Detalhe da pesquisa
1.
Exagamglogene Autotemcel for Transfusion-Dependent ß-Thalassemia.
N Engl J Med
; 390(18): 1663-1676, 2024 May 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-38657265
2.
Exagamglogene Autotemcel for Severe Sickle Cell Disease.
N Engl J Med
; 390(18): 1649-1662, 2024 May 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-38661449
3.
Autologous gene therapy for hemoglobinopathies: From bench to patient's bedside.
Mol Ther
; 32(5): 1202-1218, 2024 May 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-38454604
4.
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and ß-Thalassemia.
N Engl J Med
; 384(3): 252-260, 2021 01 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-33283989
5.
Defining curative endpoints for sickle cell disease in the era of gene therapy and gene editing.
Am J Hematol
; 99(3): 430-438, 2024 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-38010293
6.
Defining curative endpoints for transfusion-dependent ß-thalassemia in the era of gene therapy and gene editing.
Am J Hematol
; 99(3): 422-429, 2024 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-38100154
7.
Neighborhood poverty and pediatric allogeneic hematopoietic cell transplantation outcomes: a CIBMTR analysis.
Blood
; 137(4): 556-568, 2021 01 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-33104215
8.
An integrated therapeutic approach to sickle cell disease management beyond infancy.
Am J Hematol
; 98(7): 1087-1096, 2023 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-37170801
9.
Molecular and phenotypic diversity of CBL-mutated juvenile myelomonocytic leukemia.
Haematologica
; 107(1): 178-186, 2022 01 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33375775
10.
Safety, Tolerability, and Population Pharmacokinetics of Intravenous and Oral Isavuconazonium Sulfate in Pediatric Patients.
Antimicrob Agents Chemother
; 65(8): e0029021, 2021 07 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-34031051
11.
HLA-Haploidentical Hematopoietic Cell Transplantation for Treatment of Nonmalignant Diseases Using Nonmyeloablative Conditioning and Post-Transplant Cyclophosphamide.
Biol Blood Marrow Transplant
; 26(7): 1332-1341, 2020 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-32234377
12.
The Impact of Donor Type on Outcomes and Cost of Allogeneic Hematopoietic Cell Transplantation for Pediatric Leukemia: A Merged Center for International Blood and Marrow Transplant Research and Pediatric Health Information System Analysis.
Biol Blood Marrow Transplant
; 26(9): 1747-1756, 2020 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32464284
13.
Infantile Myelofibrosis and Myeloproliferation with CDC42 Dysfunction.
J Clin Immunol
; 40(4): 554-566, 2020 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-32303876
14.
Impact of cytogenetic abnormalities on outcomes of adult Philadelphia-negative acute lymphoblastic leukemia after allogeneic hematopoietic stem cell transplantation: a study by the Acute Leukemia Working Committee of the Center for International Blood and Marrow Transplant Research.
Haematologica
; 105(5): 1329-1338, 2020 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-31558669
15.
The Concentration of Total Nucleated Cells in Harvested Bone Marrow for Transplantation Has Decreased over Time.
Biol Blood Marrow Transplant
; 25(7): 1325-1330, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30716454
16.
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and ß-Thalassemia. Reply.
N Engl J Med
; 384(23): e91, 2021 06 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34107197
17.
Influence of Age on Acute and Chronic GVHD in Children Undergoing HLA-Identical Sibling Bone Marrow Transplantation for Acute Leukemia: Implications for Prophylaxis.
Biol Blood Marrow Transplant
; 24(3): 521-528, 2018 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-29155316
18.
Phase I Study of Clofarabine and 2-Gy Total Body Irradiation as a Nonmyeloablative Preparative Regimen for Hematopoietic Stem Cell Transplantation in Pediatric Patients with Hematologic Malignancies: A Therapeutic Advances in Childhood Leukemia Consortium Study.
Biol Blood Marrow Transplant
; 23(7): 1134-1141, 2017 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-28396162
19.
Allogeneic Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for Treatment of Marrow Failure Disorders.
Biol Blood Marrow Transplant
; 23(10): 1669-1677, 2017 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-28602958
20.
Impact of cytogenetic abnormalities on outcomes of adult Philadelphia-negative acute lymphoblastic leukemia after allogeneic hematopoietic stem cell transplantation: a study by the Acute Leukemia Working Committee of the Center for International Blood and Marrow Transplant Research.
Haematologica
; 106(8): 2295-2296, 2021 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34333962