Retroviral mediated transfer of the cDNA for human glucocerebrosidase into hematopoietic stem cells of patients with gaucher disease. A phase I study

Human Gene Therapy

Volumn 7, Issue 2, 1996, Pages 231-253

  Dunbar, Cynthia ^a   Kohn, Donald ^b   Karlsson, Stefan ^c   Barton, Norman ^c   Brady, Roscoe ^c   Cottler Fox, Michele ^d   Crooks, Gay ^b   Emmons, Robert ^a   Esplin, Joan ^e   Leitman, Susan ^d   Lenarsky, Carl ^b   Nolta, Jan ^b   Parkman, Robertson ^b   Pensiero, Michael ^f   Schifmann, Raphael ^c   Tolstoshev, Paul ^f   Weinberg, Kenneth ^b  

^a NHLBI   (United States)

^b CHILDREN S HOSPITAL LOS ANGELES   (United States)

^c NINDS   (United States)

^d NATIONAL INSTITUTES OF HEALTH   (United States)

^e UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^f NOVARTIS PHARMA AG   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; COMPLEMENTARY DNA; GLUCOSYLCERAMIDASE; GRANULOCYTE COLONY STIMULATING FACTOR; INTERLEUKIN 3; INTERLEUKIN 6; STEM CELL FACTOR;

ARTICLE; BONE MARROW; CLINICAL PROTOCOL; GAUCHER DISEASE; GENE EXPRESSION; GENE TRANSFER; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HUMAN; MACROPHAGE; PERIPHERAL BLOOD STEM CELL; PHASE 1 CLINICAL TRIAL; RETROVIRUS; STROMA;

UNIDENTIFIED RETROVIRUS;

EID: 19144372650     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.2-231     Document Type: Article

References (51)

1. Brady RO, Barranger JA, Glucosylceramide Lipidosis: Gaucher disease. In: Stanbury JB, Wyngaarden JB, Frederickson DS, Goldstein JL, Brown MS, eds. The Metabolic Basis of Inherited Disease. 5th ed. New York: McGraw-Hill, 1983;842-856.
2. Beutler E. Gaucher disease. N. Engl. J. Med. 325:1354-1360 (1991).
3. Brady RO, Kanfer JN, Shapiro D. Metabolism of glucocerebrosides. II. Evidence of an enzymatic deficiency in Gaucher disease. Biochem. Biophys. Res. Commun. 18:221-225 (1965).
4. Brady RO, Kanfer JN, Bradley RM, Shapiro D. Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher disease. J Clin Invest. 45:1112-1115 (1966).
5. Hobbs JR, Jones KH, Shaw PJ, Lindsay I, Hancock M. Beneficial effect of pre-transient splenectomy on displacement bone marrow transplantation for Gaucher syndrome. Lancet. I:1111-1115 (1987).
6. Ringden O, Groth CG, Erikson A et al. Long term follow-up of the first successful bone marrow transplantation in Gaucher disease. Transplantation. 46:66-70 (1988).
7. Erikson A, Groth CG, Mausson J-E, Percy A, Ringden O, Svennerholm L. Clinical and Biochemical Outcome of Marrow Transplantation for Gaucher Disease of the Norrbottnian type. Acta Paediatr. Scand. 79:680-685 (1990).
8. Rappeport JM, Barranger JA, Ginns EI. Bone marrow transplantation in Gaucher disease. Birth Defects. 22:101-109 (1986).
9. Tsai P, Lipton JM, Sahdev I, Najfeld V, Rankin LR, Slyper AH, Ludman M, Grabowski GA. Allogeneic bone marrow transplantation in severe Gaucher disease. Ped Res. 31:503-507 (1992).
10. Barton NW, Furbish FS, Murray GJ, Garfield M, Brady RO. Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease. Proc. Natl. Acad. Sci. USA. 87:1913-1916 (1990).
11. Barton NW, Brady RO, Dambrosia JM, Di Bisceglie AM et. al. Replacement therapy for inherited enzyme deficiency - macrophage-targeted glucocerebrosidase for Gaucher disease. N. Eng. J. Med. 324:1464-1470 (1991).
12. Beutler E, Kay A, Saven A, Carver P, Thurston D, Dawson A, Rosenbloom B. Enzyme replacement therapy for Gaucher disease. Blood. 78:1183-1189 (1991).
13. Fink JK, Correll PM, Perry LK, Brady RO, Karlsson S. Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease. Proc. Natl. Acad. Sci. USA. 87:2334-2338 (1990).
14. Nolta JA, Yu XJ, Bahner I, Kohn DB. Retroviral mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow. J. Clin. Invest. 90:343-348 (1992).
15. Williams DA. Expression of introduced genetic sequences in hematopoietic cells following retroviral-mediated gene transfer. Hum. Gene Ther. 1:229 (1990).
16. Karlsson S. Treatment of genetic defects in hematopoietic cell functions by gene transfer. Blood. 78:2481-2492 (1991).
17. Correll PM, Fink JK, Brady RO, Perry LK, Karlsson S. Production of human glucocerebrosidase in mice after retroviral gene transfer into multipotential hematopoietic progenitor cells. Proc. Natl. Acad. Sci. USA. 86:8912-8916 (1989).
18. Correll PM, Kew Y, Perry LK, Brady RO, Fink JK, Karlsson S. Expression of human glucocerebrosidase in long-term reconstituted mice following retroviral-mediated gene transfer into hematopoietic stem cell. Hum. Gene Ther. 1:227-287 (1990).
19. Weinthal J, Nolta JA, Yu XJ, Lilley J, Uribe L, Kohn DB. Expression of human glucocerebrosidase following retroviral-mediated transduction of murine hematopoietic stem cells. Bone Marrow Transplant. 8:403-412 (1991).
20. Correll PM, Colilla S, Dave HPG, Karlsson S. High levels of human glucocerebrosidase activity in macrophages of long-term reconstituted mice after retroviral infection of hematopoietic stem cells. Blood. 80:331-336 (1992).
21. Moore KA, Deisseroth AB, Reading CL, Williams DE, Belmont JW. Stromal support enhances cell-free retroviral vector transduction of human bone marrow long-term culture-initiating cells. Blood. 79:1393-1399 (1992).
22. Hughes PFD, Thacker JD, Hogge D, Sutherland MJ, Thomas TE, Lansdorf PM, Eaves CJ, Humphries RK. Retroviral gene transfer to primitive normal and leukemic hematopoietic cells using clinically applicable procedures. J. Clin. Invest. 89:1817-1824 (1992).
23. Nolta JA and Kohn DB. Comparison of the effects of growth factors on retroviral vector-mediated gene transfer and the proliferative status of human hematopoietic progenitor cells. Human Gene Therapy. 1:257-268 (1990).
24. Nolta JA, Crooks GM, Overell RW, Williams DE, Kohn DB. Retroviral vector-mediated gene transfer into primitive human hematopoietic progenitor cells: effects of Mast Cell Growth Factor (MGF) combined with other cytokines. Exp. Hematol. 20:1065-1071 (1992).
25. + hematopoietic progenitors using a clinically acceptable protcol. Blood. 80 (Suppl 1): 179a (1992).
26. Bodine DM, McDonagh KT, Braudd SJ, Ney PA, Agricola B, Byrne E, Nienhuis AW. Development of a high-titer retrovirus producer cell line capable of gene transfer into rhesus monkey hematopoietic stem cells. Proc. Natl. Acad. Sci. USA. 87:3738-3742 (1990).
27. Van Beusechem VW, Kakler A, Meidt PJ, Valerio D. Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone marrow cells. Proc. Natl. Acad. Sci. USA. 89:7640-7644 (1992).
28. Donahue RE, Kessler SW, Bodine D, McDonagh K, Dunbar C, Goodman S, Agricola B, Byrne E, Raffeld M, Moen R, Bacher J, Zsebo KM, Nienhuis AW. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J. Exp. Med. 176:1125-1135 (1992).
29. Carter RF, Abrams-Ogg ACG, Dick JE, Kruth SA, Valli VE, Kamel-Reid S, Dubé ID. Autologous transplantation of canine long-term marrow culture cells genetically marked by retroviral vectors. Blood. 79:356-364 (1992).
30. Lothrop CD Jr. Al-Lebban ZS, Niemeyer GP, Jones JB, Peterson MG, Smith JR, Baker MJ, Morgan RA, Eglitis MA, Anderson WF. Expression of a foreign gene in cats reconstituted with retroviral vector infected autologous bone marrow. Blood. 78:237 (1991).
31. Brenner MK, Rill DR, Moen RC, Krance RA, Mirro Jr. J, Anderson WF, Ihle JN. Gene-marking to trace origin of relapse after autologous bone-marrow transplantation. Lancet 341:85-86 (1993).
32. Crittenden RB, Stewart FM, Williams ME, Quesenberry PJ. "Competitive" marrow transplantation: a murine model. Blood. (in press) (1993).
33. Wu D-D, Keating A. Hematopoietic stem cells engraft in untreated transplant recipients. Experimental Hematology. 21:251-256 (1993).
34. Brecher G, Ansel JD, Micklem HS, Tjio JH, Cronkite EP. Special proliferative sites are not needed for seeding and proliferation of transfused bone marrow cells in normal syngeneic mice. PNAS. 79:5085-5087 (1982).
35. Stewart FM, Crittenden RB, Williams ME, Pearson-White S, Quesenberry PJ. Long-term engraftment of normal and post-5-fluorouracil murine marrow into normal nonmyeloablated mice. Blood. 81:2566-2571 (1993).
36. Harrison DE, Astle CM, Lerner C. Number and continuous proliferative pattern of transplanted primitive immunohematopoietic stem cells. PNAS (USA). 85:822 (1990).
37. Jordan CT, Lemnishka IR. Clonal and systemic analysis of long term hematopoiesis in the mouse. Genes and Development. 4:220 (1990).
38. Rosenthal DI, Scott JA, Barranger J, Mankin HJ, Saini S, Brady TJ, Osier LK, Doppelt S. Evaluation of Gaucher disease using magnetic resonance imaging. The Journal of Bone and Joint Surgery. 68A:802-808 (1986).
39. Van Beusechem VW, Bakx TA, Kaptein LC, Bart-Baumeister JA, et al. Retrovinis-mediated gene therapy transfer into rhesus monkey hematopoietic stem cells: the effect of viral titers on transduction efficiency. Human Gene Therapy. 4:239-247.
40. Anderson WF, McGarrity GJ, Moen RC. Report to the NIH recombinant DNA advisory committee on murine replication-competent retrovirus (RCR) assays. Human Gene Therapy 4:311-321 (1993).
41. Nolta JA, Smorgorzewska EM, Kohn DB. Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells. Blood 86:101-110 (1995).
42. Bodine DM, McDonagh KT, Seidel NE, Nienhuis AW. Survival and retrovirus infection of murine hematopoietic stem cells in vitro: effects of 5-FU and method of infection. Experimental Hematology. 19:206 (1991).
43. Bodine DM, Moritz T, Donahue RE, Luskey BD, Kessler SW, Martin DIK, Orkin SH, Nienhuis AW, Williams DA. Long term expression of a murine adenosine deaminase (ADA) gene in rhesus hematopoietic cells of multiple lineages following retroviral mediated gene transfer into CD34+ bone marrow cells. Blood. 82:1975 (1993).
44. Hughes PFD, Eaves CJ, Hogge DE, Humphries RK. High efficiency gene transfer to human hematopoietic cells maintained in long-term marrow culture. Blood. 74:1915 (1989).
45. Hughes PFD, Thacker JD, Hogge D, Sutherland HJ, Thomas TE, Lansdorp PM, Eaves CJ, Humphries RK. Retroviral gene transfer to primitive normal and leukemic hematopoietic cells using clinically applicable procedures. J. Clin. Invest. 89:1817-1824 (1992).
46. Flanagan JG, Chan DC, Leder P. Transmembrane form of the kit ligand growth factor is determined by alternative splicing and is missing in the Sld mutant. Cell 64:1025 (1991).
47. Sutherland HJ, Eaves CJ, Eaves AC, Dragowska W, Lansdorp PM. Characterization and partial purification of human marrow cells capable of initiating long-term hematopoiesis in vitro. Blood. 74:1563 (1989).
48. Turham AJ, Humphries RK, Eaves CJ, Barnett MJ, Phillips GL, Kalousek DK, Klingman HG, Landsdorp PL, Reece DE, Shephard JD, Eaves CA. Detection of breakpoint cluster region-negative and non-clonal hematorpoiesis in vitro and in vivo after transplantation of cells selected in cultures of chronic myeloid leukemia marrow. Blood. 76:2404 (1990).
49. + bone marrow cells. Blood. 82:1975-1980 (1993).
50. 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 (LTCICs) from bone marrow of a patient with Gaucher disease. Gene Ther. 2:1-9 (1995).
51. Brenner MK, Rill DR, Holladay MS, Heslop HE, et al. Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients. Lancet. 342:1134-1137 (1993).