-
1
-
-
59449098985
-
Gene therapy for immunodeficiency due to adenosine deaminase deficiency
-
Aiuti, A., Cattaneo, F., Galimberti, S., et al. (2009). Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N. Engl. J. Med. 360, 447-458.
-
(2009)
N. Engl. J. Med.
, vol.360
, pp. 447-458
-
-
Aiuti, A.1
Cattaneo, F.2
Galimberti, S.3
-
2
-
-
0030983279
-
Clonogenic capacity and ex vivo expansion potential of umbilical cord blood progenitor cells are not impaired by cryopreservation
-
Almici, C., Carlo-Stella, C., Wagner, J.E., et al. (1997). Clonogenic capacity and ex vivo expansion potential of umbilical cord blood progenitor cells are not impaired by cryopreservation. Bone Marrow Transplant. 19, 1079-1084. (Pubitemid 27267730)
-
(1997)
Bone Marrow Transplantation
, vol.19
, Issue.11
, pp. 1079-1084
-
-
Almici, C.1
Carlo-Stella, C.2
Wagner, J.E.3
Mangoni, L.4
Garau, D.5
Re, A.6
Giachetti, R.7
Cesana, C.8
Rizzoli, V.9
-
3
-
-
47749098306
-
Relationship between mixed chimerism and rejection after bone marrow transplantation in thalassaemia
-
DOI 10.2450/2008.0051-07
-
Andreani, M., Testi, M., Battarra, M., et al. (2008). Relationship between mixed chimerism and rejection after bone marrow transplantation in thalassemia. Blood Transfusion 6, 143-149. (Pubitemid 352031647)
-
(2008)
Blood Transfusion
, vol.6
, Issue.3
, pp. 143-149
-
-
Andreani, M.1
Testi, M.2
Battarra, M.3
Indigeno, P.4
Guagnano, A.5
Polchi, P.6
Federici, G.7
Lucarelli, G.8
-
4
-
-
0029983005
-
Long-term cryopreservation of bone marrow for autologous transplantation
-
Attarian, H., Feng, Z., Buckner, C.D., et al. (1996). Long-term cryopreservation of bone marrow for autologous transplantation. Bone Marrow Transplant. 17, 425-430. (Pubitemid 26114504)
-
(1996)
Bone Marrow Transplantation
, vol.17
, Issue.3
, pp. 425-430
-
-
Attarian, H.1
Feng, Z.2
Buckner, C.D.3
MacLeod, B.4
Rowley, S.D.5
-
5
-
-
0025870705
-
Identification and comparison of CD34-positive cells and their subpopulations from normal peripheral blood and bone marrow using multicolor flow cytometry
-
Bender, J.G., Unverzagt, K.L., Walker, D.E., et al. (1991). Identification and comparison of CD34-positive cells and their subpopulations from normal peripheral blood and bone marrow using multicolor flow cytometry. Blood 77, 2591-2596.
-
(1991)
Blood
, vol.77
, pp. 2591-2596
-
-
Bender, J.G.1
Unverzagt, K.L.2
Walker, D.E.3
-
6
-
-
29744459415
-
Survival and complications in thalassemia
-
DOI 10.1196/annals.1345.006
-
Borgna-Pignatti, C., Cappellini, M.D., De Stefano, P., et al. (2005). Survival and complications in thalassemia. Ann. Acad. Sci. 1054, 40-47. (Pubitemid 43031009)
-
(2005)
Annals of the New York Academy of Sciences
, vol.1054
, pp. 40-47
-
-
Borgna-Pignatti, C.1
Cappellini, M.D.2
De Stefano, P.3
Del Vecchio, G.C.4
Forni, G.L.5
Gamberini, M.R.6
Ghilardi, R.7
Origa, R.8
Piga, A.9
Romeo, M.A.10
Zhao, H.11
Cnaan, A.12
-
7
-
-
20844460608
-
Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine
-
Borgna-Pignatti, C., Rugolotto, S., De Stefano, P., et al. (2004). Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica 89, 1187-1193. (Pubitemid 39390442)
-
(2004)
Haematologica
, vol.89
, Issue.10
, pp. 1187-1193
-
-
Borgna-Pignatti, C.1
Rugolotto, S.2
De Stefano, P.3
Zhao, H.4
Cappellini, M.D.5
Del Vecchio, G.C.6
Romeo, M.A.7
Forni, G.L.8
Gamberini, M.R.9
Ghilardi, R.10
Piga, A.11
Cnaan, A.12
-
8
-
-
0036327614
-
Induction of a hypercoagulability state and endothelial cell activation by granulocyte colony-stimulating factor in peripheral blood stem cell donors
-
DOI 10.1089/15258160260194820
-
Canales, M.A., Arrieta, R., Gomez-Rioja, R., et al. (2002). Induction of a hypercoagulability state and endothelial cell activation by granulocyte colony-stimulating factor in peripheral blood stem cell donors. J. Hematother. Stem Cell Res. 11, 675-681. (Pubitemid 34851799)
-
(2002)
Journal of Hematotherapy and Stem Cell Research
, vol.11
, Issue.4
, pp. 675-681
-
-
Canales, M.A.1
Arrieta, R.2
Gomez-Rioja, R.3
Diez, J.4
Jimenez-Yuste, V.5
Hernandez-Navarro, F.6
-
9
-
-
70449427834
-
Hematopoietic stem cell gene therapy with a lentivi-ral vector in X-linked adrenoleukodystrophy
-
Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C.C., et al. (2009). Hematopoietic stem cell gene therapy with a lentivi-ral vector in X-linked adrenoleukodystrophy. Science 326, 818-823.
-
(2009)
Science
, vol.326
, pp. 818-823
-
-
Cartier, N.1
Hacein-Bey-Abina, S.2
Bartholomae, C.C.3
-
10
-
-
77956928344
-
Transfusion independence and HMGA2 activation after gene therapy of human b-thalassaemia
-
Cavazzana-Calvo, M., Payen, E., Negre, O., et al. (2010). Transfusion independence and HMGA2 activation after gene therapy of human b-thalassaemia. Nature 467, 318-322.
-
(2010)
Nature
, vol.467
, pp. 318-322
-
-
Cavazzana-Calvo, M.1
Payen, E.2
Negre, O.3
-
11
-
-
0036092892
-
The hypercoagulable state in thalassemia
-
Eldor, A., and Rachmilewitz, E.A. (2002). The hypercoagulable state in thalassemia. Blood 99, 36-43.
-
(2002)
Blood
, vol.99
, pp. 36-43
-
-
Eldor, A.1
Rachmilewitz, E.A.2
-
12
-
-
0345355164
-
Spontaneous rupture of spleen during peripheral blood stem-cell mobilisation in a healthy donor
-
Falzetti, F., Aversa, F., Minelli, O., et al. (1999). Spontaneous rupture of spleen during peripheral blood stem-cell mobilization in a healthy donor. Lancet 353, 555. (Pubitemid 29078428)
-
(1999)
Lancet
, vol.353
, Issue.9152
, pp. 555
-
-
Falzetti, F.1
Aversa, F.2
Minelli, O.3
Tabilio, A.4
-
13
-
-
0028339641
-
Predominance of myeloid antigens in CD34-positive peripheral blood stem cells over those in bone marrow after administration of granulocyte colony- stimulating factor
-
Fukuda, T., Okamura, S., Shimoda, K., et al. (1994). Predominance of myeloid antigens in CD34-positive peripheral blood stem cells over those in bone marrow after administration of granulocyte colony-stimulating factor. Eur. J. Hae-matol. 52, 201-206. (Pubitemid 24205472)
-
(1994)
European Journal of Haematology
, vol.52
, Issue.4
, pp. 201-206
-
-
Fukuda, T.1
Okamura, S.2
Shimoda, K.3
Takamatsu, Y.4
Inaba, S.5
Harada, M.6
Niho, Y.7
-
14
-
-
77954833516
-
Efficacy of gene therapy for X-linked severe combined immunodeficiency
-
Hacein-Bey-Abina, S., Hauer, J., Lim, A., et al. (2010). Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 22, 355-364.
-
(2010)
N. Engl. J. Med.
, vol.22
, pp. 355-364
-
-
Hacein-Bey-Abina, S.1
Hauer, J.2
Lim, A.3
-
15
-
-
58149260470
-
Severe events in donors after allogeneic hematopoietic stem cell donation
-
Halter, J., Kodera, Y., Ispizua, A.U., et al. (2009). Severe events in donors after allogeneic hematopoietic stem cell donation. Haematologica 94, 94-101.
-
(2009)
Haematologica
, vol.94
, pp. 94-101
-
-
Halter, J.1
Kodera, Y.2
Ispizua, A.U.3
-
16
-
-
33947245648
-
Human progenitor cells rapidly mobilized by AMD3100 repopulate NOD/SCID mice with increased frequency in comparison to cells from the same donor mobilized by granulocyte colony stimulating factor
-
DOI 10.1016/j.bbmt.2006.12.445, PII S1083879106013073
-
Hess, D.A., Bonde, J., Craft, T.P., et al. (2007). Human progenitor cells rapidly mobilized by AMD3100 repopulate NOD/SCID mice with increased frequency in comparison to cells from the same donor mobilized by granulocyte colony stimulating factor. Biol. Blood Marrow Transplant. 13, 398-411. (Pubitemid 46421895)
-
(2007)
Biology of Blood and Marrow Transplantation
, vol.13
, Issue.4
, pp. 398-411
-
-
Hess, D.A.1
Bonde, J.2
Craft, T.C.3
Wirthlin, L.4
Hohm, S.5
Lahey, R.6
Todt, L.M.7
Dipersio, J.F.8
Devine, S.M.9
Nolta, J.A.10
-
17
-
-
67349103412
-
Correlation between characteristics of unrelated bone marrow donor and cell density of total nucleated cell in bone marrow harvest
-
Kao, R.H., Li, C.C., Shaw, C.K., et al. (2009). Correlation between characteristics of unrelated bone marrow donor and cell density of total nucleated cell in bone marrow harvest. Int. J. Hematol. 89, 227-230.
-
(2009)
Int. J. Hematol.
, vol.89
, pp. 227-230
-
-
Kao, R.H.1
Li, C.C.2
Shaw, C.K.3
-
18
-
-
34548677736
-
Similar Outcomes of Cryopreserved Allogeneic Peripheral Stem Cell Transplants (PBSCT) Compared to Fresh Allografts
-
DOI 10.1016/j.bbmt.2007.07.003, PII S1083879107003485
-
Kim, D.H., Jamal, N., Saragosa, R., et al. (2007). Similar outcomes of cryopreserved allogeneic peripheral stem cell transplants (PBSCT) compared with fresh allografts. Biol. Blood Marrow Transplant. 13, 1233-1243. (Pubitemid 47413022)
-
(2007)
Biology of Blood and Marrow Transplantation
, vol.13
, Issue.10
, pp. 1233-1243
-
-
Kim, D.H.1
Jamal, N.2
Saragosa, R.3
Loach, D.4
Wright, J.5
Gupta, V.6
Kuruvilla, J.7
Lipton, J.H.8
Minden, M.9
Messner, H.A.10
-
19
-
-
33646398840
-
AMD3100 mobilizes hematopoietic stem cells with long-term re-populating capacity in nonhuman primates
-
Larochelle, A., Krouse, A., Metzger, M., et al. (2006). AMD3100 mobilizes hematopoietic stem cells with long-term re-populating capacity in nonhuman primates. Blood 107, 3772-3778.
-
(2006)
Blood
, vol.107
, pp. 3772-3778
-
-
Larochelle, A.1
Krouse, A.2
Metzger, M.3
-
20
-
-
0033008609
-
A prospective study of G-CSF effects on hemostasis in allogeneic blood stem cell donors
-
LeBlanc, R., Roy, J., Demers, C., et al. (1999). A prospective study of G-CSF effects on hemostasis in allogeneic blood stem cell donors. Bone Marrow Transplant. 23, 991-996. (Pubitemid 29250842)
-
(1999)
Bone Marrow Transplantation
, vol.23
, Issue.10
, pp. 991-996
-
-
LeBlanc, R.1
Roy, J.2
Demers, C.3
Vu, L.4
Cantin, G.5
-
21
-
-
0033048789
-
Granulocyte colony-stimulating factor-mobilized peripheral blood stem cells in β-thalassemia patients: Kinetics of mobilization and composition of apheresis product
-
DOI 10.1016/S0301-472X(98)00045-9, PII S0301472X98000459
-
Li, K., Wong, A., Li, C.K., et al. (1999). Granulocyte colony-stimulating factor-mobilized peripheral blood stem cells in b-thalassemia patients: Kinetics of mobilization and composition of apheresis product. Exp. Hematol. 27, 526-532. (Pubitemid 29125062)
-
(1999)
Experimental Hematology
, vol.27
, Issue.3
, pp. 526-532
-
-
Li, K.1
Wong, A.2
Li, C.K.3
Shing, M.M.K.4
Chik, K.W.5
Tsang, K.S.6
Lai, H.7
Leung, T.F.8
Yuen, P.M.P.9
-
22
-
-
39549112062
-
Advances in the allogeneic transplantation for thalassemia
-
DOI 10.1016/j.blre.2007.10.001, PII S0268960X07000677
-
Lucarelli, G., and Gaziev, J. (2008). Advances in the allogeneic transplantation for thalassemia. Blood Rev. 22, 53-63. (Pubitemid 351282443)
-
(2008)
Blood Reviews
, vol.22
, Issue.2
, pp. 53-63
-
-
Lucarelli, G.1
Gaziev, J.2
-
23
-
-
0027184519
-
Marrow transplantation in patients with thalassemia responsive to iron chelation therapy
-
DOI 10.1056/NEJM199309163291204
-
Lucarelli, G., Galimberti, M., Polchi, P., et al. (1993). Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. N. Engl. J. Med. 329, 840-844. (Pubitemid 23267211)
-
(1993)
New England Journal of Medicine
, vol.329
, Issue.12
, pp. 840-844
-
-
Lucarelli, G.1
Galimberti, M.2
Polchi, P.3
Angelucci, E.4
Baronciani, D.5
Giardini, C.6
Andreani, M.7
Agostinelli, F.8
Albertini, F.9
Clift, R.A.10
-
24
-
-
45749135187
-
Vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of b-thalassemia
-
Miccio, A., Cesari, R., Lotti, F., et al. (2008). In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of b-thalassemia. Proc. Natl. Acad. Sci. U.S.A. 105, 10547-10552.
-
(2008)
Proc. Natl. Acad. Sci. U.S.A.
, vol.105
, pp. 10547-10552
-
-
Miccio, A.1
Cesari, R.2
Lotti, F.3
-
25
-
-
1842483157
-
Effect of peripheral blood progenitor cell dose on hematopoietic recovery: Identification of minimal progenitor cell requirements for rapid engraftment
-
DOI 10.1038/sj.bmt.1704412
-
Miyamoto, T., Shinozuka, T., Maeda, H., et al. (2004). Effect of peripheral blood progenitor cell dose on hematopoietic recovery: Identification of minimal progenitor cell requirements for rapid engraftment. Bone Marrow Transplant. 33, 589-595. (Pubitemid 38455920)
-
(2004)
Bone Marrow Transplantation
, vol.33
, Issue.6
, pp. 589-595
-
-
Miyamoto, T.1
Shinozuka, T.2
Maeda, H.3
Hirasawa, T.4
Muramatsu, T.5
Murakami, M.6
Makino, T.7
Itagaki, H.8
Nakamura, Y.9
-
26
-
-
44949128064
-
Global epidemiology of haemoglobin disorders and derived service indicators
-
DOI 10.2471/BLT.06.036673
-
Modell, B., and Darlison, M. (2008). Global epidemiology of haemoglobin disorders and derived service indicators. Bull. World Health Org. 86, 480-487. (Pubitemid 351821761)
-
(2008)
Bulletin of the World Health Organization
, vol.86
, Issue.6
, pp. 480-487
-
-
Modell, B.1
Darlison, M.2
-
27
-
-
33645734405
-
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
-
Ott, M.G., Schmidt, M., Schwarzwaelder, K., et al. (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 12, 401-409.
-
(2006)
Nat. Med.
, vol.12
, pp. 401-409
-
-
Ott, M.G.1
Schmidt, M.2
Schwarzwaelder, K.3
-
28
-
-
33644692533
-
Weighing the risks of G-CSF administration, leukopheresis, and standard marrow harvest: Ethical and safety considerations for normal pediatric hematopoietic cell donors
-
DOI 10.1002/pbc.20708
-
Pulsipher, M.A., Nagler, A., Iannone, R., et al. (2006). Weighing the risks of G-CSF administration, leukopheresis, and standard marrow harvest: Ethical and safety considerations for normal pediatric hematopoietic cell donors. Pediatr. Blood Cancer 46, 422-433. (Pubitemid 43334647)
-
(2006)
Pediatric Blood and Cancer
, vol.46
, Issue.4
, pp. 422-433
-
-
Pulsipher, M.A.1
Nagler, A.2
Iannone, R.3
Nelson, R.M.4
-
29
-
-
77954086292
-
Update on clinical experience with AMD3100, an SDF-1/CXCL12-CXCR4 inhibitor, in mobilization of hematopoietic stem and progenitor cells
-
Pusic, I., and Dipersio, J.F. (2010). Update on clinical experience with AMD3100, an SDF-1/CXCL12-CXCR4 inhibitor, in mobilization of hematopoietic stem and progenitor cells. Curr. Opin. Hematol. 17, 319-326.
-
(2010)
Curr. Opin. Hematol.
, vol.17
, pp. 319-326
-
-
Pusic, I.1
Dipersio, J.F.2
-
30
-
-
77956949262
-
Correction of b-thalassemia major by gene transfer in hemato-poietic progenitors of pediatric patients
-
Roselli, E.A., Mezzadra, R., Frittoli, M.C., et al. (2010). Correction of b-thalassemia major by gene transfer in hemato-poietic progenitors of pediatric patients. EMBO Mol. Med. 2, 315-328.
-
(2010)
EMBO Mol. Med.
, vol.2
, pp. 315-328
-
-
Roselli, E.A.1
Mezzadra, R.2
Frittoli, M.C.3
-
31
-
-
33846196263
-
Therapeutic options for patients with severe β-thalassemia: The need for globin gene therapy
-
DOI 10.1089/hum.2006.151
-
Sadelain, M., Boulad, F., Galanello, R., et al. (2007). Therapeutic options for patients with severe b-thalassemia: The need for globin gene therapy. Hum. Gene Ther. 18, 1-9. (Pubitemid 46105459)
-
(2007)
Human Gene Therapy
, vol.18
, Issue.1
, pp. 1-9
-
-
Sadelain, M.1
Boulad, F.2
Galanello, R.3
Giardina, P.4
Locatelli, F.5
Maggio, A.6
Rivella, S.7
Riviere, I.8
Tisdale, J.9
-
32
-
-
79954544384
-
Allogeneic sibling donor complications of haematopoietic progenitor cell collection in children: A multi-institutional survey of the PDWP-EBMT Group [abstract 233]
-
Styczynski, J., Elarouci, N., Labopin, M., et al. (2008). Allogeneic sibling donor complications of haematopoietic progenitor cell collection in children: A multi-institutional survey of the PDWP-EBMT Group [abstract 233]. Bone Marrow Transplant. 41, S35.
-
(2008)
Bone Marrow Transplant.
, vol.41
-
-
Styczynski, J.1
Elarouci, N.2
Labopin, M.3
-
33
-
-
48149106911
-
Thalassemia and hypercoagulability
-
Taher, A.T., Otrock, Z.K., Uthman, I., et al. (2008). Thalassemia and hypercoagulability. Blood Rev. 22, 283-292.
-
(2008)
Blood Rev.
, vol.22
, pp. 283-292
-
-
Taher, A.T.1
Otrock, Z.K.2
Uthman, I.3
-
34
-
-
73949117408
-
Update on survival in thalassemia major
-
Telfer, P. (2009). Update on survival in thalassemia major. Hemoglobin 33(Suppl. 1), S76-S80.
-
(2009)
Hemoglobin
, vol.33
, Issue.SUPPL. 1
-
-
Telfer, P.1
-
35
-
-
79954519863
-
Safety and efficacy of G-CSF mobilization in major b-thalassemia
-
Presented at the October 21-24, New York, NY
-
Yannaki, E., Papayannopoulou, T., Jonlin, E., et al. (2009a). Safety and efficacy of G-CSF mobilization in major b-thalassemia. Presented at the Ninth Cooley Anemia Symposium, October 21-24, New York, NY.
-
(2009)
Ninth Cooley Anemia Symposium
-
-
Yannaki, E.1
Papayannopoulou, T.2
Jonlin, E.3
-
36
-
-
77949733300
-
Mobilization of hematopoietic stem cells in a thalassemic mouse model: Implications for human gene therapy of thalassemia
-
Yannaki, E., Psatha, N., Athanasiou, E., et al. (2009b). Mobilization of hematopoietic stem cells in a thalassemic mouse model: Implications for human gene therapy of thalassemia. Hum. Gene Ther. 21, 299-310.
-
(2009)
Hum. Gene Ther.
, vol.21
, pp. 299-310
-
-
Yannaki, E.1
Psatha, N.2
Athanasiou, E.3
|