A SIN lentiviral vector containing PIGA cDNA allows long-term phenotypic correction of CD34+ -Derived cells from patients with paroxysmal nocturnal hemoglobinuria

Molecular Therapy

Volumn 7, Issue 3, 2003, Pages 304-316

  Robert, David ^a   Mahon, François Xavier ^b,c   Richard, Emmanuel ^a   Etienne, Gabriel ^d   de Verneuil, Hubert ^a   Moreau Gaudry, François ^a  

^a INSERM   (France)

^b CNRS Bordeaux Segalen University   (France)

^c HAUT LÉVÊQUE HOSPITAL   (France)

^d Serv Maladies Infect Hosp St Andie   (France)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; CD59 ANTIGEN; COMPLEMENTARY DNA; ELONGATION FACTOR 1ALPHA; GLYCOSYLPHOSPHATIDYLINOSITOL; HLA ANTIGEN; LENTIVIRUS VECTOR; MEMBRANE PROTEIN; PHOSPHATIDYLINOSITOL; VIRUS DNA;

ALLOGENIC BONE MARROW TRANSPLANTATION; ARTICLE; BONE MARROW CELL; CELL DIFFERENTIATION; CELL SURFACE; CHRONIC DISEASE; CONTROLLED STUDY; CULTURE MEDIUM; DISEASE ASSOCIATION; DISEASE EXACERBATION; DISEASE SEVERITY; ERYTHROID CELL; GENE CASSETTE; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HEMOLYTIC ANEMIA; HLA MATCHING; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS 1; MEGAKARYOCYTE; MORBIDITY; MORTALITY; NEUTROPENIA; NONHUMAN; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PHENOTYPE; PROMOTER REGION; SOMATIC MUTATION; STEM CELL MOBILIZATION; THROMBOCYTOPENIA; THROMBOSIS; X CHROMOSOME LINKAGE;

HUMAN IMMUNODEFICIENCY VIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRUS;

EID: 0242586016     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1525-0016(03)00011-X     Document Type: Article

References (58)

1. Takeda, J, et al. (1993). Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria. Cell 73: 703-711.
2. Bessler, M, et al. (1994). Paroxysmal nocturnal haemoglobinuria (PNH) is caused by somatic mutations in the PIG-A gene. EMBO J. 13: 110-117.
3. Miyata, T, et al. (1993). Cloning of PIG-A, a component in the early step of GPI-anchor biosynthesis. Science 259: 1318-1320.
4. Tiede, A., Nischan, C., Schubert, J., and Schmidt, R. E. (2000). Characterisation of the enzymatic complex for the first step in glycosylphosphatidylinositol biosynthesis. Int. J. Biochem. Cell Biol. 32: 339-350.
5. Watanabe, R., et al. (1998). The first step of glycosylphosphatidylinositol biosynthesis is mediated by a complex of PIG-A, PIG-H, PIG-C and GPI1. EMBO J. 17: 877-885.
6. Rosse, W. F., and Ware, R. E. (1995). The molecular basis of paroxysmal nocturnal hemoglobinuria. Blood 86: 3277-3286.
7. Kinoshita, T., Inoue, N., and Takeda, J. (1995). Defective glycosyl phosphatidylinositol anchor synthesis and paroxysmal nocturnal hemoglobinuria. Adv. Immunol. 60: 57-103.
8. Nishimura, J., Smith, C. A., Phillips, K. L., Ware, R. E., and Rosse, W. F. (1998). Paroxysmal nocturnal hemoglobinuria: molecular pathogenesis and molecular therapeutic approaches. Hematopathol. Mol. Hematol. 11: 119-146.
9. Hillmen, P., Lewis, S. M., Bessler, M., Luzzatto, L., and Dacie, J. V. (1995). Natural history of paroxysmal nocturnal hemoglobinuria. N. Engl. J. Med. 333: 1253-1258.
10. Bemba, M., et al. (1999). Bone marrow transplantation for paroxysmal nocturnal haemoglobinuria. Br. J. Haematol. 105: 366-368.
11. Saso, R., et al. (1999). Bone marrow transplants for paroxysmal nocturnal haemoglobinuria. Br. J. Haematol. 104: 392-396.
12. Nishimura, J., et al. (2001). Efficient retrovirus-mediated PIG-A gene transfer and stable restoration of GPI-anchored protein expression in cells with the PNH phenotype. Blood 97: 3004-3010.
13. Endo, M., Beatty, P. G., Vreek, T. M., Wittwer, C. T., Singh, S. P., and Parker, C. J. (1996). Syngeneic bone marrow transplantation without conditioning in a patient with paroxysmal nocturnal hemoglobinuria: in vivo evidence that the mutant stem cells have a survival advantage. Blood 88: 742-750.
14. Vigna, E., and Naldini, L. (2000). Lentiviral vectors: excellent tools for experimental gene transfer and promising candidates for gene therapy. J. Gene Med. 2: 308-316.
15. Uchida, N., et al. (1998). HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 95: 11939-11944.
16. Case, S. S., et al. (1999). Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors. Proc. Natl. Acad. Sci. USA 96: 2988-2993.
17. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283: 682-686.
18. Saulnier, S. O., et al, (2000). Lentivirus-mediated gene transfer of gp91 phox corrects chronic granulomatous disease (CGD) phenotype in human X-CGD cells. J. Gene Med. 2: 317-325.
19. Moreau-Gaudry, F., et al. (2001). High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors. Blood 98: 2664 -2672.
20. May, C., et al. (2002). Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene. Blood 99: 1902-1908.
21. Richard, E., et al. (2001). Gene therapy of a mouse model of protoporphyria with a self-inactivating erythroid-specific lentiviral vector without preselection. Mol. Ther. 4: 331-338.
22. Zennou, V., et al. (2000). HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 101: 173-185.
23. Sirven, A., et al. (2000). The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells. Blood 96: 4103-4110.
24. Zufferey, R., et al. (1999). Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73: 2886-2892.
25. Loeb, J. E., et al. (1999). Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy. Hum. Gene Ther. 10: 2295-2305.
26. Ramezani, A., et al. (2000). Lentiviral vectors for enhanced gene expression in human hematopoietic cells. Mol. Ther. 5: 458-469, doi:10.1006/mthe.2000.0190.
27. + cells in ADA-deficient SCID neonates. Nat. Med. 4: 775-780.
28. Hacein-Bey-Abina, S., et al. (2002). Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N. Engl. J. Med. 346: 1185-1193.
29. Qin, G., et al. (2001). Preselective gene therapy for Fabry disease. Proc. Natl. Acad. Sci. USA 98: 3428-3433.
30. Cartier, N. (2001). Gene therapy strategies for X-linked adrenoleukodystrophy. Curr. Opin. Mol. Ther. 3: 357-361.
31. + cells from peripheral blood after mobilization in a patient with congenital erythropoietic porphyria. Mol. Ther. 3: 411-417, doi:10.10006/mthe.2001.0270.
32. Bessler, M., et al. (2002). Glycosylphosphatidylinositol-linked proteins are required for maintenance of a normal peripheral lymphoid compartment but not for lymphocyte development. Eur. J. Immunol. 32: 2607-2616.
33. Karadimitris, A., et al. (2000). Abnormal T-cell repertoire is consistent with immune process underlying the pathogenesis of paroxysmal nocturnal hemoglobinuria. Blood 96: 2613-2620.
34. Karadimitris, A., et al. (2001). Related association of clonal T-cell large granular lymphocyte disease and paroxysmal nocturnal haemoglobinuria (PNH): further evidence for a pathogenetic link between T cells, aplastic anaemia and PNH. Br. J. Haematol. 115: 1010-1014.
35. Kinoshita, T., and Inoue, N. (2002). Relationship between aplastic anemia and paroxysmal nocturnal hemoglobinuria. Int. J. Hematol. 75: 117-122.
36. Suenaga, K., et al. (2001). Successful application of nonmyeloablative transplantation for paroxysmal nocturnal hemoglobinuria. Exp. Hematol. 29: 639-642.
37. Aiuti, A., et al. (2002). Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296: 2410-2413.
38. Sorrentino, B. P., et al. (1992). Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1. Science 288: 669-672.
39. Podda, S., et al. (1992). Transfer and expression of the human drug resistance gene into live mice. Proc. Natl. Acad. Sci. USA 89: 9676-9680.
40. Corey, C. A., et al. (1990). Serial transplantation of methotrexate-resistant bone marrow: protection of murine recipients from drug toxicity by progeny of transduced stem cells. Blood 75: 337-343.
41. Allay, J. A., et al. (1998). In vivo selection of retrovirally transduced hematopoietic stem cells. Nat. Med. 10: 1136-1143.
42. 6-benzylguanine and 1,3-bis(2-chloroethyl)-1-nitrosourea. Cancer Res. 57: 5093-5099.
43. Lee, K., et al. (2001). G156A MGMT-transduced human mesenchymal stem cells can be selectively enriched by O6-benzylguanine and BCNU. J. Hematother. Stem Cell Res. 5: 691-701.
44. 6-benzylguanine, and an alkyltransferase-expressing retroviral vector. Mol. Ther. 1: 78-87, doi:10.1006/mthe.2000.0223.
45. 6-methylguanine DNA ethyltransferase-transduced marrow progenitors repopulate nonmyeloablated mice after drug selection. Blood 95: 3078-3084.
46. Imren, S., et al. (2002). Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 99: 14380-14385.
47. Woods, N. B. (2002). Lentiviral transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis. Blood (in press).
48. Li, Z., et al. (2002). Murine leukaemia induced by retroviral gene marking. Science 296: 497.
49. Buckley, R. H. (2002). Gene therapy for SCID - A complication after remarkable progress. Lancet 360: 1185-1186.
50. Lemoine, N. R. (2002). Risks and benefits of gene therapy for immunodeficiency: a reality check. Gene Ther. 9: 1561-1562.
51. Murakami, Y., Kinoshita, T., Maeda, Y., Nakano, T., Kosaka, H., and Takeda, J. (1999). Different roles of glycosylphosphatidylinositol in various hematopoietic cells as revealed by a mouse model of paroxysmal nocturnal hemoglobinuria. Blood 94: 2963-2970.
52. Jasinski, M., et al. (2001). GATA1-Cre mediates PIGA gene inactivation in the erythroid/megakaryocytic lineage and leads to circulating red cells with a partial deficiency in glycosyl phosphatidylinositol-linked proteins (paroxysmal nocturnal hemoglobinuria type II cells). Blood 98: 2248-2255.
53. Keller, P., Payne, J. L., and Tremml, G. (2001). FES-Cre targets phosphatidylinositol glycan class A (PIGA) inactivation to hematopoietic stem cells in the bone marrow. J. Exp. Med. 194: 581-589.
54. Hirose, S., et al. (1992). Derivation and characterization of glycoinositol-phospholipid anchor-defective human K562 cell clones. J. Biol. Chem. 267: 5272-5278.
55. Bastisch, I., et al. (2000). Glycosylphosphatidylinositol (GPI)-deficient Jurkat T cells as a model to study functions of GPI-anchored proteins. Clin. Exp. Immunol. 122: 49-54.
56. Brodsky, R. A., et al. (1999). Resistance of paroxysmal nocturnal hemoglobinuria cells to the glycosylphosphatidylinositol-binding toxin aerolysin. Blood 93: 1749-1756.
57. Koza, V., Jindra, P., Svojgrova, M., and Cetkovsky, P. (1998). Successful autologous transplantation in a patient with severe aplastic anemia (SAA). Bone Marrow Transplant. 21: 957-959.
58. Piacibello, W., Garetto, L., and Aglietta, M. (2000). Ex vivo expansion of megakaryocytes. Transfus. Sci. 22: 107-110.