Comparison of retroviral transduction efficiency in CD34+ cells derived from bone marrow versus G-CSF-mobilized or G-CSF plus stem cell factor-mobilized peripheral blood in nonhuman primates

Stem Cells

Volumn 22, Issue 6, 2004, Pages 1062-1069

  Hematti, Peiman ^a   Tuchman, Sascha ^b   Larochelle, Andre ^a   Metzger, Mark E ^a   Donahue, Robert E ^a   Tisdale, John F ^b  

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

^b NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES   (United States)

Author keywords

Bone marrow; Gene tranfer; Retrovirus; Stem cell

Indexed keywords

CD34 ANTIGEN; FIBRONECTIN; GRANULOCYTE COLONY STIMULATING FACTOR; STEM CELL FACTOR;

ANIMAL CELL; ARTICLE; BONE MARROW CELL; CELL COMPARTMENTALIZATION; COMPARATIVE STUDY; DERIVATIZATION; GENE TRANSFER; GENETIC MANIPULATION; GENETIC TRANSDUCTION; IMMOBILIZED CELL; LEUKAPHERESIS; NONHUMAN; RETROVIRUS; RHESUS MONKEY; SICKLE CELL ANEMIA; STEM CELL GENE THERAPY; SUPERNATANT; TARGET CELL; TRANSACTIVATION;

ANIMALIA; MACACA MULATTA; PRIMATES; UNIDENTIFIED RETROVIRUS;

EID: 8644291701     PISSN: 10665099     EISSN: None     Source Type: Journal    
DOI: 10.1634/stemcells.22-6-1062     Document Type: Article

References (25)

1. Donahue RE, Kessler SW, Bodine D et al. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J Exp Med 1992;176:1125-1135.
2. Lozier JN, Csako G, Mondoro TH et al. Toxicity of a first-generation adenoviral vector in rhesus macaques. Hum Gene Ther 2002;13:113-124.
3. Lozier JN, Metzger ME, Donahue RE et al. Adenovirus-mediated expression of human coagulation factor IX in the rhesus macaque is associated with dose-limiting toxicity. Blood 1999;94:3968-3975.
4. Hu J, Dunbar CE. Update on hematopoietic stem cell gene transfer using non-human primate models. Curr Opin Mol Ther 2002;4:482-490.
5. Kiem H-P, Sellers S, Thomasson B et al. Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia. Mol Ther 2004;9: 389-395.
6. Tisdale JF, Hanazono Y, Sellers SE et al. Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability. Blood 1998;92:1131-1141.
7. + progenitors using clinically feasible methods. Mol Ther 2000;1:285-293.
8. Kiem HP, Andrews RG, 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 1998;92:1878-1886.
9. Goerner M, Bruno B, McSweeney PA et al. The use of granulocyte colony-stimulating factor during retroviral transduction on fibronectin fragment CH-296 enhances gene transfer into hematopoietic repopulating cells in dogs. Blood 1999;94:2287-2292.
10. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease [see comments]. Science 2000; 288:669-672.
11. Hacein-Bey-Abina S, von Kalle C, Schmidt M et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003;348:255-256.
12. May C, Rivella S, Callegari J et al. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature 2000;406: 82-86.
13. Pawliuk R, Westerman KA, Fabry ME et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 2001;294:2368-2371.
14. Persons DA, Allay ER, Sawai N et al. Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells. Blood 2003;102:506-513.
15. Levasseur DN, Ryan TM, Pawlik KM et al. Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells. Blood 2003; 102:4312-4319.
16. + cells is superior to G-CSF or G-CSF+FU3-L-mobilized cells in nonhuman primates. Blood 2003;101: 2199-2205.
17. Thomasson B, Peterson L, Thompson J et al. Direct comparison of steady-state marrow, primed marrow, and mobilized peripheral blood for transduction of hematopoietic stem cells in dogs. Hum Gene Ther 2003;14:1683-1686.
18. Adler BK, Salzman DE, Carabasi MH et al. Fatal sickle cell crisis after granulocyte colony-stimulating factor administration. Blood 2001;97:3313-3314.
19. Abboud M, Laver J, Blau CA. Granulocytosis causing sickle-cell crisis [letter]. Lancet 1998;351:959.
20. Grigg AP. Granulocyte colony-stimulating factor-induced sickle cell crisis and multiorgan dysfunction in a patient with compound heterozygous sickle cell/beta+ thalassemia. Blood 2001;97:3998-3999.
21. + cells in Thy-1 expression and cell cycle status in nonhuman primates mobilized or not mobilized with granulocyte colony-stimulating factor and/or stem cell factor. Blood 1996;87: 1644-1653.
22. Dunbar CE, Cottler-Fox M, O'Shaughnessy JA et al. Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 1995;85:3048-3057.
23. Pauling L, Itano HA, Singer SJ et al. Sickle cell anemia, a molecular disease. Science 1949;110:543.
24. Tisdale JF, Sadelain M. Toward gene therapy for disorders of globin synthesis. Semin Hematol 2001;38:382-392.
25. Rivella S, May C, Chadburn A et al. A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer. Blood 2003;101:2932-2939.