Gene therapy for β-thalassaemia: The continuing challenge

Expert Reviews in Molecular Medicine

Volumn 12, Issue , 2010, Pages

  Yannaki, Evangelia ^a   Emery, David W ^b,c   Stamatoyannopoulos, George ^b,c  

^a G PAPANICOLAOU HOSPITAL   (Greece)

^b UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF WASHINGTON   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLOBIN; GAMMA GLOBIN; GRANULOCYTE COLONY STIMULATING FACTOR; HYDROXYUREA; LENTIVIRUS VECTOR; RETROVIRUS VECTOR;

ADVERSE OUTCOME; BETA THALASSEMIA; BONE MARROW TRANSPLANTATION; CANCER RISK; CARCINOGENESIS; CHROMATIN; DISEASE ASSOCIATION; DRUG EFFICACY; GENE EXPRESSION REGULATION; GENE LOCUS; GENE THERAPY; GENE TRANSFER; GLOBIN GENE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; INSULATOR ELEMENT; MEDICAL RESEARCH; MYELOABLATIVE CONDITIONING; NONHUMAN; NUCLEAR REPROGRAMMING; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; REGULATOR GENE; REVIEW; STEM CELL GENE THERAPY; THERAPY EFFECT; VIRAL GENE DELIVERY SYSTEM; X LINKED SEVERE COMBINED IMMUNODEFICIENCY; BIOLOGICAL MODEL; GENE VECTOR;

BETA-THALASSEMIA; GENE THERAPY; GENETIC VECTORS; HUMANS; MODELS, BIOLOGICAL;

EID: 79952110958     PISSN: 14623994     EISSN: None     Source Type: Journal    
DOI: 10.1017/S1462399410001626     Document Type: Review

References (142)

1. Stamatoyannopoulos, G. et al. (2001). The Molecular Basis of Blood Diseases. W.B. Saunders, Philadelphia, PA, USA
2. Weatherall, D.J. and Clegg, J.B. (2001) Inherited haemoglobin disorders: an increasing global health problem. Bulletin of the World Health Organization 79, 704-712 (Pubitemid 32776838)
3. Lucarelli, G., Andreani, M. and Angelucci, E. (2002) The cure of thalassemia by bone marrow transplantation. Blood Reviews 16, 81-85 (Pubitemid 34742224)
4. Gaziev, D. et al. (1997) Graft-versus-host disease after bone marrow transplantation for thalassemia: an analysis of incidence and risk factors. Transplantation 63, 854-860 (Pubitemid 27150593)
5. Lucarrelli, G. et al. (1999) Bone marrow transplantation in adult thalassemic patients. Blood 93, 1164-1167
6. La Nasa, G. et al. (2002) Unrelated donor bone marrow transplantation for thalassemia: the effect of extended haplotypes. Blood 15, 4350-4356 (Pubitemid 34627200)
7. La Nasa, G. et al. (2005) Unrelated donor stem cell transplantation in adult patients with thalassemia. Bone Marrow Transplantation 36, 971-975 (Pubitemid 41741560)
8. Gaziev, D. et al. (2000) Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA-nonidentical sibling or parent transplants. Bone Marrow Transplantation 25, 815-821 (Pubitemid 30209673)
9. Lucarelli, G. and Gaziev, J. (2008) Advances in the allogeneic transplantation for thalassemia. Blood Reviews 22, 53-63 (Pubitemid 351282443)
10. Lisowski, L. and Sadelain, M. (2008) Current status of globin gene therapy for the treatment of beta-thalassaemia. British Journal of Haematology 141, 335-345 (Pubitemid 351522768)
11. Emery, D.W. et al. (2002) Hematopoietic stem cell gene therapy. International Journal of Hematology 75, 228-236 (Pubitemid 43014053)
12. Kohn, D.B. et al. (2003) American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Molecular Therapy 8, 180-187 (Pubitemid 37062828)
13. Berry, C. et al. (2006) Selection of target sites for mobile DNA integration in the human genome. PLoS Computation Biology 2, e157
14. Emery, D.W., Aker, M. and Stamatoyannopoulos, G. (2003) Chromatin insulators and position effects. In Gene Transfer and Expression in Mammalian Cells (Makrides S.C., ed.), pp. 381-395, EIC Laboratories, Inc., Norwood, MA, USA
15. Li, Q. et al. (2002) Locus control regions. Blood 100, 3077-3086 (Pubitemid 35217052)
16. Grosveld, F. et al. (1987) Position-independent, high-level expression of the human beta-globin gene in transgenic mice. Cell 51, 975-985
17. Blom van Assendelft, G. et al. (1989) The beta-globin dominant control region activates homologous and heterologous promoters in a tissue-specific manner. Cell 56, 969-977
18. Karlsson, S. et al. (1987) Retroviral-mediated transfer of genomic globin genes leads to regulated production of RNA and protein. Proceedings of the National Academy of Sciences of the United States of America 84, 2411-2415 (Pubitemid 17075877)
19. Dzierzak, E.A., Papayannopoulou, T. and Mulligan, R.C. (1988) Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells. Nature 331, 35-41 (Pubitemid 18053416)
20. Plavec, I. et al. (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, 1384-1392 (Pubitemid 23067379)
21. Novak, U. et al. (1990) High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells. Proceedings of the National Academy of Sciences of the United States of America 87, 3386-3390
22. Emery, D.W. et al. (1998) Development of a condensed locus control region cassette and testing in retrovirus vectors for A gamma-globin. Blood Cells, Molecules, and Diseases 24, 322-339 (Pubitemid 28524833)
23. Sadelain, M. et al. (1995) Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene. Proceedings of the National Academy of Sciences of the United States of America 92, 6728-6732
24. Miller, A.D. et al. (1988) Design of retrovirus vectors for transfer and expression of the human beta-globin gene. Journal of Virology 62, 4337-4345
25. Leboulch, P. et al. (1994) Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure. EMBO Journal 13, 3065-3076 (Pubitemid 24213458)
26. Emery, D.W. et al. (2002) Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo. Blood 100, 2012-2019 (Pubitemid 35001231)
27. Miyoshi, H. et al. (1999) Transduction of human CD34+cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283, 682-686
28. Kumar, M. et al. (2001) Systematic determination of the packaging limit of lentiviral vectors. Human Gene Therapy 12, 1893-1905 (Pubitemid 32947232)
29. May, C. et al. (2000) Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature 406, 82-86
30. Dull, T. et al. (1998) A third-generation lentivirus vector with a conditional packaging system. Journal of Virology 72, 8463-8471 (Pubitemid 28471781)
31. Miyoshi, H. et al. (1998) Development of a self-inactivating lentivirus vector. Journal of Virology 72, 8150-8157
32. Zufferey, R. et al. (1998) Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. Journal of Virology 72, 9873-9880 (Pubitemid 28520853)
33. Naldini, L. and Verma, I.M. (2000) Lentiviral vectors. Advances in Virus Research 55, 599-609
34. Kay, M.A., Glorioso, J.C. and Naldini, L. (2001) Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nature Medicine 7, 33-40 (Pubitemid 32095639)
35. Montini, E. et al. (2006) Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nature Biotechnology 24, 687-696 (Pubitemid 43882120)
36. Arumugam, P.I. et al. (2009) Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region. Molecular Therapy 17, 1929-1937
37. Pawliuk, R. et al. (2001) Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294, 2368-2371 (Pubitemid 33140563)
38. Imren, S. et al. (2002) Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proceedings of the National Academy of Sciences of the United States of America 99, 14380-14385 (Pubitemid 35257680)
39. Persons, D.A. et al. (2003) The degree of phenotypic correction of murine beta-thalassemia intermedia following lentiviral-mediated transfer of a human gamma-globin gene is influenced by chromosomal position effects and vector copy number. Blood 101, 2175-2183 (Pubitemid 36302055)
40. Rivella, S. et al. (2003) A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer. Blood 101, 2932-2939 (Pubitemid 36857977)
41. Miccio, A. et al. (2008) In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia. Proceedings of the National Academyof Sciences of the United States of America 105, 10547-10552
42. Perumbeti, A. et al. (2009) A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction. Blood 114, 1174-1185
43. Puthenveetil, G. et al. (2004) Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector. Blood 104, 3445-3453 (Pubitemid 39564412)
44. Aker, M. et al. (2007) Extended core sequences from the cHS4 insulator are necessary for protecting retroviral vectors from silencing position effects. Human Gene Therapy 18, 333-343 (Pubitemid 46709345)
45. Arumugam, P.I. et al. (2007) Improved human beta-globin expression from self-inactivating lentiviral vectors carrying the chicken hypersensitive site-4 (cHS4) insulator element. Molecular Therapy 15, 1863-1871 (Pubitemid 47434195)
46. May, C. et al. (2002) Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene. Blood 99, 1902-1908 (Pubitemid 34525467)
47. Cavazzana-Calvo, M. et al. (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669-672 (Pubitemid 30241569)
48. Hacein-Bey-Abina, S. et al. (2002) Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. New England Journal of Medicine 346, 1185-1193 (Pubitemid 34984579)
49. Gaspar, H.B. et al. (2004) Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 364, 2181-2187 (Pubitemid 40018447)
50. Aiuti, A. et al. (2009) Gene therapy for immunodeficiency due to adenosine deaminase deficiency. New England Journal of Medicine 360, 447-458
51. Hacein-Bey-Abina, S. et al. (2008) Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. Journal of Clinical Investigation 118, 3132-3142
52. Howe, S.J. et al. (2008) Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapyof SCID-X1 patients. Journal of Clinical Investigation 118, 3143-3150
53. Hacein-Bey-Abina, S. et al. (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302, 415-419 (Pubitemid 37296260)
54. Ott, M.G. et al. (2006) Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nature Medicine 12, 401-409
55. Stein, S. et al. (2010) Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nature Medicine 16, 198-204
56. Aiuti, A. et al. (2007) Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. Journal of Clinical Investigation 117, 2233-2240 (Pubitemid 47219568)
57. Li, Z. et al. (2002) Murine leukemia induced by retroviral gene marking. Science 296, 497 (Pubitemid 34408663)
58. Shou, Y. et al. (2006) Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy. Proceedings of the National Academy of Sciences of the United States of America 103, 11730-11735 (Pubitemid 44182519)
59. Kustikova, O. et al. (2005) Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 308, 1171-1174 (Pubitemid 40696434)
60. Li, C.L. et al. (2009) Genomic and functional assays demonstrate reduced gammaretroviral vector genotoxicity associated with use of the cHS4 chromatin insulator. Molecular Therapy 17, 716-724
61. Seggewiss, R. et al. (2006) Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque. Blood 107, 3865-3867 (Pubitemid 43726789)
62. Sadelain, M. (2004) Insertional oncogenesis in gene therapy: how much of a risk? Gene Therapy 11, 569-573
63. Baum, C. et al. (2002) Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 101, 2099-2114
64. Aker, M. et al. (2006) Integration bias of gammaretrovirus vectors following transduction and growth of primary mouse hematopoietic progenitor cells with and without selection. Molecular Therapy 14, 226-235 (Pubitemid 43942346)
65. Poeschla, E.M. (2008) Integrase, LEDGF/p75 and HIV replication. Cellular and Molecular Life Sciences 65, 1403-1424
66. Montini, E. et al. (2009) The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. Journal of Clinical Investigation 119, 964-975
67. Schroder, A.R. et al. (2002) HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110, 521-529 (Pubitemid 35232295)
68. Wu, W. et al. (2003) Transcription start regions in the human genome are favored targets for MLV integration. Science 300, 1749-1751
69. Zychlinski, D. et al. (2008) Physiological promoters reduce the genotoxic risk of integrating gene vectors. Molecular Therapy 16, 718-725 (Pubitemid 351426173)
70. Chang, A.H. and Sadelain, M. (2007) The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the LTR, and the promise of lineage-restricted vectors. Molecular Therapy 15, 445-456
71. Hargrove, P.W. et al. (2008) Globin lentiviral vector insertions can perturb the expression of endogenous genes in beta-thalassemic hematopoietic cells. Molecular Therapy 16, 525-533 (Pubitemid 351314992)
72. Gaszner, M. and Felsenfeld, G. (2006) Insulators: exploiting transcriptional and epigenetic mechanisms. Nature Reviews Genetics 7, 703-713 (Pubitemid 44260006)
73. Wallace, J.A. and Felsenfeld, G. (2007) We gather together: insulators and genome organization. Current Opinion in Genetics and Development 17, 400-407 (Pubitemid 350016897)
74. Chung, J.H., Bell, A.C. and Felsenfeld, G. (1997) Characterization of the chicken beta-globin insulator. Proceedings of the National Academy of Sciences of the United States of America 94, 575-580
75. Rivella, S. et al. (2000) The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites. Journal of Virology 74, 4679-4687 (Pubitemid 30237894)
76. Emery, D.W. et al. (2000) A chromatin insulator protects retrovirus vectors from chromosomal position effects. Proceedings of the National Academy of Sciences of the United States of America 97, 9150-9155 (Pubitemid 30626689)
77. Yannaki, E. et al. (2002) Topological constraints governing the use of the chicken HS4 chromatin insulator in oncoretrovirus vectors. Molecular Therapy 5, 589-598 (Pubitemid 34546089)
78. Evans-Galea, M.V. (2007) Suppression of clonal dominance in cultured human lymphoid cells by addition of the cHS4 insulator to a lentiviral vector. Molecular Therapy 15, 801-809
79. Ramezani, A., Hawley, T.S. and Hawley, R.G. (2008) Combinatorial incorporation of enhancer-blocking components of the chicken beta-globin 5′HS4 and human T-cell receptor alpha/delta BEAD-1 insulators in self-inactivating retroviral vectors reduces their genotoxic potential. Stem Cells 26, 3257-3266
80. Nishino, T. et al. (2006) Effects of human gamma-globin in murine beta-thalassaemia. British Journal of Haematology 134, 100-108 (Pubitemid 43830035)
81. Neff, T. et al. (2003) Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. Journal of Clinical Investigation 112, 1581-1588 (Pubitemid 38056284)
82. Ragg, S. et al. (2000) Direct reversal of DNA damage by mutant methyltransferase protein protects mice against dose-intensified chemotherapy and leads to in vivo selection of hematopoietic stem cells. Cancer Research 60, 5187-5195
83. Jin, L. et al. (2000) In vivo selection using a cell-growth switch. Nature Genetics 26, 64-66
84. Emery, D.W. et al. (2005) Selection with a regulated cell growth switch increases the likelihood of expression for a linked gamma-globin gene. Blood Cells, Molecules, and Diseases 34, 235-247 (Pubitemid 40693561)
85. Zhao, H. et al. (2009) Amelioration of murine beta-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both gamma-globin and the MGMT drug-resistance gene. Blood 113, 5747-5756
86. Burroughs, L. and Storb, R. (2005) Low-intensity allogeneic hematopoietic stem cell transplantation for myeloid malignancies: separating graft-versus-leukemia effects from graft-versus-host disease. Current Opinion in Hematology 12, 45-54 (Pubitemid 40189239)
87. Aiuti, A. et al. (2002) Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296, 2410-2413 (Pubitemid 34734219)
88. Mardiney, M. and Malech, H.L. (1996) Enhanced engraftment of hematopoietic progenitor cells in mice treated with granulocyte colony-stimulating factor before low-dose irradiation: implications for gene therapy. Blood 87, 4049-4056
89. Rosenzwieg, M. et al. (1999) Efficient and durable gene marking of hematopoietic progenitor cells in nonhuman primates after nonablative conditioning. Blood 94, 2271-2286
90. Bordignon, C. et al. (1995) Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 270, 470-475
91. Kohn, D.B. et al. (1995) Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nature Medicine 1, 1017-1023
92. Malech, H.L. et al. (1997) Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proceedings of the National Academyof Sciences of the United States of America 94, 12133-12138 (Pubitemid 27467855)
93. Kohn, D.B. et al. (1998) T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nature Medicine 4, 775-780 (Pubitemid 28331017)
94. Dunbar, C.E. et al. (1998) Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation. Human Gene Therapy 9, 2629-2640 (Pubitemid 29004635)
95. Schuening, F. et al. (1997) Retrovirus-mediated transfer of thecDNAfor human glucocerebrosidase into peripheral blood repopulating cells of patients with Gaucher's disease. Human Gene Therapy 8, 2143-2160 (Pubitemid 128756732)
96. Kelly, P. et al. (2007) Stem cell collection and gene transfer in Fanconi anemia. Molecular Therapy 15, 211-219 (Pubitemid 46043667)
97. Kang, E.M. et al. (2010) Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils. Blood 115, 783-791
98. Gaspar, H.B. et al. (2006) Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Molecular Therapy 14, 505-513 (Pubitemid 44339393)
99. Cartier, N. et al. (2009) Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326, 818-823
100. Bank, A., Dorazio, R. and Leboulch, P. (2005) A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia. Annals of the New York Academy of Sciences, 1054, 308-316 (Pubitemid 43031038)
101. Kaiser, J. (2010) Gene therapy. Beta-thalassemia treatment succeeds, with a caveat. Science 326, 1468-1469
102. Andreani, M. et al. (1996) Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood 87, 3494-3499 (Pubitemid 26115383)
103. Iannone, R. et al. (2003) Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biology of Blood and Marrow Transplantation 9, 519-528 (Pubitemid 38351796)
104. Persons, D.A. et al. (2001) Functional requirements for phenotypic correction of murine betathalassemia: implications for human gene therapy. Blood 97, 3275-3282
105. Gratwohl, A. et al. (2006)EBMTactivity survey 2004 and changes in disease indication over the past 15 years. Bone Marrow Transplantation 37, 1069-1085 (Pubitemid 43871516)
106. To, L.B. et al. (1997) The biology and clinical uses of blood stem cells. Blood 89, 2233-2258 (Pubitemid 27143269)
107. Dunbar, C.E. et al. (1996) Improved retroviral gene transfer into murine and Rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colonystimulating factor. Proceedings of the National Academy of Sciences of the United States of America 93, 11871-11876 (Pubitemid 26347216)
108. Horn, P.A. et al. (2004) Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol. Blood 103, 3710-3716 (Pubitemid 38596286)
109. Thomasson, B. et al. (2003) Direct comparison of steady-state marrow, primed marrow, and mobilized peripheral blood for transduction of hematopoietic stem cells in dogs. Human Gene Therapy 14, 1683-1686 (Pubitemid 37452878)
110. Hematti, P. et al. (2003) Retroviral transduction efficiency of G-CSF + SCF-mobilized peripheral blood CD34+cells is superior to G-CSF or G-CSF + Flt3-L-mobilized cells in nonhuman primates. Blood 101, 2199-2205 (Pubitemid 36302058)
111. Hematti, P. et al. (2004) Comparison of retroviral transduction efficiency in CD34+ cells derived from bone marrow versus G-CSF-mobilized or G-CSF plus stem cell factor-mobilized peripheral blood in nonhuman primates. Stem Cells 22, 1062-1069 (Pubitemid 39507092)
112. Bodine, D.M. et al. (1994) Efficient retrovirus transduction of mouse pluripotent hematopoietic stem cells mobilized into the peripheral blood by treatment with granulocyte colony-stimulating factor and stem cell factor. Blood 84, 1482-1491 (Pubitemid 24273016)
113. O'Malley, D.P., Whalen, M. and Banks, P.M. (2003) Spontaneous splenic rupture with fatal outcome following G-CSF administration for myelodysplastic syndrome. American Journal of Hematology 73, 294-295 (Pubitemid 36898891)
114. Brown, S.L. and Dale, D.C. (1997) Spontaneous splenic rupture following administration of granulocyte colony-stimulating factor (G-CSF): occurrence in an allogeneic donor of peripheral blood stem cells. Biology of Blood and Marrow Transplantation 3, 341-343
115. Becker, P.S. et al. (1997) Spontaneous splenic rupture following administration of granulocyte colony-stimulating factor (G-CSF): occurrence in an allogeneic donor of peripheral blood stem cells. Biology of Blood and Marrow Transplantation 3, 45-49 (Pubitemid 127531528)
116. Abboud, M., Laver, J. and Blau, C.A. (1998) Granulocytosis causing sickle-cell crisis. Lancet 351, 959 (Pubitemid 28138477)
117. Adler, B.K. et al. (2001) Fatal sickle cell crisis after granulocyte colony-stimulating factor administration. Blood 97, 3313-3314
118. Broxmeyer, H.E. et al. (2005) Rapid mobilization of murine and human hematopoietic stem and progenitor cells with AMD3100, a CXCR4 antagonist. Journal of Experimental Medicine 201, 1307-1318 (Pubitemid 40629073)
119. Larochelle, A. et al. (2006) AMD3100 mobilizes hematopoietic stem cells with long-term repopulating capacity in nonhuman primates. Blood 107, 3772-3778
120. Li, K. et al. (1999) Granulocyte colony-stimulating factor-mobilized peripheral blood stem cells in beta-thalassemia patients: kinetics of mobilization and composition of apheresis product. Experimental Hematology 27, 526-532 (Pubitemid 29125062)
121. Yannaki, E. et al. (2010) Mobilization of hematopoietic stem cells in a thalassemic mouse model: implications for human gene therapy of thalassemia. Human Gene Therapy 21, 299-310
122. Yannaki, E. and Stamatoyannopoulos, G. (2010) Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease. Annals of the New York Academy of Sciences 1202, 59-63
123. Sadelain, M. et al. (2007) Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy. Human Gene Therapy 18, 1-9 (Pubitemid 46105459)
124. Persons, D.A. (2009) Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders. Hematology/The Education Program of the American Society of Hematology 2009, 690-697
125. Pathak, V.K. and Temin, H.M. (1990) Broad spectrum of in vivo forward mutations, hypermutations, and mutational hotspots in a retroviral shuttle vector after a single replication cycle: deletions and deletions with insertions. Proceedings of theNational Academyof Sciences of the United States of America 87, 6024-6028
126. Patel, M. and Yang, S. (2010) Advances in reprogramming somatic cells to induced pluripotent stem cells. Stem Cell Reviews 6, 367-380
127. Rashid, S.T. and Vallier, L. (2010) Induced pluripotent stem cells - alchemist's tale or clinical reality? Expert Reviews in Molecular Medicine 12, e25
128. Takahashi, K. and Yamanaka, S. (2006) Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 126, 663-676 (Pubitemid 44233629)
129. Yu, J. et al. (2007) Induced pluripotent stem cell lines derived from human somatic cells. Science 318, 1917-1920
130. Yu, J. et al. (2009) Human induced pluripotent stem cells free of vector and transgene sequences. Science 324, 797-801
131. Kim, D. et al. (2009) Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins. Cell Stem Cells 4, 472-476
132. Ebert, A.D. et al. (2009) Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature 457, 277-280
133. Lee, G. et al. (2009) Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs. Nature 461, 402-406
134. Kiskinis, E. and Eggan, K. (2010) Progress toward the clinical application of patient-specific pluripotent stem cells. Journal of Clinical Investigation 120, 51-59
135. Park, I. et al. (2008) Disease-specific induced pluripotent stem cells. Cell 134, 877-886
136. Hanna, J. et al. (2007) Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 318, 1920-1923
137. Rega, A. et al. (2009) Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature 460, 53-49
138. Ye, L. et al. (2009) Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases. Proceedings of the National Academy of Sciences of the United States of America 106, 9826-9830
139. Okita, K. and Yamanaka, S. (2010) Induction of pluripotency by defined factors. Experimental Cell Research 316, 2565-2570
140. Yamanaka, S. and Blau, H.M. (2010) Nuclear reprogramming to a pluripotent state by three approaches. Nature 465, 704-712
141. Aiuti, A. and Roncarolo, M.G. (2010) Ten years of gene therapy for primary immune deficiencies. Hematology/The Education Program of the American Society of Hematology 2009, 682-689
142. Gaspar, H.B. et al. (2009) How I treat ADA deficiency. Blood 114, 3524-3532