Detalhe da pesquisa
1.
Base editing of key residues in the BCL11A-XL-specific zinc finger domains derepresses fetal globin expression.
Mol Ther
; 32(3): 663-677, 2024 Mar 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-38273654
2.
Treosulfan Exposure Predicts Thalassemia-Free Survival in Patients with Beta Thalassemia Major Undergoing Allogeneic Hematopoietic Cell Transplantation.
Clin Pharmacol Ther
; 115(1): 116-125, 2024 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37846495
3.
Biomarkers for early complications post hematopoietic cell transplantation: Insights and challenges.
Front Immunol
; 14: 1100306, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-36817455
4.
Metformin pretreatment ameliorates busulfan-induced liver endothelial toxicity during haematopoietic stem cell transplantation.
PLoS One
; 18(10): e0293311, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37883349
5.
Modulating retinoid-X-receptor alpha (RXRA) expression sensitizes chronic myeloid leukemia cells to imatinib in vitro and reduces disease burden in vivo.
Front Pharmacol
; 14: 1187066, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37324449
6.
Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of ß-hemoglobinopathies.
Mol Ther Nucleic Acids
; 32: 671-688, 2023 Jun 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-37215154
7.
Erythroid lineage-specific lentiviral RNAi vectors suitable for molecular functional studies and therapeutic applications.
Sci Rep
; 12(1): 14033, 2022 08 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-35982069
8.
Endothelial Activation and Stress Index-Measured Pretransplantation Predicts Transplantation-Related Mortality in Patients with Thalassemia Major Undergoing Transplantation with Thiotepa, Treosulfan, and Fludarabine Conditioning.
Transplant Cell Ther
; 28(7): 356.e1-356.e6, 2022 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-35550442
9.
Efficient and error-free correction of sickle mutation in human erythroid cells using prime editor-2.
Front Genome Ed
; 4: 1085111, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-36605051
10.
Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin.
Elife
; 112022 02 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-35147495
11.
Direct Generation of Immortalized Erythroid Progenitor Cell Lines from Peripheral Blood Mononuclear Cells.
Cells
; 10(3)2021 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33804564
12.
NUDT15 c.415C>T Polymorphism Predicts 6-MP Induced Early Myelotoxicity in Patients with Acute Lymphoblastic Leukemia Undergoing Maintenance Therapy.
Pharmgenomics Pers Med
; 14: 1303-1313, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34629890
13.
Pharmacokinetics and Efficacy of Generic Melphalan Is Comparable to Innovator Formulation in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplantation.
Clin Lymphoma Myeloma Leuk
; 20(2): 130-135.e1, 2020 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-31791694
14.
Prognostic plasma biomarkers of early complications and graft-versus-host disease in patients undergoing allogeneic hematopoietic stem cell transplantation.
EJHaem
; 1(1): 219-229, 2020 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-32885223