Detalhe da pesquisa
1.
Sickle Cell Disease: From Genetics to Curative Approaches.
Annu Rev Genomics Hum Genet;
24: 255-275, 2023 08 25.
Artigo
em Inglês
| MEDLINE
| ID: mdl-37624668
2.
Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs.
Blood;
135(15): 1219-1231, 2020 04 09.
Artigo
em Inglês
| MEDLINE
| ID: mdl-32040546
3.
A new step in understanding stem cell mobilization in patients with Fanconi anemia: A bridge to gene therapy.
Transfusion;
62(1): 165-172, 2022 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-34751952
4.
Gene therapy of hemoglobinopathies: progress and future challenges.
Hum Mol Genet;
28(R1): R24-R30, 2019 10 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31322165
5.
Gene Therapy in Patients with Transfusion-Dependent ß-Thalassemia.
N Engl J Med;
378(16): 1479-1493, 2018 04 19.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29669226
6.
Lentiviral and genome-editing strategies for the treatment of ß-hemoglobinopathies.
Blood;
134(15): 1203-1213, 2019 10 10.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31467062
7.
Vascular access for optimal hematopoietic stem cell collection.
J Clin Apher;
36(1): 12-19, 2021 Feb.
Artigo
em Inglês
| MEDLINE
| ID: mdl-32854142
8.
Bone Marrow Transplantation in Congenital Erythropoietic Porphyria: Sustained Efficacy but Unexpected Liver Dysfunction.
Biol Blood Marrow Transplant;
26(4): 704-711, 2020 04.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31843562
9.
Gene Therapy in a Patient with Sickle Cell Disease.
N Engl J Med;
376(9): 848-855, 2017 03 02.
Artigo
em Inglês
| MEDLINE
| ID: mdl-28249145
10.
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human ß-globin locus.
Blood;
131(17): 1960-1973, 2018 04 26.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29519807
11.
Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapy.
Haematologica;
105(5): 1240-1247, 2020 05.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31537695
12.
Pre- and post-transplant minimal residual disease predicts relapse occurrence in children with acute lymphoblastic leukaemia.
Br J Haematol;
180(5): 680-693, 2018 03.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29359790
13.
Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.
Haematologica;
103(5): 778-786, 2018 05.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29472357
14.
A Case of T-cell Acute Lymphoblastic Leukemia Relapsed As Myeloid Acute Leukemia.
Pediatr Blood Cancer;
63(9): 1660-3, 2016 09.
Artigo
em Inglês
| MEDLINE
| ID: mdl-27149388
15.
The tyrosine phosphatase SHP-1 inhibits proliferation of activated hepatic stellate cells by impairing PDGF receptor signaling.
Biochim Biophys Acta;
1843(2): 288-98, 2014 Feb.
Artigo
em Inglês
| MEDLINE
| ID: mdl-24140598
16.
Generation of adult human T-cell progenitors for immunotherapeutic applications.
J Allergy Clin Immunol;
141(4): 1491-1494.e4, 2018 04.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29208547
17.
Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy.
Cell Rep Med;
4(2): 100919, 2023 02 21.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36706754
18.
Src tyrosine kinase preactivation is associated with platelet hypersensitivity in essential thrombocythemia and polycythemia vera.
Blood;
115(3): 667-76, 2010 Jan 21.
Artigo
em Inglês
| MEDLINE
| ID: mdl-19965650
19.
Interaction between the SH3 domain of Src family kinases and the proline-rich motif of HTLV-1 p13: a novel mechanism underlying delivery of Src family kinases to mitochondria.
Biochem J;
439(3): 505-16, 2011 Nov 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-21732913
20.
Long-term outcomes of lentiviral gene therapy for the ß-hemoglobinopathies: the HGB-205 trial.
Nat Med;
28(1): 81-88, 2022 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-35075288