Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC)

Human Gene Therapy

Volumn 10, Issue 14, 1999, Pages 2337-2346

  Liu, Johnson M ^a   Kim, Sonnie ^a   Read, Elizabeth J ^b   Futaki, Makoto ^a   Dokal, Inderjeet ^c   Carter, Charles S ^b   Leitman, Susan F ^b   Pensiero, Michael ^d   Young, Neal S ^a   Walsh, Christopher E ^e  

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

^b NATIONAL INSTITUTES OF HEALTH   (United States)

^c HAMMERSMITH HOSPITAL   (United Kingdom)

^d NOVARTIS PHARMA AG   (Switzerland)

^e UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; MITOMYCIN C;

ADOLESCENT; ADULT; ANEMIA; ARTICLE; AUTOSOMAL RECESSIVE DISORDER; BONE MARROW CULTURE; CASE REPORT; CHILD; CYTOGENETICS; FANCONI ANEMIA; FEMALE; GENE MUTATION; GENE THERAPY; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; MALE;

ADOLESCENT; ADULT; ANTIGENS, CD34; BONE MARROW CELLS; CELL CYCLE PROTEINS; CHILD; COLONY-FORMING UNITS ASSAY; DNA-BINDING PROTEINS; FANCONI ANEMIA; FANCONI ANEMIA COMPLEMENTATION GROUP C PROTEIN; FANCONI ANEMIA COMPLEMENTATION GROUP PROTEINS; FEMALE; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; HUMANS; LEUKOCYTES, MONONUCLEAR; MALE; NUCLEAR PROTEINS; PROTEINS; RETROVIRIDAE;

EID: 0033588822     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950016988     Document Type: Article

References (31)

1. BORDIGNON, C., NOTARANGELO, L.D., NOBILI, N., FERRARI, G., CASORATI, G., PANINA, P., MAZZOLARI, E., MAGGIONI, D., ROSSI, C., SERVIDA, P., UGAZIO, A.G., and MAVILIO, F. (1995). Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 270, 470-475.
2. BRENNER, M.K. (1996). Gene transfer to hematopoietic cells. N. Engl. J. Med. 335, 337-339.
3. BRENNER, M.K., RILL, D.R., HOLLADAY, M.S., HESLOP, H.E., MOEN, R.C., BUSCHLE, M., KRANCE, R.A., SANTANA, V.M., ANDERSON, W.F., and IHLE, J.N. (1993). Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients. Lancet 342, 1134-1137.
4. CARREAU, M., and BUCHWALD, M. (1998). Fanconi's anemia: What have we learned from the genes so far? Mol. Med. Today 4, 201-206.
5. CUMMING, R.C., LIU, J.M., YOUSSOUFIAN, H., and BUCHWALD, M. (1996). Suppression of apoptosis in hematopoietic factor-dependent progenitor cell lines by expression of the FAC gene. Blood 88, 4558-4567.
6. DE WINTER, J.P., WAISFISZ, Q., ROOIMANS, M.A., VAN BERKEL, C.G.M., BOSNOYAN-COLLINS, L., ALON, N., CARREAU, M., BENDER, O., DEMUTH, I., SCHINDLER, D., PRONK, J.C., ARWERT, F., HOEHN, H., DIGWEED, M., BUCHWALD, M., and JOENJE, H. (1998). The Fanconi anaemia group G gene FANCG is identical with XRCC9. Nature Genet. 20, 281-283.
7. DUNBAR, C.E., COTTLER-FOX, M., O'SHAUGHNESSY, J.A., DOREN, S., CARTER, C., BERENSON, R., BROWN, S., MOEN, R.C., GREENBLATT, J., STEWART, F.M., LEITMAN, S.F., WILSON, W.H., COWAN, K., YOUNG, N.S., and NIENHUIS, A.W. (1995). Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85, 3048-3057.
8. FANCONI, G. (1967). Familial constitutional panmyelocytopathy, Fanconi's anemia. I. Clinical aspects. Semin. Hematol. 4, 233-240.
9. FANCONI ANAEMIA/BREAST CANCER CONSORTIUM. (1996). Positional cloning of the Fanconi anaemia group A gene. Nature Genet. 14, 324-328.
10. FU, K.-L., LO TEN FOE, J.R., JOENJE, H., RAO, K.W., LIU, J.M., and WALSH, C.E. (1997). Functional correction of Fanconi anemia group A hematopoietic cells by retroviral gene transfer. Blood 90, 3296-3303.
11. GILLIO, A.P., VERLANDER, P.C., BATISH, S.D., GIAMPIETRO, P.F., and AUERBACH, A.D. (1997). Phenotypic consequences of mutations in the Fanconi anemia FAC gene: An International Fanconi Anemia Registry study. Blood 90, 105-110.
12. GLUCKMAN, E., BROXMEYER H.E., AUERBACH, A.D., FRIEDMAN, H.S., DOUGLAS, G.W., DEVERGIE, A., ESPEROU, H., THIERRY, D., SOCIE, G., LEHN, P., COOPER, S., ENGLISH, D., KURTZBERG, J., BARD, J., and BOYSE, E.A. (1989). Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling. N. Engl. J. Med. 321, 1174-1178.
13. GLUCKMAN, E., AUERBACH, A.D., HOROWITZ, M.M., SOBOCINSKI, K.A., ASH, R.C., BORTIN, M.M., BUTTURINI, A., CAMITTA, B.M., CHAMPLIN, R.E., FRIEDRICH, W., GOOD, R.A., GORDON-SMITH, E.C., HARRIS, R.E., KLEIN, J.P., ORTEGA, J.J., PASQUINI, R., RAMSAY, N.K.C., SPECK, B., VOWELS, M.R., ZHANG, M.-J., and GALE, R.P. (1995). Bone marrow transplantation for Fanconi anemia. Blood 86, 2856-2862.
14. JOENJE, H., OOSTRA, A.B., WIJKER, M., DI SUMMA, F.M., VANBERKEL, C.G., ROOIMANS, M.A., EBELL, W., VAN WEEL, M., PRONK, J.C., BUCHWALD, M., and ARWERT, F. (1997). Evidence for at least eight Fanconi anemia genes. Am. J. Hum. Genet. 61, 940-944.
15. KOHN, D.B., WEINBERG, K.I., NOLTA, J.A., HEISS, L.N., LENARSKY, C., CROOKS, G.M., HANLEY, M.E., ANNETT, G., BROOKS, J.S., EL-KHOUREIY, A., LAWRENCE, K., WELLS, S., MOEN, R.C., BASTIAN, J., WILLIAMS-HERMAN, D.E., ELDER, M., WARA, D., BOWEN, T., HERSHFIELD, M.S., MULLEN, C.A., BLAESE, R.M., and PARKMAN, R. (1995). Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nature Med. 1, 1017-1023.
16. KRUYT, F.A., HOSHINO, T., LIU, J.M., JOSEPH, P., JAISWAL, A.K., YOUSSOUFIAN, H.A. (1998). Abnormal microsomal detoxification implicated in Fanconi anemia group C by interaction of the FAC protein with NADPH cytochrome P450 reductase. Blood 92, 3050-3056.
17. LIU, J.M. (1998). Gene transfer for the eventual treatment of Fanconi's anemia. Semin. Hematol. 35, 168-179.
18. LIU, J.M., BUCHWALD, M., WALSH, C.E., and YOUNG, N.S. (1994). Fanconi anemia and novel strategies for therapy. Blood 84, 3995-4007.
19. LIU, J.M., KIM, S., and WALSH, C.E. (1995a). Retroviral-mediated transduction of the Fanconi anemia C complementing (FACC) gene in two murine transplantation models. Blood Cells Mol. Dis. 21, 56-63.
20. LIU, J.M., WALSH, C.E., and NIENHUIS, A.W. (1995b). Gene therapy for hematologic disorders in Hematology: Basic Principles and Practice. R. Hoffman, E.J. Benz, S.J. Shattil, B. Furie, H.J. Cohen, and L.E. Silberstein, eds. (Churchill Livingstone, New York), pp. 427-435.
21. LO TEN FOE, J.R., ROOIMANS, M.A., BOSNOYAN-COLLINS, L., ALON, N., WIJKER, M., PARKER, L., LIGHTFOOT, J., CARREAU, M., CALLEN, D.F., SAVOIA, A., CHENG, N.C., VAN BERKEL, C.G., STRUNK, M.H., GILLE, J.J., PALS, G., KRUYT, F.A., PRONK, J.C., ARWERT, F., BUCHWALD, M., and JOENJE, H. (1996). Expression cloning of a cDNA for the major Fanconi anaemia gene, FAA. Nature Genet. 14, 320-323.
22. LO TEN FOE, J.E., KWEE, M.L., ROOIMANS, M.A., OOSTRA, A.B., VEERMAN, A.J., VAN WEEL, M., PAULI, R.M., SHAHIDI, N.T., DOKAL, I., ROBERTS, I., ALTAY, C., GLUCKMAN, E., GIBSON, R.A., MATHEW, C.G., ARWERT, F., and JOENJE, H. (1997). Somatic mosaicism in Fanconi anaemia: Molecular basis and clinical significance. Eur. J. Hum. Genet. 5, 137-148.
23. LU, L., SHEN, R.-N., and BROXMEYER, H.E. (1996). Stem cells from bone marrow, umbilical cord blood and peripheral blood for clinical application: current status and future application. Crit. Rev. Oncol. Hematol. 22, 61-78.
24. + cell dose predicts survival, posttransplant morbidity, and rate of hematologic recovery after allogeneic marrow transplants for hematologic malignancies. Blood 88, 3223-3229.
25. OSAWA, M., HANADA, K., HAMADA, H., and NAKAUCHI, H. (1996). Long-term lymphohematopoietic reconstitution by a single CD34-low/negative hematopoietic stem cell. Science 273, 242-245.
26. SELVAGGI, T.A., WALKER, R.E., and FLEISHER, T.A. (1997). Development of antibodies to fetal calf serum with arthus-like reactions in human immunodeficiency virus-infected patients given syngeneic lymphocyte infusions. Blood 89, 776-779.
27. STRATHDEE, C.A., GAVISH, H., SHANNON, W.R., and BUCHWALD, M. (1992). Cloning of cDNAs for Fanconi's anaemia by functional complementation. Nature (London) 356, 763-767.
28. WALSH, C.E., GROMPE, M., VANIN, E., BUCHWALD, M., YOUNG, N.S., NIENHUIS, A.W., and LIU, J.M. (1994a). A functionally active retrovirus vector for gene therapy in Fanconi anemia group C. Blood 84, 453-159.
29. WALSH, C.E., NIENHUIS, A.W., SAMULSKI, R.J., BROWN, M.E., MILLER, J.L., YOUNG, N.S., and LIU, J.M. (1994b). Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J. Clin. Invest. 94, 1449-1448.
30. WALSH, C.E., MANN, M.M., EMMONS, R.V.B., WANG, S., and LIU, J.M. (1995). Transduction of CD34-enriched human peripheral and umbilical cord blood progenitors using a retroviral vector with the Fanconi anemia group C gene. J. Invest. Med. 43, 379-385.
31. YAMASHITA, T., KUPFER, G.M., NAF, D., SULIMAN, A., JOENJE, H., ASANO, S., and D'ANDREA, A.D. (1998). The Fanconi anemia pathway requires FAA phosphorylation and FAA/FAC nuclear accumulation. Proc. Natl. Acad. Sci. U.S.A. 95, 13085-13090.