10 Current status of stem cell therapy and prospects for gene therapy for the disorders of globin synthesis
Tài liệu tham khảo
Abbound, 1996, Neurologic complications following bone marrow transplantation for sickle cell disease, Bone Marrow Transplantation, 17, 405
Abboud, 1998, Granulocytosis causing sickle-cell crisis, Lancet, 351, 959, 10.1016/S0140-6736(05)60614-9
Abkowitz, 1996, Evidence that hematopoiesis may be a stochastic process in vivo, Nature Medicine, 2, 190, 10.1038/nm0296-190
Adams, 1992, The use of transcranial ultrasonography to predict stroke in sickle cell disease, New England Journal of Medicine, 326, 605, 10.1056/NEJM199202273260905
Allay, 1997, Sensitization of hematopoietic stem and progenitor cells to trimetrexate using nucleoside transport inhibitors, Blood, 90, 3546, 10.1182/blood.V90.9.3546
Amin, 1991, Monozygotic twins with sickle cell anemia and discordant clinical courses: clinical and laboratory studies, Hemoglobin, 15, 247, 10.3109/03630269109027877
Andreani, 1996, Persistence of mixed chimerism in patients transplanted for the treatment of thalassaemia, Blood, 87, 3494, 10.1182/blood.V87.8.3494.bloodjournal8783494
Angelucci, 1992, Sudden cardiac tamponade after chemotherapy for marrow transplantation in thalassemia, Lancet, 339, 287, 10.1016/0140-6736(92)91343-7
Angelucci, 1994, Cardiac tamponade in thalassemia, Bone Marrow Transplantation, 13, 827
Angelucci, 1997, Phlebotomy to reduce iron overload in patients cured of thalassemia by bone marrow transplantation, Blood, 90, 994, 10.1182/blood.V90.3.994
Aran, 1994, Drug-selected coexpression of human glucocerebrosidase and P-glycoprotein using a bicistronic vector, 91, 3176
Bender, 1989, A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells, Molecular and Cellular Biology, 9, 1426, 10.1128/MCB.9.4.1426
Blau, 1996, The hematologic effects of folate analogs: implications for using the dihydrofolate reductase gene for in vivo selection, Human Gene Therapy, 7, 2069, 10.1089/hum.1996.7.17-2069
Blau, 1997, Cytokine prestimulation as a gene therapy strategy: implications for using the MDR1 gene as a dominant selectable marker, Blood, 89, 146, 10.1182/blood.V89.1.146
Blau, 1997, A proliferation switch for genetically modified cells, 94, 3076
LeBoulch, 1994, Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission if an active transcriptional structure, EMBO Journal, 13, 3065, 10.1002/j.1460-2075.1994.tb06605.x
Bregni, 1992, Human peripheral blood hematopoietic progenitors are optimal targets of retroviral-mediated gene transfer, Blood, 80, 1418, 10.1182/blood.V80.6.1418.1418
Brenner, 1993, Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients, Lancet, 342, 1134, 10.1016/0140-6736(93)92122-A
Brichard, 1996, Persistence of fetal hemoglobin production after successful transplantation of cord blood stem cells in a patient with sickle cell anemia, Journal of Pediatrics, 128, 241, 10.1016/S0022-3476(96)70398-0
Cohen, 1992, A modified transfusion program for prevention of stroke in sickle cell disease, Blood, 79, 1657, 10.1182/blood.V79.7.1657.1657
Contu, 1994, Successful unrelated bone marrow transplantation in betathalassemia, Bone Marrow Transplantation, 13, 329
Deeg, 1993, Graft-versus-host disease: host and donor views, 30, 110
Dunbar, 1995, Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation, Blood, 85, 3048, 10.1182/blood.V85.11.3048.bloodjournal85113048
Dzierzak, 1988, Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells, Nature, 331, 35, 10.1038/331035a0
Ferster, 1992, Bone marrow transplantation for severe sickle cell anaemia, British Journal of Haematology, 80, 102, 10.1111/j.1365-2141.1992.tb06407.x
Ferster, 1993, Bone marrow transplantation corrects the splenic reticuloendothelial dysfunction in sickle cell anemia, Blood, 81, 1102, 10.1182/blood.V81.4.1102.bloodjournal8141102
Ferster, 1995, Neurologic complications after bone marrow transplantation for sickle cell anemia, Blood, 86, 408, 10.1182/blood.V86.1.408.bloodjournal861408
Ferster, 1995, Transplanted sickle-cell disease patients with autologous bone marrow recovery after graft failure develop increased levels of fetal haemoglobin which corrects disease severity, British Journal of Haematology, 90, 804, 10.1111/j.1365-2141.1995.tb05199.x
Flake, 1997, In utero hematopoietic stem cell transplantation. A status report, Journal of the American Medical Association, 278, 932, 10.1001/jama.1997.03550110070039
Gaziev, 1997, Malignancies after bone marrow transplantation for thalassemia, Bone Marrow Transplantation, 19, 142
Giardini, 1995, Desferioxamine therapy acclerates clearance of iron deposits after bone marrow transplantation for thalassaemia, British Journal of Haematology, 89, 868, 10.1111/j.1365-2141.1995.tb08426.x
Gottesman, 1993, Biochemistry of multidrug resistance mediated by the multidrug transporter, Annual Review of Biochemistry, 62, 385, 10.1146/annurev.bi.62.070193.002125
Grosveld, 1987, Position-independent, high-level expression of the human β-globin gene in transgenic mice, Cell, 51, 975, 10.1016/0092-8674(87)90584-8
Harrison, 1988, Number and continuous proliferative pattern of transplanted primitive immunohematopoietic stem cells, 85, 822
Hirata, 1996, Transduction of hematopoietic cells by foamy virus vectors, Blood, 88, 3654, 10.1182/blood.V88.9.3654.bloodjournal8893654
Issaragrisil, 1994, Cord blood transplantation in thalassemia, Blood Cells, 20, 259
Iyengar, 1997, Striking augmentation of hematopoietic cell chimerism in noncytoablated allogeneic bone marrow recipients by FLT3 ligand and tacrolimus, Transplantation, 63, 1193, 10.1097/00007890-199705150-00001
Jin, 1998, Stimulating cell proliferation through the pharmacologic activation of c-kit, Blood, 91, 890, 10.1182/blood.V91.3.890
Jin, 1998, Targeted expansion of genetically modified bone marrow cells, 95, 8093
Johnson, 1984, Bone-marrow transplantation in a patient with sickle-cell anemia, New England Journal of Medicine, 311, 780, 10.1056/NEJM198409203111207
Johnson, 1994, American Journal of Pediatric Hematology/Oncology, 16, 22
Jordan, 1990, Clonal and systemic analysis of long-term hematopoiesis in the mouse, Genes and Development, 4, 220, 10.1101/gad.4.2.220
Kapelushnik, 1995, Analysis of β-globin mutations shows stable mixed chimerism in patients with thalassemia after bone marrow transplantation, Blood, 86, 3241, 10.1182/blood.V86.8.3241.3241
Kelly, 1997, Umbilical cord blood stem cells: application for the treatment of patients with hemoglobinopathies, Journal of Pediatrics, 130, 695, 10.1016/S0022-3476(97)80009-1
Kiem, 1997, 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, 4638, 10.1182/blood.V90.11.4638
Lemischka, 1986, Developmental potential and dynamic behaviour of hematopoietic stem cells, Cell, 45, 917, 10.1016/0092-8674(86)90566-0
Ljungman, 1997, High busulfan concentrations are associated with increased transplant-related mortality in allogeneic marrow transplant patients, Bone Marrow Transplantation, 20, 909, 10.1038/sj.bmt.1700994
Lucarelli, 1991, For debate: bone marrow transplantation for severe thalassaemia, British Journal of Haematology, 78, 300, 10.1111/j.1365-2141.1991.tb04440.x
Lucarelli, 1997, Bone marrow transplantation in thalassemia, 305
Lucarelli, 1990, Bone marrow transplantation in patients with thalassemia, New England Journal of Medicine, 322, 417, 10.1056/NEJM199002153220701
Lucarelli, 1993, Fate of iron stores in thalassaemia after bone-marrow transplantation, Lancet, 342, 1388, 10.1016/0140-6736(93)92753-G
Lucarelli, 1993, Marrow transplantation in patients with thalassemia responsive to iron chelation therapy, New England Journal of Medicine, 329, 840, 10.1056/NEJM199309163291204
Lucarelli, 1996, Marrow transplanation for patients with thalassemia: results in class 3 patients, Blood, 87, 2082, 10.1182/blood.V87.5.2082.2082
Mentzer, 1994, Availability of related donors for bone marrow transplantation in sickle cell anemia, American Journal of Pediatric Hematology/Oncology, 16, 27
Micklem, 1987, Numbers and dispersion of repopulating hematopoietic cell clones in radiation chimeras as functions of injected cell dose, Experimental Hematology, 15, 251
Migita, 1995, Selection of transduced CD34+ peogenitors and enzymatic correction of cells from Gaucher patients, with bicistronic vectors, 92, 12075
Miller, 1990, Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection, Molecular and Cellular Biology, 10, 4239, 10.1128/MCB.10.8.4239
de Montalembert, 1995, Transfusion-dependent thalassemia: viral complications (epidemiology and follow-up), 32, 280
Muretto, 1994, Bone marrow transplantation in thalassemia: modification of hepatic iron overload and related pathologies after long-term engrafting, Liver, 14, 14, 10.1111/j.1600-0676.1994.tb00002.x
Naldini, 1996, In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector, Science, 272, 263, 10.1126/science.272.5259.263
Neff, 1997, Stem cell gene therapy, position effects and chromatin insulators, Stem Cells, 15, 265
Nesci, 1992, Mixed chimerism in thalassemic patients after bone marrow transplantation, Bone Marrow Transplantation, 10, 143
Orlic, 1994, What defines a pluripotent hematopoietic stem cell (PHSC): will the real PHSC please stand up!, Blood, 84, 3991, 10.1182/blood.V84.12.3991.bloodjournal84123991
Orlic, 1996, The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction, 93, 11097
van Os, 1997, Host conditioning with 5-fluorouracil and kit-ligand to provide for longterm bone marrow engraftment, Blood, 89, 2376, 10.1182/blood.V89.7.2376
Papayannopoulou, 1993, Peripheralization of hemopoietic progenitors in primates treated with anti-VLA4 integrin, 90, 9374
Pawlowska, 1997, Relationship of plasma pharmacokinetics of high-dose oral busulfan to the outcome of allogeneic bone marrow transplantation in children with thalassemia, Bone Marrow Transplantation, 20, 915, 10.1038/sj.bmt.1701001
Peters, 1996, Ex vivo expansion of murine marrow cells with interleukin-3 (IL-3), IL-6, IL-11, and stem cell factor leads to impaired engraftment in irradiated hosts, Blood, 87, 30, 10.1182/blood.V87.1.30.30
Pettengell, 1993, Transplantation potential of hematopoietic cells released into the circulation during routine chemotherapy for non-Hodgkin's lymphoma, Blood, 82, 2239, 10.1182/blood.V82.7.2239.2239
Platt, 1991, Pain in sickle cell disease. Rates and risk factors, New England Journal of Medicine, 325, 11, 10.1056/NEJM199107043250103
Platt, 1994, Mortality in sickle cell disease. Life expectancy and risk factors for early death, New England Journal of Medicine, 330, 1639, 10.1056/NEJM199406093302303
Plavec, 1993, A human beta-globin gene fused to the human beta globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus transduced hematopoietic stem cells, Blood, 81, 1384, 10.1182/blood.V81.5.1384.1384
Raftopoulos, 1997, Long-term transfer and expression of the human β-globin gene in a mouse transplant model, Blood, 90, 3414, 10.1182/blood.V90.9.3414
Ren, 1996, Production of genetically stable high-titer retroviral vectors that carry a human gamma-globin gene under the control of the alpha-globin locus control region, Blood, 87, 2518, 10.1182/blood.V87.6.2518.bloodjournal8762518
Russell, 1998, Human gene targeting by viral vectors, Nature Genetics, 18, 325, 10.1038/ng0498-325
Russell, 1976, Transfusion therapy for cerebrovascular abnormalities in sickle cell disease, Journal of Pediatrics, 88, 382, 10.1016/S0022-3476(76)80251-X
Sadelain, 1997, Genetic treatment of the haemoglobinopathies: recombinations and new combinations, British Journal of Haematology, 98, 247, 10.1046/j.1365-2141.1997.2313048.x
Sadelain, 1995, Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene, 92, 6728
de Sanctis, 1991, Gonadal function after allogenic bone marrow transplantation for thalassaemia, Archives of Disease in Childhood, 66, 517, 10.1136/adc.66.4.517
de Sanctis, 1993, Pubertal development in thalassaemic patients after allogenic bone marrow transplantation, European Journal of Pediatrics, 152, 993, 10.1007/BF01957223
Shesely, 1991, Correction of a human beta-globin gene by gene targeting, 88, 4294
Simonsen, 1983, Isolation and expression of an altered mouse dihydrofolate reductase cDNA, 80, 2495
Sorrentino, 1992, Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1, Science, 257, 99, 10.1126/science.1352414
Spencer, 1993, Controlling signal transduction with synthetic ligands, Science, 262, 1019, 10.1126/science.7694365
Stamatoyannopoulos, 1994, Hemoglobin switching, 107
Storb, 1997, Stable mixed hematopoietic chimerism in DLA-identical littermate dogs given sublethal total body irradiation before and pharmacological immunosuppression after marrow transplantation, Blood, 89, 3048, 10.1182/blood.V89.8.3048
Styles, 1997, New therapies and approaches to transfusion in sickle cell disease in children, Current Opinion in Pediatrics, 9, 41, 10.1097/00008480-199702000-00010
Sykes, 1997, Induction of high levels of allogeneic hematopoietic reconstitution and donor-specific tolerance without myelosuppressive conditioning, Nature Medicine, 3, 783, 10.1038/nm0797-783
Thomas, 1982, Marrow transplantation for thalassemia, Lancet, 2, 227, 10.1016/S0140-6736(82)90319-1
Turner, 1996, Haemopoietic progenitor homing and mobilization, British Journal of Haematology, 94, 592, 10.1046/j.1365-2141.1996.d01-1835.x
Tutschka, 1987, Bone marrow transplantation for leukemia following a new busulfan and cyclophosphamide regimen, Blood, 70, 1382, 10.1182/blood.V70.5.1382.1382
Vermylen, 1994, Bone marrow transplantation for sickle cell disease: the European experience, American Journal of Pediatric Hematology/Oncology, 16, 18
Vermylen, 1988, Bone marrow transplantation in five children with sickle cell anaemia, Lancet, 1, 1427, 10.1016/S0140-6736(88)92239-8
Vichinsky, 1994, A cautionary note regarding hydroxyurea in sickle cell disease, Blood, 83, 1124, 10.1182/blood.V83.4.1124.1124
Walters, 1994, Bone marrow transplantation for thalassemia: the USA experience, American Journal of Pediatric Hematology/Oncology, 16, 11
Walters, 1995, Neurologic complications after allogeneic marrow transplantation for sickle cell anemia, Blood, 85, 879, 10.1182/blood.V85.4.879.bloodjournal854879
Walters, 1996, Bone marrow transplantation for sickle cell disease, New England Journal of Medicine, 335, 369, 10.1056/NEJM199608083350601
Weinstein, 1954, Radioactive sodium chromate for the study of survival of red blood cells. III. The abnormal hemoglobin syndromes, Blood, 9, 1155, 10.1182/blood.V9.12.1155.1155
