1 coupled with transforming growth factor β neutralization induces cell-cycle entry and increases retroviral transduction of primitive human hematopoietic cells. Proc Natl Acad Sci USA 95:13006, 1998
19. Kiem H-P, Andrews RG, Morris J, Peterson L, Heyward S, Allen JM, Rasko JEJ, Potter J, Miller AD: 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 92:1878, 1998
20. Tisdale JF, Hanazono Y, Sellers SE, Agricola BA, Metzger ME, Donahue RE, Dunbar CE: Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability. Blood 92:1131, 1998
21. Naldini L, Blomer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, Trono D: In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272:263, 1996
22. Poeschla E, Corbeau P, Wong-Staal F: Development of HIV vectors for anti-HIV gene therapy. Proc Natl Acad Sci USA 93:11395, 1996
23. Nolta JA, Kohn DB: Comparison of the effects of growth factors on retroviral vector-mediated gene transfer and the proliferative status of human hematopoietic progenitor cells. Hum Gene Ther 1:257, 1990