-
3
-
-
0035320886
-
Phenotype-genotype relationships in monogenic disease: Lessons from the thalassaemias
-
Weatherall DJ. Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias. Nat Rev Genet. 2001;2:245-255.
-
(2001)
Nat Rev Genet
, vol.2
, pp. 245-255
-
-
Weatherall, D.J.1
-
4
-
-
0343628721
-
Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin
-
May C, Rivella S, Callegari J, et al. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature. 2000; 406:82-86.
-
(2000)
Nature
, vol.406
, pp. 82-86
-
-
May, C.1
Rivella, S.2
Callegari, J.3
-
5
-
-
20844460608
-
Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine
-
Borgna-Pignatti C, Rugolotto S, De Stephano P, et al. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica. 2004;89:1187-1193.
-
(2004)
Haematologica
, vol.89
, pp. 1187-1193
-
-
Borgna-Pignatti, C.1
Rugolotto, S.2
De Stephano, P.3
-
6
-
-
0019967483
-
Marrow transplantation for thalassaemia
-
Thomas ED, Buckner CD, Sanders JE, et al. Marrow transplantation for thalassaemia. Lancet. 1982;2(8292):227-229.
-
(1982)
Lancet
, vol.2
, Issue.8292
, pp. 227-229
-
-
Thomas, E.D.1
Buckner, C.D.2
Sanders, J.E.3
-
7
-
-
0025070260
-
Bone marrow transplantation in patients with thalassemia
-
Lucarelli G, Galimberti M, Polchi P, et al. Bone marrow transplantation in patients with thalassemia. N Engl J Med. 1990;322(7):417-421.
-
(1990)
N Engl J Med
, vol.322
, Issue.7
, pp. 417-421
-
-
Lucarelli, G.1
Galimberti, M.2
Polchi, P.3
-
8
-
-
33846210823
-
Gene therapy for homozygous b-thalassemia
-
Kline R, ed. New York: Informa Healthcare
-
Boulad F. Gene therapy for homozygous b-thalassemia In: Kline R, ed. Pediatric Hematopoietic Stem Cell Transplantation. New York: Informa Healthcare; 2006:383-389.
-
(2006)
Pediatric Hematopoietic Stem Cell Transplantation
, pp. 383-389
-
-
Boulad, F.1
-
9
-
-
0036301696
-
The cure of thalassemia by bone marrow transplantation
-
Lucarelli G, Andreani M, Angelucci E. The cure of thalassemia by bone marrow transplantation. Blood Rev. 2002;16:81-85.
-
(2002)
Blood Rev
, vol.16
, pp. 81-85
-
-
Lucarelli, G.1
Andreani, M.2
Angelucci, E.3
-
10
-
-
0037097807
-
Unrelated donor bone marrow transplantation for thalassemia: The effect of extended haplotypes
-
La Nasa G, Giardini C, Argiolu F, et al. Unrelated donor bone marrow transplantation for thalassemia: the effect of extended haplotypes. Blood. 2002;99(12):4350-4356.
-
(2002)
Blood
, vol.99
, Issue.12
, pp. 4350-4356
-
-
La Nasa, G.1
Giardini, C.2
Argiolu, F.3
-
11
-
-
29744432093
-
Unrelated bone marrow transplantation for beta-thalassemia patients: The experience of the Italian bone marrow transplant group
-
La Nasa G, Argiolu F, Giardini C, et al. Unrelated bone marrow transplantation for beta-thalassemia patients: the experience of the Italian bone marrow transplant group. Ann N Y Acad Sci. 2005;1054:186-195.
-
(2005)
Ann N y Acad Sci
, vol.1054
, pp. 186-195
-
-
La Nasa, G.1
Argiolu, F.2
Giardini, C.3
-
12
-
-
0034036968
-
Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA non-identical sibling or parent transplants
-
Gaziev D, Galimberti M, Lucarelli G, et al. Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA non-identical sibling or parent transplants. Bone Marrow Transplant. 2000;25(8):815-821.
-
(2000)
Bone Marrow Transplant
, vol.25
, Issue.8
, pp. 815-821
-
-
Gaziev, D.1
Galimberti, M.2
Lucarelli, G.3
-
13
-
-
0037085774
-
Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene
-
May C, Rivella S, Chadburn A, Sadelain M. Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene. Blood. 2002;99:1902-1908.
-
(2002)
Blood
, vol.99
, pp. 1902-1908
-
-
May, C.1
Rivella, S.2
Chadburn, A.3
Sadelain, M.4
-
14
-
-
0037443467
-
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
-
Persons DA, Hargrove PW, Allay ER, Hanawa H, Nienhuis AW. 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. 2003;101:2175-2183.
-
(2003)
Blood
, vol.101
, pp. 2175-2183
-
-
Persons, D.A.1
Hargrove, P.W.2
Allay, E.R.3
Hanawa, H.4
Nienhuis, A.W.5
-
15
-
-
0037195091
-
Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells
-
Imren S, Payen E, Westerman KA, et al. Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc Natl Acad Sci U S A. 2002;99:14380-14385.
-
(2002)
Proc Natl Acad Sci U S A
, vol.99
, pp. 14380-14385
-
-
Imren, S.1
Payen, E.2
Westerman, K.A.3
-
16
-
-
33646779583
-
Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia
-
Sadelain M. Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia. Curr Opin Hematol. 2006;13:142-148.
-
(2006)
Curr Opin Hematol
, vol.13
, pp. 142-148
-
-
Sadelain, M.1
-
17
-
-
0037606048
-
A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer
-
Rivella S, May C, Chadburn A, Riviere I, Sadelain M. A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer. Blood. 2003;101:2932-2939.
-
(2003)
Blood
, vol.101
, pp. 2932-2939
-
-
Rivella, S.1
May, C.2
Chadburn, A.3
Riviere, I.4
Sadelain, M.5
-
18
-
-
15844397404
-
Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia
-
Andreani M, Manna M, Lucarelli G, et al. Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood. 1996;87:3494-3499.
-
(1996)
Blood
, vol.87
, pp. 3494-3499
-
-
Andreani, M.1
Manna, M.2
Lucarelli, G.3
-
19
-
-
0037189401
-
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
-
Aiuti A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science. 2002;296:2410-2413.
-
(2002)
Science
, vol.296
, pp. 2410-2413
-
-
Aiuti, A.1
Slavin, S.2
Aker, M.3
-
20
-
-
33645734405
-
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
-
Ott MG, Schmidt M, Schwarzwaelder K, et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006;12:401-409.
-
(2006)
Nat Med
, vol.12
, pp. 401-409
-
-
Ott, M.G.1
Schmidt, M.2
Schwarzwaelder, K.3
-
21
-
-
0032006125
-
Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases
-
Slavin S, Nagler A, Naparstek E, et al. Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood. 1998;91:756-763.
-
(1998)
Blood
, vol.91
, pp. 756-763
-
-
Slavin, S.1
Nagler, A.2
Naparstek, E.3
-
22
-
-
12144289379
-
Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia
-
Iannone R, Casella JF, Fuchs EJ, et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant. 2003;9:519-528.
-
(2003)
Biol Blood Marrow Transplant
, vol.9
, pp. 519-528
-
-
Iannone, R.1
Casella, J.F.2
Fuchs, E.J.3
-
23
-
-
29544433568
-
Gene therapy of severe combined immunodeficiency disease: Proof of principle of efficiency and safety issues. Gene therapy primary immunodeficiencies retrovirus lentivirus genome
-
discussion 786-1778
-
Fischer A, Hacein-Bey-Abina S, Lagresle C, et al. [Gene therapy of severe combined immunodeficiency disease: proof of principle of efficiency and safety issues. Gene therapy, primary immunodeficiencies, retrovirus, lentivirus, genome]. Bull Acad Natl Med. 2005;189:779-785. discussion 786-1778
-
(2005)
Bull Acad Natl Med
, vol.189
, pp. 779-785
-
-
Fischer, A.1
Hacein-Bey-Abina, S.2
Lagresle, C.3
-
24
-
-
0037443457
-
Side effects of retroviral gene transfer into hematopoietic stem cells
-
Baum C, Dullmann J, Li Z, et al. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003;101:2099-2114.
-
(2003)
Blood
, vol.101
, pp. 2099-2114
-
-
Baum, C.1
Dullmann, J.2
Li, Z.3
-
25
-
-
22044450389
-
Stem cell gene transfer: Insights into integration and hematopoiesis from primate genetic marking studies
-
Dunbar CE. Stem cell gene transfer: insights into integration and hematopoiesis from primate genetic marking studies. Ann N Y Acad Sci. 2005;1044:178-182.
-
(2005)
Ann N y Acad Sci
, vol.1044
, pp. 178-182
-
-
Dunbar, C.E.1
|