-
1
-
-
43449084027
-
The classification of inherited epidermolysis bullosa (EB): report of the Third International Consensus Meeting on Diagnosis and Classification of EB
-
Fine J.D., Eady R.A., Bauer E.A., et al. The classification of inherited epidermolysis bullosa (EB): report of the Third International Consensus Meeting on Diagnosis and Classification of EB. J Am Acad Dermatol 2008, 58:931-950.
-
(2008)
J Am Acad Dermatol
, vol.58
, pp. 931-950
-
-
Fine, J.D.1
Eady, R.A.2
Bauer, E.A.3
-
2
-
-
70450267632
-
Dystrophic epidermolysis bullosa: pathogenesis and clinical features
-
Bruckner-Tuderman L. Dystrophic epidermolysis bullosa: pathogenesis and clinical features. Dermatol Clin 2010, 28:107-114.
-
(2010)
Dermatol Clin
, vol.28
, pp. 107-114
-
-
Bruckner-Tuderman, L.1
-
3
-
-
0032717715
-
Targeted inactivation of the type VII collagen gene (Col7a1) in mice results in severe blistering phenotype: a model for recessive dystrophic epidermolysis bullosa
-
Heinonen S., Mannikko M., Klement J.F., Whitaker-Menezes D., Murphy G.F., Uitto J. Targeted inactivation of the type VII collagen gene (Col7a1) in mice results in severe blistering phenotype: a model for recessive dystrophic epidermolysis bullosa. JCell Sci 1999, 112(Pt 21):3641-3648.
-
(1999)
JCell Sci
, vol.112
, Issue.PT 21
, pp. 3641-3648
-
-
Heinonen, S.1
Mannikko, M.2
Klement, J.F.3
Whitaker-Menezes, D.4
Murphy, G.F.5
Uitto, J.6
-
4
-
-
60849091919
-
Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells
-
Tolar J., Ishida-Yamamoto A., Riddle M., et al. Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells. Blood 2009, 113:1167-1174.
-
(2009)
Blood
, vol.113
, pp. 1167-1174
-
-
Tolar, J.1
Ishida-Yamamoto, A.2
Riddle, M.3
-
5
-
-
51349152151
-
Bone marrow cell transfer into fetal circulation can ameliorate genetic skin diseases by providing fibroblasts to the skin and inducing immune tolerance
-
Chino T., Tamai K., Yamazaki T., et al. Bone marrow cell transfer into fetal circulation can ameliorate genetic skin diseases by providing fibroblasts to the skin and inducing immune tolerance. Am J Pathol 2008, 173:803-814.
-
(2008)
Am J Pathol
, vol.173
, pp. 803-814
-
-
Chino, T.1
Tamai, K.2
Yamazaki, T.3
-
6
-
-
77956293326
-
Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice
-
Fujita Y., Abe R., Inokuma D., et al. Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice. Proc Natl Acad Sci USA 2010, 107:14345-14350.
-
(2010)
Proc Natl Acad Sci USA
, vol.107
, pp. 14345-14350
-
-
Fujita, Y.1
Abe, R.2
Inokuma, D.3
-
7
-
-
77956621310
-
Bone marrow transplantation for recessive dystrophic epidermolysis bullosa
-
Wagner J.E., Ishida-Yamamoto A., McGrath J.A., et al. Bone marrow transplantation for recessive dystrophic epidermolysis bullosa. N Engl J Med 2010, 363:629-639.
-
(2010)
N Engl J Med
, vol.363
, pp. 629-639
-
-
Wagner, J.E.1
Ishida-Yamamoto, A.2
McGrath, J.A.3
-
8
-
-
79957595404
-
Concise review: transplantation of human hematopoietic cells for extracellular matrix protein deficiency in epidermolysis bullosa
-
Tolar J., Blazar B.R., Wagner J.E. Concise review: transplantation of human hematopoietic cells for extracellular matrix protein deficiency in epidermolysis bullosa. Stem Cells 2011, 29:900-906.
-
(2011)
Stem Cells
, vol.29
, pp. 900-906
-
-
Tolar, J.1
Blazar, B.R.2
Wagner, J.E.3
-
9
-
-
78649656327
-
Stem cell therapies for recessive dystrophic epidermolysis bullosa
-
Petrova A., Ilic D., McGrath J.A. Stem cell therapies for recessive dystrophic epidermolysis bullosa. Br J Dermatol 2010, 163:1149-1156.
-
(2010)
Br J Dermatol
, vol.163
, pp. 1149-1156
-
-
Petrova, A.1
Ilic, D.2
McGrath, J.A.3
-
10
-
-
79952765436
-
Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa
-
Tolar J., Xia L., Riddle M.J., et al. Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa. J Invest Dermatol 2011, 131:848-856.
-
(2011)
J Invest Dermatol
, vol.131
, pp. 848-856
-
-
Tolar, J.1
Xia, L.2
Riddle, M.J.3
-
11
-
-
0017118656
-
Susceptibility of Fanconi's anaemia fibroblasts to chromosome damage by carcinogens
-
Auerbach A.D., Wolman S.R. Susceptibility of Fanconi's anaemia fibroblasts to chromosome damage by carcinogens. Nature 1976, 261:494-496.
-
(1976)
Nature
, vol.261
, pp. 494-496
-
-
Auerbach, A.D.1
Wolman, S.R.2
-
12
-
-
0027521988
-
Bone marrow transplantation in Fanconi's anemia
-
Gluckman E. Bone marrow transplantation in Fanconi's anemia. Stem Cells 1993, 11(suppl 2):180-183.
-
(1993)
Stem Cells
, vol.11
, Issue.SUPPL. 2
, pp. 180-183
-
-
Gluckman, E.1
-
13
-
-
33846416168
-
Matched sibling donor haematopoietic stem cell transplantation in Fanconi anaemia: an update of the Cincinnati Children's experience
-
Farzin A., Davies S.M., Smith F.O., et al. Matched sibling donor haematopoietic stem cell transplantation in Fanconi anaemia: an update of the Cincinnati Children's experience. Br J Haematol 2007, 136:633-640.
-
(2007)
Br J Haematol
, vol.136
, pp. 633-640
-
-
Farzin, A.1
Davies, S.M.2
Smith, F.O.3
-
14
-
-
0031436199
-
A fludarabine-based protocol for bone marrow transplantation in Fanconi's anemia
-
Kapelushnik J., Or R., Slavin S., Nagler A. A fludarabine-based protocol for bone marrow transplantation in Fanconi's anemia. Bone Marrow Transplant 1997, 20:1109-1110.
-
(1997)
Bone Marrow Transplant
, vol.20
, pp. 1109-1110
-
-
Kapelushnik, J.1
Or, R.2
Slavin, S.3
Nagler, A.4
-
15
-
-
33645315485
-
Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia patients undergoing genotypically identical donor hematopoietic cell transplantation
-
Tan P.L., Wagner J.E., Auerbach A.D., Defor T.E., Slungaard A., Macmillan M.L. Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia patients undergoing genotypically identical donor hematopoietic cell transplantation. Pediatr Blood Cancer 2006, 46:630-636.
-
(2006)
Pediatr Blood Cancer
, vol.46
, pp. 630-636
-
-
Tan, P.L.1
Wagner, J.E.2
Auerbach, A.D.3
Defor, T.E.4
Slungaard, A.5
Macmillan, M.L.6
-
16
-
-
0242609134
-
Low risk of graft-versus-host disease with transplantation of CD34 selected peripheral blood progenitor cells from alternative donors for Fanconi anemia
-
Boyer M.W., Gross T.G., Loechelt B., Leemhuis T., Filipovich A., Harris R.E. Low risk of graft-versus-host disease with transplantation of CD34 selected peripheral blood progenitor cells from alternative donors for Fanconi anemia. J Pediatr Hematol Oncol 2003, 25:890-895.
-
(2003)
J Pediatr Hematol Oncol
, vol.25
, pp. 890-895
-
-
Boyer, M.W.1
Gross, T.G.2
Loechelt, B.3
Leemhuis, T.4
Filipovich, A.5
Harris, R.E.6
-
17
-
-
39749121663
-
Fludarabine-based cytoreductive regimen and T-cell-depleted grafts from alternative donors for the treatment of high-risk patients with Fanconi anaemia
-
Chaudhury S., Auerbach A.D., Kernan N.A., et al. Fludarabine-based cytoreductive regimen and T-cell-depleted grafts from alternative donors for the treatment of high-risk patients with Fanconi anaemia. Br J Haematol 2008, 140:644-655.
-
(2008)
Br J Haematol
, vol.140
, pp. 644-655
-
-
Chaudhury, S.1
Auerbach, A.D.2
Kernan, N.A.3
-
18
-
-
32944459632
-
Haematopoietic stem cell transplantation for Shwachman-Diamond disease: a study from the European Group for blood and marrow transplantation
-
Cesaro S., Oneto R., Messina C., et al. Haematopoietic stem cell transplantation for Shwachman-Diamond disease: a study from the European Group for blood and marrow transplantation. Br J Haematol 2005, 131:231-236.
-
(2005)
Br J Haematol
, vol.131
, pp. 231-236
-
-
Cesaro, S.1
Oneto, R.2
Messina, C.3
-
19
-
-
50049114380
-
Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman-Diamond syndrome
-
Bhatla D., Davies S.M., Shenoy S., et al. Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman-Diamond syndrome. Bone Marrow Transplant 2008, 42:159-165.
-
(2008)
Bone Marrow Transplant
, vol.42
, pp. 159-165
-
-
Bhatla, D.1
Davies, S.M.2
Shenoy, S.3
-
20
-
-
0029967609
-
Allogeneic marrow transplantation for aplastic anaemia associated with dyskeratosis congenita
-
Langston A.A., Sanders J.E., Deeg H.J., et al. Allogeneic marrow transplantation for aplastic anaemia associated with dyskeratosis congenita. Br J Haematol 1996, 92:758-765.
-
(1996)
Br J Haematol
, vol.92
, pp. 758-765
-
-
Langston, A.A.1
Sanders, J.E.2
Deeg, H.J.3
-
21
-
-
78651373073
-
Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita
-
Dietz A.C., Orchard P.J., Baker K.S., Giller R.H., Savage S.A., Alter B.P., et al. Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita. Bone Marrow Transplant 2011, 46:98-104.
-
(2011)
Bone Marrow Transplant
, vol.46
, pp. 98-104
-
-
Dietz, A.C.1
Orchard, P.J.2
Baker, K.S.3
Giller, R.H.4
Savage, S.A.5
Alter, B.P.6
-
22
-
-
20844460608
-
Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine
-
Borgna-Pignatti C., Rugolotto S., De Stefano 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 Stefano, P.3
-
23
-
-
77951712618
-
Improved survival of children and adolescents with sickle cell disease
-
Quinn C.T., Rogers Z.R., McCavit T.L., Buchanan G.R. Improved survival of children and adolescents with sickle cell disease. Blood 2010, 115:3447-3452.
-
(2010)
Blood
, vol.115
, pp. 3447-3452
-
-
Quinn, C.T.1
Rogers, Z.R.2
McCavit, T.L.3
Buchanan, G.R.4
-
24
-
-
0034779987
-
Allogeneic stem cell transplantation for sickle cell disease. A study of patients' decisions
-
van Besien K., Koshy M., Anderson-Shaw L., et al. Allogeneic stem cell transplantation for sickle cell disease. A study of patients' decisions. Bone Marrow Transplant 2001, 28:545-549.
-
(2001)
Bone Marrow Transplant
, vol.28
, pp. 545-549
-
-
van Besien, K.1
Koshy, M.2
Anderson-Shaw, L.3
-
25
-
-
27344441977
-
Health related quality of life in sickle cell patients: the PiSCES project
-
McClish D.K., Penberthy L.T., Bovbjerg V.E., et al. Health related quality of life in sickle cell patients: the PiSCES project. Health Qual Life Outcomes 2005, 3:50.
-
(2005)
Health Qual Life Outcomes
, vol.3
, pp. 50
-
-
McClish, D.K.1
Penberthy, L.T.2
Bovbjerg, V.E.3
-
26
-
-
59249104210
-
Recent advances in bone marrow transplantation in hemoglobinopathies
-
Michlitsch J.G., Walters M.C. Recent advances in bone marrow transplantation in hemoglobinopathies. Curr Mol Med 2008, 8:675-689.
-
(2008)
Curr Mol Med
, vol.8
, pp. 675-689
-
-
Michlitsch, J.G.1
Walters, M.C.2
-
27
-
-
29744438595
-
Bone marrow transplantation in adults with thalassemia: treatment and long-term follow-up
-
Gaziev J., Sodani P., Polchi P., Andreani M., Lucarelli G. Bone marrow transplantation in adults with thalassemia: treatment and long-term follow-up. Ann N Y Acad Sci 2005, 1054:196-205.
-
(2005)
Ann N Y Acad Sci
, vol.1054
, pp. 196-205
-
-
Gaziev, J.1
Sodani, P.2
Polchi, P.3
Andreani, M.4
Lucarelli, G.5
-
28
-
-
19244366141
-
New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years
-
Sodani P., Gaziev D., Polchi P., et al. New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years. Blood 2004, 104:1201-1203.
-
(2004)
Blood
, vol.104
, pp. 1201-1203
-
-
Sodani, P.1
Gaziev, D.2
Polchi, P.3
-
29
-
-
35348884167
-
Status of donor-recipient HLA class I ligands and not the KIR genotype is predictive for the outcome of unrelated hematopoietic stem cell transplantation in beta-thalassemia patients
-
La Nasa G., Littera R., Locatelli F., et al. Status of donor-recipient HLA class I ligands and not the KIR genotype is predictive for the outcome of unrelated hematopoietic stem cell transplantation in beta-thalassemia patients. Biol Blood Marrow Transplant 2007, 13:1358-1368.
-
(2007)
Biol Blood Marrow Transplant
, vol.13
, pp. 1358-1368
-
-
La Nasa, G.1
Littera, R.2
Locatelli, F.3
-
30
-
-
54849419695
-
Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major
-
Bernardo M.E., Zecca M., Piras E., et al. Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major. Br J Haematol 2008, 143:548-551.
-
(2008)
Br J Haematol
, vol.143
, pp. 548-551
-
-
Bernardo, M.E.1
Zecca, M.2
Piras, E.3
-
31
-
-
12144289379
-
Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia
-
Iannone R., Casella J.F., Fuchs E.J., 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
-
32
-
-
71849118976
-
Allogeneic hematopoietic stem-cell transplantation for sickle cell disease
-
Hsieh M.M., Kang E.M., Fitzhugh C.D., et al. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med 2009, 361:2309-2317.
-
(2009)
N Engl J Med
, vol.361
, pp. 2309-2317
-
-
Hsieh, M.M.1
Kang, E.M.2
Fitzhugh, C.D.3
-
33
-
-
0037443543
-
Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease
-
Locatelli F., Rocha V., Reed W., et al. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood 2003, 101:2137-2143.
-
(2003)
Blood
, vol.101
, pp. 2137-2143
-
-
Locatelli, F.1
Rocha, V.2
Reed, W.3
-
34
-
-
80051666676
-
Umbilical cord blood transplantation for children with thalassemia and sickle cell disease
-
Ruggeri A., Eapen M., Scaravadou A., et al. Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant 2011, 17:1375-1382.
-
(2011)
Biol Blood Marrow Transplant
, vol.17
, pp. 1375-1382
-
-
Ruggeri, A.1
Eapen, M.2
Scaravadou, A.3
-
35
-
-
77949532641
-
Purified T-depleted, CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia
-
Sodani P., Isgro A., Gaziev J., et al. Purified T-depleted, CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia. Blood 2010, 115:1296-1302.
-
(2010)
Blood
, vol.115
, pp. 1296-1302
-
-
Sodani, P.1
Isgro, A.2
Gaziev, J.3
-
36
-
-
55549090680
-
Reduced intensity HLA-haploidentical BMT with post transplantation cyclophosphamide in nonmalignant hematologic diseases
-
Brodsky R.A., Luznik L., Bolanos-Meade J., Leffell M.S., Jones R.J., Fuchs E.J. Reduced intensity HLA-haploidentical BMT with post transplantation cyclophosphamide in nonmalignant hematologic diseases. Bone Marrow Transplant 2008, 42:523-527.
-
(2008)
Bone Marrow Transplant
, vol.42
, pp. 523-527
-
-
Brodsky, R.A.1
Luznik, L.2
Bolanos-Meade, J.3
Leffell, M.S.4
Jones, R.J.5
Fuchs, E.J.6
|