-
1
-
-
77953952024
-
The inherited diseases of hemoglobin are an emerging global health burden
-
DJ Weatherall The inherited diseases of hemoglobin are an emerging global health burden Blood 115 2010 4331 4336
-
(2010)
Blood
, vol.115
, pp. 4331-4336
-
-
Weatherall, D.J.1
-
4
-
-
0035676917
-
Towards molecular medicine; reminiscences of the haemoglobin field, 1960-2000
-
DOI 10.1046/j.1365-2141.2001.03227.x
-
DJ Weatherall Towards molecular medicine; reminiscences of the haemoglobin field, 1960-2000 Br J Haematol 115 2001 729 738 (Pubitemid 34042887)
-
(2001)
British Journal of Haematology
, vol.115
, Issue.4
, pp. 729-738
-
-
Weatherall, D.J.1
-
5
-
-
0036839549
-
Locus control regions
-
DOI 10.1182/blood-2002-04-1104
-
Q Li, KR Peterson, X Fang, G Stamatoyannopoulos Locus control regions Blood 100 2002 3077 3086 (Pubitemid 35217052)
-
(2002)
Blood
, vol.100
, Issue.9
, pp. 3077-3086
-
-
Li, Q.1
Peterson, K.R.2
Fang, X.3
Stamatoyannopoulos, G.4
-
6
-
-
39049092286
-
β-globin regulation and long-range interactions
-
RJ Palstra, W de Laat, F Grosveld β-globin regulation and long-range interactions Adv Genet 61 2008 107 142
-
(2008)
Adv Genet
, vol.61
, pp. 107-142
-
-
Palstra, R.J.1
De Laat, W.2
Grosveld, F.3
-
7
-
-
0000831381
-
Hemoglobin switching
-
G. Stamatoyannopoulos P.W. Majerus R.M. Perlmutter H. Varmus 3rd edn. WB Saunders Company Philadelphia
-
G Stamatoyannopoulos, F Grosveld Hemoglobin switching G Stamatoyannopoulos PW Majerus RM Perlmutter H Varmus The molecular basis of blood diseases 3rd edn. 2001 WB Saunders Company Philadelphia 136 182
-
(2001)
The Molecular Basis of Blood Diseases
, pp. 136-182
-
-
Stamatoyannopoulos, G.1
Grosveld, F.2
-
8
-
-
41949095010
-
Long-range regulation of α globin gene expression during erythropoiesis
-
DOI 10.1097/MOH.0b013e3282f734c4, PII 0006275220080500000005
-
DR Higgs, WG Wood Long-range regulation of alpha globin gene expression during erythropoiesis Curr Opin Hematol 15 2008 176 183 (Pubitemid 351507568)
-
(2008)
Current Opinion in Hematology
, vol.15
, Issue.3
, pp. 176-183
-
-
Higgs, D.R.1
Wood, W.G.2
-
9
-
-
84856391499
-
A developmental approach to hematopoiesis
-
M.H. Steinberg B.G. Forget D.R. Higgs D.J. Weatherall 2nd edn. Cambridge University Press Cambridge
-
E Dzierzak A developmental approach to hematopoiesis MH Steinberg BG Forget DR Higgs DJ Weatherall Disorders of hemoglobin: genetics, pathophysiology and clinical management 2nd edn. 2009 Cambridge University Press Cambridge 3 23
-
(2009)
Disorders of Hemoglobin: Genetics, Pathophysiology and Clinical Management
, pp. 3-23
-
-
Dzierzak, E.1
-
10
-
-
41949100415
-
Ontogeny of erythropoiesis
-
DOI 10.1097/MOH.0b013e3282f97ae1, PII 0006275220080500000002
-
J Palis Ontogeny of erythropoiesis Curr Opin Hematol 15 2008 155 161 (Pubitemid 351507565)
-
(2008)
Current Opinion in Hematology
, vol.15
, Issue.3
, pp. 155-161
-
-
Palis, J.1
-
11
-
-
84856375895
-
Erythropoiesis
-
M.H. Steinberg B.G. Forget D.R. Higgs D.J. Weatherall 2nd edn. Cambridge University Press Cambridge
-
S Philipsen, WG Wood Erythropoiesis MH Steinberg BG Forget DR Higgs DJ Weatherall Disorders of hemoglobin 2nd edn. 2009 Cambridge University Press Cambridge 24 45
-
(2009)
Disorders of Hemoglobin
, pp. 24-45
-
-
Philipsen, S.1
Wood, W.G.2
-
13
-
-
44149110031
-
Fine tuning of globin gene expression by DNA methylation
-
A Goren, G Simchen, E Fibach et al. Fine tuning of globin gene expression by DNA methylation PLoS One 1 2006 e46
-
(2006)
PLoS One
, vol.1
, pp. 46
-
-
Goren, A.1
Simchen, G.2
Fibach, E.3
-
14
-
-
34548016809
-
Developmental- and differentiation-specific patterns of human γ- and β-globin promoter DNA methylation
-
DOI 10.1182/blood-2007-01-068635
-
R Mabaera, CA Richardson, K Johnson, M Hsu, S Fiering, CH Lowrey Developmental- and differentiation-specific patterns of human γ- and β-globin promoter DNA methylation Blood 110 2007 1343 1352 (Pubitemid 47281435)
-
(2007)
Blood
, vol.110
, Issue.4
, pp. 1343-1352
-
-
Mabaera, R.1
Richardson, C.A.2
Johnson, K.3
Hsu, M.4
Fiering, S.5
Lowrey, C.H.6
-
16
-
-
39549112484
-
Intergenic transcription, cell-cycle and the developmentally regulated epigenetic profile of the human beta-globin locus
-
J Miles, JA Mitchell, L Chakalova et al. Intergenic transcription, cell-cycle and the developmentally regulated epigenetic profile of the human beta-globin locus PLoS One 2 2007 e630
-
(2007)
PLoS One
, vol.2
, pp. 630
-
-
Miles, J.1
Mitchell, J.A.2
Chakalova, L.3
-
17
-
-
0034610814
-
The language of covalent histone modifications
-
DOI 10.1038/47412
-
BD Strahl, CD Allis The language of covalent histone modifications Nature 403 2000 41 45 (Pubitemid 30038513)
-
(2000)
Nature
, vol.403
, Issue.6765
, pp. 41-45
-
-
Strahl, B.D.1
Allis, C.D.2
-
18
-
-
39049113889
-
Three-dimensional organization of gene expression in erythroid cells
-
W de Laat, P Klous, J Kooren et al. Three-dimensional organization of gene expression in erythroid cells Curr Top Dev Biol 82 2008 117 139
-
(2008)
Curr Top Dev Biol
, vol.82
, pp. 117-139
-
-
De Laat, W.1
Klous, P.2
Kooren, J.3
-
19
-
-
0037071383
-
Transcriptional regulation of erythropoiesis: An affair involving multiple partners
-
DOI 10.1038/sj.onc.1205326
-
AB Cantor, SH Orkin Transcriptional regulation of erythropoiesis: an affair involving multiple partners Oncogene 21 2002 3368 3376 (Pubitemid 34587709)
-
(2002)
Oncogene
, vol.21
, pp. 3368-3376
-
-
Cantor, A.B.1
Orkin, S.H.2
-
20
-
-
77949846880
-
Advances in the understanding of haemoglobin switching
-
VG Sankaran, J Xu, SH Orkin Advances in the understanding of haemoglobin switching Br J Haematol 149 2010 181 194
-
(2010)
Br J Haematol
, vol.149
, pp. 181-194
-
-
Sankaran, V.G.1
Xu, J.2
Orkin, S.H.3
-
21
-
-
13844318521
-
Control of globin gene expression during development and erythroid differentiation
-
DOI 10.1016/j.exphem.2004.11.007
-
G Stamatoyannopoulos Control of globin gene expression during development and erythroid differentiation Exp Hematol 33 2005 259 271 (Pubitemid 40261307)
-
(2005)
Experimental Hematology
, vol.33
, Issue.3
, pp. 259-271
-
-
Stamatoyannopoulos, G.1
-
22
-
-
33747453473
-
Insulators: Exploiting transcriptional and epigenetic mechanisms
-
DOI 10.1038/nrg1925, PII NRG1925
-
M Gaszner, G Felsenfeld Insulators: exploiting transcriptional and epigenetic mechanisms Nat Rev Genet 7 2006 703 713 (Pubitemid 44260006)
-
(2006)
Nature Reviews Genetics
, vol.7
, Issue.9
, pp. 703-713
-
-
Gaszner, M.1
Felsenfeld, G.2
-
23
-
-
79953216652
-
Systematic documentation and analysis of human genetic variation in hemaglobinopathies using the microattribution approach
-
B Giardine, J Borg, DR Higgs et al. Systematic documentation and analysis of human genetic variation in hemaglobinopathies using the microattribution approach Nat Genet 43 2011 295 301
-
(2011)
Nat Genet
, vol.43
, pp. 295-301
-
-
Giardine, B.1
Borg, J.2
Higgs, D.R.3
-
24
-
-
0037365343
-
Screening and genetic diagnosis of haemoglobin disorders
-
DOI 10.1016/S0268-960X(02)00061-9
-
JM Old Screening and genetic diagnosis of haemoglobin disorders Blood Rev 17 2003 43 53 (Pubitemid 36176210)
-
(2003)
Blood Reviews
, vol.17
, Issue.1
, pp. 43-53
-
-
Old, J.M.1
-
25
-
-
0031824755
-
The hemoglobin E syndromes
-
DOI 10.1111/j.1749-6632.1998.tb10490.x
-
DC Rees, J Styles, EP Vichinsky, JB Clegg, DJ Weatherall The hemoglobin E syndromes Ann N Y Acad Sci 850 1998 334 343 (Pubitemid 28369774)
-
(1998)
Annals of the New York Academy of Sciences
, vol.850
, pp. 334-343
-
-
Rees, D.C.1
Styles, L.2
Vichinsky, E.P.3
Clegg, J.B.4
Weatherall, D.J.5
-
27
-
-
0021014396
-
β-Globin gene inactivation by DNA translocation in γβ-thalassaemia
-
D Kioussis, E Vanin, T deLange, RA Flavell, FG Grosveld β-globin gene inactivation by DNA translocation in γ β-thalassaemia Nature 306 1983 662 666 (Pubitemid 14201849)
-
(1983)
Nature
, vol.306
, Issue.5944
, pp. 662-666
-
-
Kioussis, D.1
Vanin, E.2
DeLange, T.3
-
28
-
-
0031871713
-
Molecular basis of hereditary persistence of fetal hemoglobin
-
DOI 10.1111/j.1749-6632.1998.tb10460.x
-
BG Forget Molecular basis of hereditary persistence of fetal hemoglobin Ann N Y Acad Sci 850 1998 38 44 (Pubitemid 28369744)
-
(1998)
Annals of the New York Academy of Sciences
, vol.850
, pp. 38-44
-
-
Forget, B.G.1
-
29
-
-
84932648232
-
The molecular basis of β thalassemia, δβ thalassemia and hereditary persistence of fetal hemoglobin
-
M.H. Steinberg B.G. Forget D.R. Higgs D.J. Weatherall 2nd edn. Cambridge University Press Cambridge
-
SL Thein, WG Wood The molecular basis of β thalassemia, δβ thalassemia and hereditary persistence of fetal hemoglobin MH Steinberg BG Forget DR Higgs DJ Weatherall Disorders of hemoglobin 2nd edn. 2009 Cambridge University Press Cambridge 323 356
-
(2009)
Disorders of Hemoglobin
, pp. 323-356
-
-
Thein, S.L.1
Wood, W.G.2
-
30
-
-
77958494782
-
ATR-X syndrome protein targets tandem repeats and influences allele-specific expression in a size-dependent manner
-
MJ Law, KM Lower, HP Voon et al. ATR-X syndrome protein targets tandem repeats and influences allele-specific expression in a size-dependent manner Cell 143 2010 367 378
-
(2010)
Cell
, vol.143
, pp. 367-378
-
-
Law, M.J.1
Lower, K.M.2
Voon, H.P.3
-
31
-
-
77956622584
-
Haploinsufficiency for the erythroid transcription factor KLF1 causes hereditary persistence of fetal hemoglobin
-
J Borg, P Papadopoulos, M Georgitsi et al. Haploinsufficiency for the erythroid transcription factor KLF1 causes hereditary persistence of fetal hemoglobin Nat Genet 42 2010 801 805
-
(2010)
Nat Genet
, vol.42
, pp. 801-805
-
-
Borg, J.1
Papadopoulos, P.2
Georgitsi, M.3
-
32
-
-
21444433652
-
GATA transcription factors in hematologic disease
-
DOI 10.1532/IJH97.04180
-
AB Cantor GATA transcription factors in hematologic disease Int J Hematol 81 2005 378 384 (Pubitemid 40917679)
-
(2005)
International Journal of Hematology
, vol.81
, Issue.5
, pp. 378-384
-
-
Cantor, A.B.1
-
33
-
-
77956630402
-
KLF1 regulates BCL11A expression and γ- To β-globin gene switching
-
D Zhou, K Liu, CW Sun, KM Pawlik, TM Townes KLF1 regulates BCL11A expression and γ- to β-globin gene switching Nat Genet 42 2010 742 744
-
(2010)
Nat Genet
, vol.42
, pp. 742-744
-
-
Zhou, D.1
Liu, K.2
Sun, C.W.3
Pawlik, K.M.4
Townes, T.M.5
-
34
-
-
78649469071
-
Fine-mapping at three loci known to affect fetal hemoglobin levels explains additional genetic variation
-
G Galarneau, CD Palmer, VG Sankaran, SH Orkin, JN Hirschhorn, G Lettre Fine-mapping at three loci known to affect fetal hemoglobin levels explains additional genetic variation Nat Genet 42 2010 1049 1051
-
(2010)
Nat Genet
, vol.42
, pp. 1049-1051
-
-
Galarneau, G.1
Palmer, C.D.2
Sankaran, V.G.3
Orkin, S.H.4
Hirschhorn, J.N.5
Lettre, G.6
-
35
-
-
34748864128
-
A QTL influencing F cell production maps to a gene encoding a zinc-finger protein on chromosome 2p15
-
DOI 10.1038/ng2108, PII NG2108
-
S Menzel, C Garner, I Gut et al. A QTL influencing F cell production maps to a gene encoding a zinc-finger protein on chromosome 2p15 Nat Genet 39 2007 1197 1199 (Pubitemid 47482679)
-
(2007)
Nature Genetics
, vol.39
, Issue.10
, pp. 1197-1199
-
-
Menzel, S.1
Garner, C.2
Gut, I.3
Matsuda, F.4
Yamaguchi, M.5
Heath, S.6
Foglio, M.7
Zelenika, D.8
Boland, A.9
Rooks, H.10
Best, S.11
Spector, T.D.12
Farrall, M.13
Lathrop, M.14
Thein, S.L.15
-
36
-
-
34547450531
-
Intergenic variants of HBS1L-MYB are responsible for a major quantitative trait locus on chromosome 6q23 influencing fetal hemoglobin levels in adults
-
DOI 10.1073/pnas.0611393104
-
SL Thein, S Menzel, X Peng et al. Intergenic variants of HBS1L-MYB are responsible for a major quantitative trait locus on chromosome 6q23 influencing fetal hemoglobin levels in adults Proc Natl Acad Sci USA 104 2007 11346 11351 (Pubitemid 47175143)
-
(2007)
Proceedings of the National Academy of Sciences of the United States of America
, vol.104
, Issue.27
, pp. 11346-11351
-
-
Thein, S.L.1
Menzel, S.2
Peng, X.3
Best, S.4
Jiang, J.5
Close, J.6
Silver, N.7
Gerovasilli, A.8
Ping, C.9
Yamaguchi, M.10
Wahlberg, K.11
Ulug, P.12
Spector, T.D.13
Garner, C.14
Matsuda, F.15
Farrall, M.16
Lathrop, M.17
-
37
-
-
77949807007
-
Control of fetal hemoglobin: New insights emerging from genomics and clinical implications
-
SL Thein, S Menzel, M Lathrop, C Garner Control of fetal hemoglobin: new insights emerging from genomics and clinical implications Hum Mol Genet 18 2009 R216 R223
-
(2009)
Hum Mol Genet
, vol.18
-
-
Thein, S.L.1
Menzel, S.2
Lathrop, M.3
Garner, C.4
-
38
-
-
50149117726
-
DNA polymorphisms at the BCL11A, HBSIL-MYB, and β-globin loci associate with fetal hemoglobin levels and pain crises in sickle cell disease
-
G Lettre, VG Sankaran, MAC Bezerra et al. DNA polymorphisms at the BCL11A, HBSIL-MYB, and β-globin loci associate with fetal hemoglobin levels and pain crises in sickle cell disease Proc Natl Acad Sci USA 105 2008 11869 11874
-
(2008)
Proc Natl Acad Sci USA
, vol.105
, pp. 11869-11874
-
-
Lettre, G.1
Sankaran, V.G.2
Bezerra, M.A.C.3
-
39
-
-
77949274495
-
A genome-wide association identified the common genetic variants influence disease severity in β0-thalassemia/hemoglobin e
-
M Nuinoon, W Makarasara, T Mushiroda et al. A genome-wide association identified the common genetic variants influence disease severity in β0-thalassemia/hemoglobin E Hum Genet 127 2010 303 314
-
(2010)
Hum Genet
, vol.127
, pp. 303-314
-
-
Nuinoon, M.1
Makarasara, W.2
Mushiroda, T.3
-
40
-
-
40349092939
-
Genome-wide association study shows BCL11A associated with persistent fetal hemoglobin and amelioration of the phenotype of β-thalassemia
-
DOI 10.1073/pnas.0711566105
-
M Uda, R Galanello, S Sanna et al. Genome-wide association study shows BCL11A associated with persistent fetal hemoglobin and amelioration of the phenotype of β-thalassemia Proc Natl Acad Sci USA 105 2008 1620 1625 (Pubitemid 351346564)
-
(2008)
Proceedings of the National Academy of Sciences of the United States of America
, vol.105
, Issue.5
, pp. 1620-1625
-
-
Uda, M.1
Galanello, R.2
Sanna, S.3
Lettre, G.4
Sankaran, V.G.5
Chen, W.6
Usala, G.7
Busonero, F.8
Maschio, A.9
Albai, G.10
Piras, M.G.11
Sestu, N.12
Lai, S.13
Dei, M.14
Mulas, A.15
Crisponi, L.16
Naitza, S.17
Asunis, I.18
Deiana, M.19
Nagaraja, R.20
Perseu, L.21
Satta, S.22
Cipollina, M.D.23
Sollaino, C.24
Moi, P.25
Hirschhorn, J.N.26
Orkin, S.H.27
Abecasis, G.R.28
Schlessinger, D.29
Cao, A.30
more..
-
41
-
-
77955905915
-
The role of antioxidants and iron chelators in the treatment of oxidative stress in thalassemia
-
E Fibach, EA Rachmilewitz The role of antioxidants and iron chelators in the treatment of oxidative stress in thalassemia Ann N Y Acad Sci 1202 2010 10 16
-
(2010)
Ann N y Acad Sci
, vol.1202
, pp. 10-16
-
-
Fibach, E.1
Rachmilewitz, E.A.2
-
42
-
-
77955906936
-
Hepcidin and Hfe in iron overload in β-thalassemia
-
S Gardenghi, P Ramos, A Follenzi et al. Hepcidin and Hfe in iron overload in β-thalassemia Ann N Y Acad Sci 1202 2010 221 225
-
(2010)
Ann N y Acad Sci
, vol.1202
, pp. 221-225
-
-
Gardenghi, S.1
Ramos, P.2
Follenzi, A.3
-
43
-
-
57349129112
-
Controlling α-globin: A review of α-globin expression and its impact on β-thalassemia
-
HP Voon, J Vadolas Controlling α-globin: a review of α-globin expression and its impact on β-thalassemia Haematologica 93 2008 1868 1876
-
(2008)
Haematologica
, vol.93
, pp. 1868-1876
-
-
Voon, H.P.1
Vadolas, J.2
-
44
-
-
44949128064
-
Global epidemiology of haemoglobin disorders and derived service indicators
-
DOI 10.2471/BLT.06.036673
-
B Modell, M Darlison Global epidemiology of haemoglobin disorders and derived service indicators Bull World Health Organ 86 2008 480 487 (Pubitemid 351821761)
-
(2008)
Bulletin of the World Health Organization
, vol.86
, Issue.6
, pp. 480-487
-
-
Modell, B.1
Darlison, M.2
-
46
-
-
0025776299
-
Reproductive behavior and health in consanguineous marriages
-
AH Bittles, WM Mason, J Greene, NA Rao Reproductive behavior and health in consanguineous marriages Science 252 1991 789 794 (Pubitemid 121000514)
-
(1991)
Science
, vol.252
, Issue.5007
, pp. 789-794
-
-
Bittles, A.H.1
Mason, W.M.2
Greene, J.3
Rao, N.A.4
-
47
-
-
14644392013
-
Thalassemia
-
AR Cohen, R Galanello, DJ Pennell, MJ Cunningham, E Vichinsky Thalassemia Hematology Am Soc Hematol Educ Program 2004 14 34
-
(2004)
Hematology Am Soc Hematol Educ Program
, pp. 14-34
-
-
Cohen, A.R.1
Galanello, R.2
Pennell, D.J.3
Cunningham, M.J.4
Vichinsky, E.5
-
48
-
-
42049093484
-
Studies in haemoglobin E beta-thalassaemia
-
DOI 10.1111/j.1365-2141.2008.07126.x
-
NF Olivieri, GM Muraca, A O'Donnell, A Premawardhena, C Fisher, DJ Weatherall Studies in haemoglobin E beta-thalassaemia Br J Haematol 141 2008 388 397 (Pubitemid 351521155)
-
(2008)
British Journal of Haematology
, vol.141
, Issue.3
, pp. 388-397
-
-
Olivieri, N.F.1
Muraca, G.M.2
O'Donnell, A.3
Premawardhena, A.4
Fisher, C.5
Weatherall, D.J.6
-
49
-
-
0032445486
-
Thalassaemia: Clinical management
-
NF Olivieri Thalassaemia: clinical management Baillieres Clin Haematol 11 1998 147 162
-
(1998)
Baillieres Clin Haematol
, vol.11
, pp. 147-162
-
-
Olivieri, N.F.1
-
50
-
-
84932611834
-
Clinical aspects of beta thalassemia and related disorders
-
M.H. Steinberg B.G. Forget D.R. Higgs D.J. Weatherall 2nd edn. Cambridge University Press Cambridge
-
NF Olivieri, DJ Weatherall Clinical aspects of beta thalassemia and related disorders MH Steinberg BG Forget DR Higgs DJ Weatherall Disorders of hemoglobin 2nd edn. 2009 Cambridge University Press Cambridge 357 416
-
(2009)
Disorders of Hemoglobin
, pp. 357-416
-
-
Olivieri, N.F.1
Weatherall, D.J.2
-
51
-
-
20844460608
-
Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine
-
C Borgna-Pignatti, S Rugolotto, P De Stefano et al. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine Haematologica 89 2004 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
-
52
-
-
0028059813
-
Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major
-
DOI 10.1056/NEJM199409013310902
-
GM Brittenham, PM Griffith, AW Nienhuis et al. Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major N Engl J Med 331 1994 567 573 (Pubitemid 24265698)
-
(1994)
New England Journal of Medicine
, vol.331
, Issue.9
, pp. 567-573
-
-
Brittenham, G.M.1
Griffith, P.M.2
Nienhuis, A.W.3
McLaren, C.E.4
Young, N.S.5
Tucker, E.E.6
Allen, C.J.7
Farrell, D.E.8
Harris, J.W.9
-
53
-
-
0028086414
-
Survival in medically treated patients with homozygous β-thalassemia
-
DOI 10.1056/NEJM199409013310903
-
NF Olivieri, DG Nathan, JH MacMillan et al. Survival in medically treated patients with homozygous β-thalassemia N Engl J Med 331 1994 574 578 (Pubitemid 24265699)
-
(1994)
New England Journal of Medicine
, vol.331
, Issue.9
, pp. 574-578
-
-
Olivieri, N.F.1
Nathan, D.G.2
Macmillan, J.H.3
Wayne, A.S.4
Liu, P.P.5
Mcgee, A.6
Martin, M.7
Koren, G.8
Cohen, A.R.9
-
54
-
-
37049033278
-
A randomized controlled 1-year study of daily deferiprone plus twice weekly desferoxamine compared with daily deferiprone monotherapy in patients with thalassemia major
-
DOI 10.3324/haematol.11414
-
Y Aydinok, Z Ulger, D Nart et al. A randomized controlled 1-year study of daily deferiprone plus twice weekly desferrioxamine compared with daily deferiprone monotherapy in patients with thalassemia major Haematologica 92 2007 1599 1606 (Pubitemid 350248238)
-
(2007)
Haematologica
, vol.92
, Issue.12
, pp. 1599-1606
-
-
Aydinok, Y.1
Ulger, Z.2
Nart, D.3
Terzi, A.4
Cetiner, N.5
Ellis, G.6
Zimmermann, A.7
Manz, C.8
-
55
-
-
33646414765
-
A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia
-
MD Cappellini, A Cohen, A Piga et al. A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia Blood 107 2006 3455 3462
-
(2006)
Blood
, vol.107
, pp. 3455-3462
-
-
Cappellini, M.D.1
Cohen, A.2
Piga, A.3
-
56
-
-
77950617863
-
Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia
-
DJ Pennell, JB Porter, MD Cappellini et al. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia Blood 115 2010 2364 2371
-
(2010)
Blood
, vol.115
, pp. 2364-2371
-
-
Pennell, D.J.1
Porter, J.B.2
Cappellini, M.D.3
-
57
-
-
77955967904
-
Deferasirox - Current knowledge and future challenges
-
JB Porter Deferasirox - current knowledge and future challenges Ann NY Acad Sci 1202 2010 87 93
-
(2010)
Ann NY Acad Sci
, vol.1202
, pp. 87-93
-
-
Porter, J.B.1
-
59
-
-
33750089012
-
Splenectomy: A strong risk factor for pulmonary hypertension in patients with thalassaemia
-
DOI 10.1136/hrt.2005.079970
-
A Phrommintikul, A Sukonthasarn, R Kanjanavanit, W Nawarawong Splenectomy: a strong risk factor for pulmonary hypertension in patients with thalassaemia Heart 92 2006 1467 1472 (Pubitemid 44578845)
-
(2006)
Heart
, vol.92
, Issue.10
, pp. 1467-1472
-
-
Phrommintikul, A.1
Sukonthasarn, A.2
Kanjanavanit, R.3
Nawarawong, W.4
-
60
-
-
33748102660
-
Pulmonary hypertension in thalassemia: Association with platelet activation and hypercoagulable state
-
DOI 10.1002/ajh.20640
-
ST Singer, FA Kuypers, L Styles, EP Vichinsky, D Foote, H Rosenfeld Pulmonary hypertension in thalassemia: association with platelet activation and hypercoagulable state Am J Hematol 81 2006 670 675 (Pubitemid 44306825)
-
(2006)
American Journal of Hematology
, vol.81
, Issue.9
, pp. 670-675
-
-
Singer, S.T.1
Kuypers, F.A.2
Styles, L.3
Vichinsky, E.P.4
Foote, D.5
Rosenfeld, H.6
-
61
-
-
78249266304
-
Noninvasive approaches to prenatal diagnosis of hemoglobinopathies using fetal DNA in maternal plasma
-
YM Lo, RW Chiu Noninvasive approaches to prenatal diagnosis of hemoglobinopathies using fetal DNA in maternal plasma Hematol Oncol Clin North Am 24 2002 1179 1186
-
(2002)
Hematol Oncol Clin North Am
, vol.24
, pp. 1179-1186
-
-
Lo, Y.M.1
Chiu, R.W.2
-
63
-
-
42049105635
-
Genetic modifiers of the β-haemoglobinopathies
-
SL Thein Genetic modifiers of the β-haemoglobinopathies Br J Haematol 141 2008 357 366
-
(2008)
Br J Haematol
, vol.141
, pp. 357-366
-
-
Thein, S.L.1
-
64
-
-
33745126299
-
Understanding mechanisms of γ-globin gene regulation to develop strategies for pharmacological fetal hemoglobin induction
-
DOI 10.1002/dvdy.20802
-
BS Pace, S Zein Understanding mechanisms of γ-globin gene regulation to develop strategies for pharmacological fetal hemoglobin induction Dev Dyn 235 2006 1727 1737 (Pubitemid 43902333)
-
(2006)
Developmental Dynamics
, vol.235
, Issue.7
, pp. 1727-1737
-
-
Pace, B.S.1
Zein, S.2
-
65
-
-
45349107618
-
Fetal globin stimulant therapies in the beta-hemoglobinopathies: Principles and current potential
-
SP Perrine Fetal globin stimulant therapies in the β- hemoglobinopathies: principles and current potential Pediatr Ann 37 2008 339 346 (Pubitemid 351847506)
-
(2008)
Pediatric Annals
, vol.37
, Issue.5
, pp. 339-346
-
-
Perrine, S.P.1
-
66
-
-
58449087328
-
Haemoglobin F modulation in childhood sickle cell disease
-
S Trompeter, I Roberts Haemoglobin F modulation in childhood sickle cell disease Br J Haematol 144 2009 308 316
-
(2009)
Br J Haematol
, vol.144
, pp. 308-316
-
-
Trompeter, S.1
Roberts, I.2
-
67
-
-
36649010161
-
Hematopoietic Cell Transplantation for Hemoglobinopathies
-
DOI 10.1016/j.cppeds.2007.10.002, PII S1538544207000879
-
L Krishnamurti, HF Bunn, AM Williams, J Tolar Hematopoietic cell transplantation for hemoglobinopathies Curr Probl Pediatr Adolesc Health Care 38 2008 6 18 (Pubitemid 350194044)
-
(2008)
Current Problems in Pediatric and Adolescent Health Care
, vol.38
, Issue.1
, pp. 6-18
-
-
Krishnamurti, L.1
Bunn, H.F.2
Williams, A.M.3
Tolar, J.4
-
68
-
-
0037275448
-
Bone marrow transplantation for β-thalassaemia major: The UK experience in two paediatric centres
-
DOI 10.1046/j.1365-2141.2003.04065.x
-
SE Lawson, IA Roberts, P Amrolia, I Dokal, R Szydlo, PJ Darbyshire Bone marrow transplantation for β-thalassaemia major: the UK experience in two paediatric centres Br J Haematol 120 2003 289 295 (Pubitemid 36194180)
-
(2003)
British Journal of Haematology
, vol.120
, Issue.2
, pp. 289-295
-
-
Lawson, S.E.1
Roberts, I.A.G.2
Amrolia, P.3
Dokal, I.4
Szydlo, R.5
Darbyshire, P.J.6
-
69
-
-
0025070260
-
Bone marrow transplantation in patients with thalassemia
-
G Lucarelli, M Galimberti, P Polchi et al. Bone marrow transplantation in patients with thalassemia N Engl J Med 322 1990 417 421 (Pubitemid 20059509)
-
(1990)
New England Journal of Medicine
, vol.322
, Issue.7
, pp. 417-421
-
-
Lucarelli, G.1
Galimberti, M.2
Polchi, P.3
Angelucci, E.4
Baronciani, D.5
Giardini, C.6
Politi, P.7
Durazzi, S.M.T.8
Muretto, P.9
Albertini, F.10
-
70
-
-
42049107587
-
Cord blood stem cell transplantation for haemoglobinopathies
-
DOI 10.1111/j.1365-2141.2008.07016.x
-
FO Pinto, I Roberts Cord blood stem cell transplantation for haemoglobinopathies Br J Haematol 141 2008 309 324 (Pubitemid 351521145)
-
(2008)
British Journal of Haematology
, vol.141
, Issue.3
, pp. 309-324
-
-
Pinto, F.O.1
Roberts, I.2
-
71
-
-
77951137730
-
Hematopoietic stem cell transplantation for hemoglobinopathies: Current practice and emerging trends
-
FJ Smiers, L Krishnamurti, G Lucarelli Hematopoietic stem cell transplantation for hemoglobinopathies: current practice and emerging trends Pediatr Clin North Am 57 2010 181 205
-
(2010)
Pediatr Clin North Am
, vol.57
, pp. 181-205
-
-
Smiers, F.J.1
Krishnamurti, L.2
Lucarelli, G.3
-
72
-
-
29744438595
-
Bone marrow transplantation in adults with thalassemia: Treatment and long-term follow-up
-
DOI 10.1196/annals.1345.024
-
J Gaziev, P Sodani, P Polchi, M Andreani, G Lucarelli Bone marrow transplantation in adults with thalassemia: treatment and long-term follow-up Ann N Y Acad Sci 1054 2005 196 205 (Pubitemid 43031025)
-
(2005)
Annals of the New York Academy of Sciences
, vol.1054
, pp. 196-205
-
-
Gaziev, J.1
Sodani, P.2
Polchi, P.3
Andreani, M.4
Lucarelli, G.5
-
73
-
-
0037097807
-
Unrelated donor bone marrow transplantation for thalassemia: The effect of extended haplotypes
-
DOI 10.1182/blood.V99.12.4350
-
G La Nasa, C Giardini, F Argiolu et al. Unrelated donor bone marrow transplantation for thalassemia: the effect of extended haplotypes Blood 99 2002 4350 4356 (Pubitemid 34627200)
-
(2002)
Blood
, vol.99
, Issue.12
, pp. 4350-4356
-
-
La Nasa, G.1
Giardini, C.2
Argiolu, F.3
Locatelli, F.4
Arras, M.5
De Stefano, P.6
Ledda, A.7
Pizzati, A.8
Sanna, M.A.9
Vacca, A.10
Lucarelli, G.11
Contu, L.12
-
74
-
-
28544436719
-
Unrelated donor stem cell transplantation in adult patients with thalassemia
-
DOI 10.1038/sj.bmt.1705173, PII 1705173
-
G La Nasa, G Caocci, F Argiolu et al. Unrelated donor stem cell transplantation in adult patients with thalassemia Bone Marrow Transplant 36 2005 971 975 (Pubitemid 41741560)
-
(2005)
Bone Marrow Transplantation
, vol.36
, Issue.11
, pp. 971-975
-
-
La Nasa, G.1
Caocci, G.2
Argiolu, F.3
Giardini, C.4
Locatelli, F.5
Vacca, A.6
Orofino, M.G.7
Piras, E.8
Addari, M.C.9
Ledda, A.10
Contu, L.11
-
76
-
-
0034036968
-
Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA-nonidentical sibling or parent transplants
-
D Gaziev, M Galimberti, G Lucarelli et al. Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA-nonidentical sibling or parent transplants Bone Marrow Transplant 25 2000 815 821 (Pubitemid 30209673)
-
(2000)
Bone Marrow Transplantation
, vol.25
, Issue.8
, pp. 815-821
-
-
Gaziev, D.1
Galimberti, M.2
Lucarelli, G.3
Polchi, P.4
Giardini, C.5
Angelucci, E.6
Baronciani, D.7
Sodani, P.8
Erer, B.9
De Biagi, M.10
Andreani, M.11
Agostinelli, F.12
Donati, M.13
Nesci, S.14
Talevi, N.15
-
77
-
-
39549112062
-
Advances in the allogeneic transplantation for thalassemia
-
DOI 10.1016/j.blre.2007.10.001, PII S0268960X07000677
-
G Lucarelli, J Gaziev Advances in the allogeneic transplantation for thalassemia Blood Rev 22 2008 53 63 (Pubitemid 351282443)
-
(2008)
Blood Reviews
, vol.22
, Issue.2
, pp. 53-63
-
-
Lucarelli, G.1
Gaziev, J.2
-
78
-
-
77953022728
-
Cord blood transplantation in patients with hemoglobinopathies
-
A Boncimino, A Bertaina, F Locatelli Cord blood transplantation in patients with hemoglobinopathies Transfus Apher Sci 42 2010 277 281
-
(2010)
Transfus Apher Sci
, vol.42
, pp. 277-281
-
-
Boncimino, A.1
Bertaina, A.2
Locatelli, F.3
-
79
-
-
2942627262
-
Unrelated umbilical cord blood transplantation for an infant with β-thalassemia major
-
DOI 10.1097/00043426-200406000-00010
-
JG Hall, PL Martin, S Wood, J Kurtzberg Unrelated umbilical cord blood transplantation for an infant with β-thalassemia major J Pediatr Hematol Oncol 26 2004 382 385 (Pubitemid 38747730)
-
(2004)
Journal of Pediatric Hematology/Oncology
, vol.26
, Issue.6
, pp. 382-385
-
-
Hall, J.G.1
Martin, P.L.2
Wood, S.3
Kurtzberg, J.4
-
80
-
-
17444402874
-
Rapid and complete donor chimerism after unrelated mismatched cord blood transplantation in 5 children with β-thalassemia major
-
DOI 10.1016/j.bbmt.2005.02.003, PII S1083879105001515
-
TH Jaing, IJ Hung, CP Yang, SH Chen, CF Sun, R Chow Rapid and complete donor chimerism after unrelated mismatched cord blood transplantation in 5 children with β-thalassemia major Biol Blood Marrow Transplant 11 2005 349 353 (Pubitemid 40542927)
-
(2005)
Biology of Blood and Marrow Transplantation
, vol.11
, Issue.5
, pp. 349-353
-
-
Jaing, T.-H.1
Hung, I.-J.2
Yang, C.-P.3
Chen, S.-H.4
Sun, C.-F.5
Chow, R.6
-
81
-
-
0015492624
-
Gene therapy for human genetic disease?
-
T Friedmann, R Roblin Gene therapy for human genetic disease? Science 175 1972 949 955
-
(1972)
Science
, vol.175
, pp. 949-955
-
-
Friedmann, T.1
Roblin, R.2
-
82
-
-
0142084745
-
LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
-
DOI 10.1126/science.1088547
-
S Hacein-Bey-Abina, C Von Kalle, M Schmidt et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 Science 302 2003 415 419 (Pubitemid 37296260)
-
(2003)
Science
, vol.302
, Issue.5644
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
McCormack, M.P.4
Wulffraat, N.5
Leboulch, P.6
Lim, A.7
Osborne, C.S.8
Pawliuk, R.9
Morillon, E.10
Sorensen, R.11
Forster, A.12
Fraser, P.13
Cohen, J.I.14
De Saint Basile, G.15
Alexander, I.16
Wintergerst, U.17
Frebourg, T.18
Aurias, A.19
Stoppa-Lyonnet, D.20
Romana, S.21
Radford-Weiss, I.22
Gross, F.23
Valensi, F.24
Delabesse, E.25
Macintyre, E.26
Sigaux, F.27
Soulier, J.28
Leiva, L.E.29
Wissler, M.30
Prinz, C.31
Rabbitts, T.H.32
Le Deist, F.33
Fischer, A.34
Cavazzana-Calvo, M.35
more..
-
83
-
-
77954833516
-
Efficacy of gene therapy for X-linked severe combined immunodeficiency
-
S Hacein-Bey-Abina, J Hauer, A Lim et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency N Engl J Med 363 2010 355 364
-
(2010)
N Engl J Med
, vol.363
, pp. 355-364
-
-
Hacein-Bey-Abina, S.1
Hauer, J.2
Lim, A.3
-
84
-
-
77950690992
-
Update on gene therapy for immunodeficiencies
-
DB Kohn Update on gene therapy for immunodeficiencies Clin Immunol 135 2010 247 254
-
(2010)
Clin Immunol
, vol.135
, pp. 247-254
-
-
Kohn, D.B.1
-
85
-
-
0037195091
-
Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells
-
DOI 10.1073/pnas.212507099
-
S Imren, E Payen, KA Westerman et al. Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells Proc Natl Acad Sci USA 99 2002 14380 14385 (Pubitemid 35257680)
-
(2002)
Proceedings of the National Academy of Sciences of the United States of America
, vol.99
, Issue.22
, pp. 14380-14385
-
-
Imren, S.1
Payen, E.2
Westerman, K.A.3
Pawliuk, R.4
Fabry, M.E.5
Eaves, C.J.6
Cavilla, B.7
Wadsworth, L.D.8
Beuzard, Y.9
Bouhassira, E.E.10
Russell, R.11
London, I.M.12
Nagel, R.L.13
Leboulch, P.14
Humphries, R.K.15
-
86
-
-
0343628721
-
Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
-
DOI 10.1038/35017565
-
C May, S Rivella, J Callegari et al. Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin Nature 406 2000 82 86 (Pubitemid 30460215)
-
(2000)
Nature
, vol.406
, Issue.6791
, pp. 82-86
-
-
Chad, M.1
Rivella, S.2
Callegari, J.3
Heller, G.4
Gaensler, K.M.L.5
Luzzatto, L.6
Sadelain, M.7
-
87
-
-
0037443467
-
The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number
-
DOI 10.1182/blood-2002-07-2211
-
DA Persons, PW Hargrove, ER Allay, H Hanawa, AW Nienhuis The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number Blood 101 2003 2175 2183 (Pubitemid 36302055)
-
(2003)
Blood
, vol.101
, Issue.6
, pp. 2175-2183
-
-
Persons, D.A.1
Hargrove, P.W.2
Allay, E.R.3
Hanawa, H.4
Nienhuis, A.W.5
-
88
-
-
0037085774
-
Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene
-
DOI 10.1182/blood.V99.6.1902
-
C May, S Rivella, A Chadburn, M Sadelain Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene Blood 99 2002 1902 1908 (Pubitemid 34525467)
-
(2002)
Blood
, vol.99
, Issue.6
, pp. 1902-1908
-
-
May, C.1
Rivella, S.2
Chadburn, A.3
Sadelain, M.4
-
89
-
-
0037606048
-
A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human β-globin gene transfer
-
DOI 10.1182/blood-2002-10-3305
-
S Rivella, C May, A Chadburn, I Rivire, M Sadelain A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human β-globin gene transfer Blood 101 2003 2932 2939 (Pubitemid 36857977)
-
(2003)
Blood
, vol.101
, Issue.8
, pp. 2932-2939
-
-
Rivella, S.1
May, C.2
Chadburn, A.3
Riviere, I.4
Sadelain, M.5
-
91
-
-
77956928344
-
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
-
M Cavazzana-Calvo, E Payen, O Negre et al. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia Nature 467 2010 318 322
-
(2010)
Nature
, vol.467
, pp. 318-322
-
-
Cavazzana-Calvo, M.1
Payen, E.2
Negre, O.3
-
92
-
-
72149123930
-
Gene therapy. β-thalassemia treatment succeeds, with a caveat
-
J Kaiser Gene therapy. β-thalassemia treatment succeeds, with a caveat Science 326 2010 1468 1469
-
(2010)
Science
, vol.326
, pp. 1468-1469
-
-
Kaiser, J.1
-
93
-
-
77949441974
-
Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders
-
DA Persons Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders Hematology Am Soc Hematol Educ Program 1 2009 690 697
-
(2009)
Hematology Am Soc Hematol Educ Program
, vol.1
, pp. 690-697
-
-
Persons, D.A.1
-
94
-
-
33846196263
-
Therapeutic options for patients with severe β-thalassemia: The need for globin gene therapy
-
DOI 10.1089/hum.2006.151
-
M Sadelain, F Boulad, R Galanello et al. Therapeutic options for patients with severe β-thalassemia: the need for globin gene therapy Hum Gene Ther 18 2007 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
-
95
-
-
33747195353
-
Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors
-
DOI 10.1016/j.cell.2006.07.024, PII S0092867406009767
-
K Takahashi, S Yamanaka Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors Cell 126 2006 663 676 (Pubitemid 44233629)
-
(2006)
Cell
, vol.126
, Issue.4
, pp. 663-676
-
-
Takahashi, K.1
Yamanaka, S.2
-
96
-
-
36749043230
-
Induced pluripotent stem cell lines derived from human somatic cells
-
J Yu, MA Vodyanik, K Smuga-Otto et al. Induced pluripotent stem cell lines derived from human somatic cells Science 318 2007 1917 1920
-
(2007)
Science
, vol.318
, pp. 1917-1920
-
-
Yu, J.1
Vodyanik, M.A.2
Smuga-Otto, K.3
-
97
-
-
37549030199
-
Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin
-
J Hanna, M Wernig, S Markoulaki et al. Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin Science 318 2007 1920 1923
-
(2007)
Science
, vol.318
, pp. 1920-1923
-
-
Hanna, J.1
Wernig, M.2
Markoulaki, S.3
-
98
-
-
40049084511
-
A new dawn for stem-cell therapy
-
DR Higgs A new dawn for stem-cell therapy N Engl J Med 358 2008 964 966
-
(2008)
N Engl J Med
, vol.358
, pp. 964-966
-
-
Higgs, D.R.1
-
99
-
-
67649880603
-
Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases
-
L Ye, JC Chang, C Lin, X Sun, J Yu, YW Kan Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases Proc Natl Acad Sci USA 106 2009 9826 9830
-
(2009)
Proc Natl Acad Sci USA
, vol.106
, pp. 9826-9830
-
-
Ye, L.1
Chang, J.C.2
Lin, C.3
Sun, X.4
Yu, J.5
Kan, Y.W.6
-
100
-
-
29744461542
-
Stem cell transplantation and gene therapy for hemoglobinopathies
-
J Gaziev, G Lucarelli Stem cell transplantation and gene therapy for hemoglobinopathies Curr Hematol Rep 4 2005 126 131
-
(2005)
Curr Hematol Rep
, vol.4
, pp. 126-131
-
-
Gaziev, J.1
Lucarelli, G.2
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