Prolonged multilineage clonal hematopoiesis in a rhesus recipient of CD34 positive cells marked with a RD114 pseudotyped oncoretroviral vector

Blood Cells, Molecules, and Diseases

Volumn 30, Issue 1, 2003, Pages 132-143

  Kelly, Patrick F ^a   Donahue, Robert E ^b   Vandergriff, Jody A ^a   Takatoku, Masaaki ^b   Bonifacino, Aylin C ^b   Agricola, Brian A ^b   Metzger, Mark E ^b   Dunbar, Cynthia E ^b   Nienhuis, Arthur W ^a   Vanin, Elio F ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

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

Author keywords

Gene transfer; Hematopoiesis; Inverse PCR; Oncoretroviral; RD114; Rhesus

Indexed keywords

CD34 ANTIGEN; RETROVIRUS VECTOR;

ARTICLE; CELL CLONE; CONTROLLED STUDY; ENDOGENOUS VIRUS; GENE INSERTION; GENE TARGETING; GENE TRANSFER; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC TISSUE; NONHUMAN; PRIORITY JOURNAL; RECIPIENT; RHESUS MONKEY;

ANIMALIA; FELIDAE; FELINE ENDOGENOUS VIRUS; INSERTION SEQUENCES; MACACA; MACACA MULATTA; PRIMATES;

EID: 12244279028     PISSN: 10799796     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1079-9796(03)00005-6     Document Type: Article

References (46)

1. Sorrentino B.P., Nienhuis A.W. Gene therapy for hematopoietic diseases. Stamatoyannopoulos G., Majerus P.W., Perlmutter R.M., Varmus H. The molecular basis of blood diseases. 3rd ed :2001;969-1003 Saunders, Philadelphia.
2. Guenechea G., Gan O.I., Dorrell C., Dick J.E. Distinct classes of human stem cells that differ in proliferative and self-renewal potential. Nat. Immunol. 2:2001;75-82.
3. Roe T., Reynolds T.C., Yu G., Brown P.O. Integration of murine leukemia virus DNA depends on mitosis. EMBO J. 12:1993;2099-2108.
4. Dick J.E., Magli M.C., Huszar D., Phillips R.A., Bernstein A. Introduction of a selectable gene into primitive stem cells capable of long-term reconstitution of the hematopoietic system of W/Wv mice. Cell. 42:1985;71-79.
5. Lemischka I.R., Raulet D.H., Mulligan R.C. Developmental potential and dynamic behavior of hematopoietic stem cells. Cell. 45:1986;917-927.
6. Persons D.A., et al. Use of the green fluorescent protein as a marker to identify and track genetically modified hematopoietic cells. Nat. Med. 4:1998;1201-1205.
7. Bodine D.M., Moritz T., Donahue R.E., Luskey B.D., Kessler S.W., Martin D.I., Orkin S.H., Nienhuis A.W., Williams D.A. Long-term in vivo expression of a murine adenosine deaminase gene in rhesus monkey hematopoietic cells of multiple lineages after retroviral mediated gene transfer into CD34+ bone marrow cells. Blood. 82:1993;1975-1980.
8. Dunbar C.E., Seidel N.E., Sellers D.S., Cline A.P., Metzger M.E., Agricola B.A., Donahue R.E., Bodine D.M. 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. 93:1996;11871-11876.
9. Donahue R.E., Wersto R.P., Allay J.A., Agricola B.A., Metzger M.E., Nienhuis A.W., Persons D.A., Sorrentino B.P. High levels of lymphoid expression of enhanced green fluorescent protein in nonhuman primates transplanted with cytokine-mobilized blood CD34(+) cells. Blood. 95:2000;445-452.
10. Kim H.J., Tisdale J.F., Wu T., Takatoku M., Sellers S.E., Zickler P., Metzger M.E., Agricola B.A., Malley J.D., Kato I., Donahue R.E., Brown K.E., Dunbar C.E. Many multipotential gene-marked progenitor or stem cell clones contribute to hematopoiesis in nonhuman primates. Blood. 96:2000;1-8.
11. Wu T., Kim H.J., Sellers S.E., Meade K.E., Agricola B.A., Metzger M.E., Kato I., Donahue R.E., Dunbar C.E., Tisdale J.F. Prolonged high-level detection of retrovirally marked hematopoietic cells in nonhuman primates after transduction of CD34+ progenitors using clinically feasible methods. Mol. Ther. 1:2000;285-293.
12. Heim D.A., Hanazono Y., Giri N., Wu T., Childs R., Sellers S.E., Muul L., Agricola B.A., Metzger M.E., Donahue R.E., Tisdale J.F., Dunbar C.E. Introduction of a xenogeneic gene via hematopoietic stem cells leads to specific tolerance in a rhesus monkey model. Mol. Ther. 1:2000;533-544.
13. Takatoku M., Sellers S., Agricola B.A., Metzger M.E., Kato I., Donahue R.E. Dunbar, C.E. Avoidance of stimulation improves engraftment of cultured and retrovirally transduced hematopoietic cells in primates. J. Clin. Invest. 108:2001;447-455.
14. Kiem H.-P., Heyward S., Winkler A., Potter J., Allen J.M., Miller A.D., Andrews R.G. 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. 90:1997;4638-4645.
15. Kiem H.-P., Andrews R.G., Morris J., Peterson L., Allen J.M., Miller A.D., Andrews R.G. 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;1878-1886.
16. Kurre P., Morris J., Horn P.A., Harkey M.A., Andrews R.G., Kiem H.-P. Gene transfer into baboon repopulating cells a comparison of Flt-3 ligand and megakaryocyte growth and development factor versus IL-3 during ex vivo transduction . Mol. Ther. 3:2001;920-927.
17. Kiem H.-P., Darovsky B., von Kalle C., Goehle S., Stewart D., Graham T., Hackman R., Appelbaum F.R., Deeg H.J., Miller A.D., et al. Retrovirus mediated gene transduction into canine peripheral blood repopulating cells. Blood. 83:1994;1467-1473.
18. Kiem H.-P., McSweeney P.A., Bruno B., Goerner M., Buron G., Morris J., Storb R., Miller A.D. Improved gene transfer into canine hematopoietic repopulating cells using CD34-enriched marrow cells in combination with gibbon ape leukemia virus-pseudotyped retroviral vector. Gene Ther. 6:1999;966-972.
19. Goerner M., Bruno B., McSweeney P.A., Buron G., Storb R., Kiem H.-P. 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. 94:1999;2287-2292.
20. Goerner M., Horn P.A., Peterson L., Kurre P., Storb R., Rasko J.E., Kiem H.-P. Sustained multilineage gene persistence and expression in dogs transplanted with CD34(+) marrow cells transduced by RD114-pseudotyped oncoretrovirus vectors. Blood. 98:2001;2065-2070.
21. Cavazzana-Calvo M., Hacein-Bey S., de Saint Basile G., Gross F., Yvon E., Nusbaum P., Selz F., Hue C., Certain S., Casanova J.L., Bousso P., Deist F.L., Fischer A. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 288:2000;669-672.
22. Abonour R., Williams D.A., Einhorn L., Hall K.M., Chan J., Coffman J., Traycoff C.M., Bank A., Kato I., Ward M., Williams S.D., Hromas R., Robertson M.J., Smith F.O., Woo D., Mills B., Srour E.F., Cornetta J. Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic cells. Nat. Med. 6:2000;652-658.
23. May C., Rivella S., Callegari J., Heller G., Gaensler K.M., Luzzatto L., Sadelain M. Therapeutic haemoglobin synthesis in beta- thalassemic mice expressing lentivirus-encoded human beta-globin. Nature. 406:2000;82-86.
24. Pawliuk R., Westerman K.A., Fabry M.E., Payen E., Tighe R., Bouhassira E.E., Acharya S.A., Ellis J., London I.M., Eaves C.J., Humphries R.K., Beuzard Y., Nagel R.L., Leboulch P. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science. 294:2001;2368-2372.
25. Qin G., Takenaka T., Telsch K., Kelley L., Howard T., Levade T., Deans R., Howard B.H., Malech H.L., Brady R.O., Medin J.A. Preselective gene therapy for Fabry disease. Proc. Natl. Acad. Sci. USA. 98:2001;3428-3433.
26. Mardiney M. 3rd, Jackson S.H., Spratt S.K., Li F., Holland S.M., Malech H.L. Enhanced host defense after gene transfer in the murine p47phox-deficient model of chronic granulomatous disease. Blood. 89:1997;2268-2275.
27. Bunting K.D., Sangster M.Y., Ihle J.N., Sorrentino B.P. Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer. Nat. Med. 4:1998;58-64.
28. Otsu M., Anderson S.M., Bodine D.M., Puck J.M., O'Shea J.J., Candotti F. Lymphoid development and function in X-linked severe combined immunodeficiency mice after stem cell gene therapy. Mol. Ther. 1:2000;145-153.
29. Bjorgvinsdottir H., Ding C., Pech N., Gifford M.A., Li L.L., Dinauer M.C. Retroviral-mediated gene transfer of gp91phox into bone marrow cells rescues defect in host defense against Aspergillus fumigatus in murine X-linked chronic granulomatous disease. Blood. 89:1997;41-48.
30. Lemig T., Mann L., del Pilar Martin M., Bonten E., Persons D., Knowles J., Allay J.A., Cunningham J., Nienhuis A.W., Smeyne R., d'Azzo A. Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells. Blood. 99:2002;3169-3178.
31. Hanenberg H., Xiao X.L., Dilloo D., Hashino K., Kato I., Williams D.A. Optimization of fibronectin-assisted retroviral gene transfer into human CD34+ hematopoietic cells. Hum. Gene Ther. 8:1997;2193-2206.
32. Kelly P.F., Vandergriff J., Nathwani A., Nienhuis A.W., Vanin E.F. Highly efficient gene transfer into cord blood NOD/SCID repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. Blood. 96:2000;1206-1214.
33. Schilz A.J., Brouns G., Knoss H., Ottmann O.G., Hoelzer D., Fauser A.A., Thrasher A.J., Grez M. High efficiency gene transfer to human hematopoietic SCID- repopulating cells under serum-free conditions. Blood. 92:1998;3163-3171.
34. Hennemann B., Conneally E., Pawliuk R., Leboulch P., Rose-John S., Reid D., Chuo J.Y., Humphries R.K., Eaves C.J. Optimization of retroviral-mediated gene transfer to human NOD/SCID mouse repopulating cord blood cells through a systematic analysis of protocol variables. Exp. Hematol. 27:1999;817-825.
35. 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;3487-3493.
36. Pollock K.E., Van Der Loo J.C.M., Cooper R.J., Hartwell J.R., Miles K.R., Breese R., Williams E.P., Montel A., Seshadri R., Hanenberg H., Williams D.A. Differential transduction efficiency of SCID-repopulating cells derived from umbilical cood blood and granulocyte colony-stimulating factor-mobilized peripheral blood. Hum. Gene Ther. 12:2001;2095-2108.
37. Cosset F.L., Takeuchi Y., Battini J.L., Weiss R.A., Collins M.K. High-titer packaging cells producing recombinant retroviruses resistant to human serum. J. Virol. 69:1995;7430-7436.
38. Nolta J.A., Doa M.A., Wells S., Smogorzewska E.M., Kohn D.B. Transduction of pluripotent human hematopoietic stem cells as demonstrated by clonal analysis after engraftment in immune deficient mice. Proc. Natl. Acad. Sci. USA. 93:1996;2414-2419.
39. Donahue R.E., Dunbar C.E. Update on the use of nonhuman primate models for preclinical testing of gene therapy approaches targeting hematopoietic cells. Hum. Gene Ther. 12:2001;607-617.
40. Halene S., Wang L., Cooper R.M., Bockstoce D.C., Robbins P.B., Kohn D.B. Improved expression in hematopoietic and lymphoid cells in mice after transplantation of bone marrow transduced with a modified retroviral vector. Blood. 94:1999;3349-3357.
41. Abkowitz J.L., Catlin S.N., Guttorp P. Strategies from hematopoietic stem cell gene therapy insight from computer simulation studies . Blood. 89:1997;3192-3198.
42. Abkowitz J.L., Persik M.T., Shelton G.H., Ott R.L., Kiklevich J.V., Catlin S.N., Guttorp P. Behavior of hematopoietic stem cells in the large animal. Proc. Natl. Acad. Sci. USA. 92:1995;2031-2035.
43. Goerner M., Horn P.A., Peterson L., Kurre P., Storb R., Rasko J.E., Keim H.P. Sustained multilineage gene persistence and expression in dogs transplanted with CD34(+) marrow cells transduced by RD114-pseudotyped oncoretrovirus vector. Blood. 98:2001;2065-2070.
44. Kelly P.F., Carrington J., Nathwani A., Vanin E.F. RD114-pseudotyped oncoretroviral vectors. Biological and physical properties. Ann. N.Y. Acad. Sci. 938:2001;262-276.
45. Gatlin J., Melkus M.W., Padgett A., Kelly P.F., Garcia J.V. Engraftment of NOD/SCID mice with human CD34+ cells transduced by concentrated oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. J. Virol. 75:2001;9995-9999.
46. + cells from patients with human X-linked chronic granulomatous disease. Blood. 98:2001;209a.