Detalhe da pesquisa
1.
NRASQ61R mutation drives elevated angiopoietin-2 expression in human endothelial cells and a genetic mouse model.
Pediatr Blood Cancer
; : e31032, 2024 May 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-38711167
2.
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI).
Gene Ther
; 30(3-4): 216-221, 2023 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-34493840
3.
NRASQ61R mutation in human endothelial cells causes vascular malformations.
Angiogenesis
; 25(3): 331-342, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35391614
4.
Early initiation of hydroxyurea (hydroxycarbamide) using individualised, pharmacokinetics-guided dosing can produce sustained and nearly pancellular expression of fetal haemoglobin in children with sickle cell anaemia.
Br J Haematol
; 194(3): 617-625, 2021 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-34227124
5.
Implementation of near-universal hydroxyurea uptake among children with sickle cell anemia: A single-center experience.
Pediatr Blood Cancer
; 68(6): e29008, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33742510
6.
Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis.
Haematologica
; 105(4): 1147-1157, 2020 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-31289207
7.
Pulmonary macrophage transplantation therapy.
Nature
; 514(7523): 450-4, 2014 Oct 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-25274301
8.
G9a/GLP-dependent histone H3K9me2 patterning during human hematopoietic stem cell lineage commitment.
Genes Dev
; 26(22): 2499-511, 2012 Nov 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-23105005
9.
Foamy Virus Vector Carries a Strong Insulator in Its Long Terminal Repeat Which Reduces Its Genotoxic Potential.
J Virol
; 92(1)2018 01 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29046446
10.
Association between diffuse myocardial fibrosis and diastolic dysfunction in sickle cell anemia.
Blood
; 130(2): 205-213, 2017 07 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-28507082
11.
Early initiation of disease-modifying therapy can impede or prevent diffuse myocardial fibrosis in sickle cell anemia.
Blood
; 140(11): 1322-1324, 2022 09 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-35857896
12.
Robust clinical and laboratory response to hydroxyurea using pharmacokinetically guided dosing for young children with sickle cell anemia.
Am J Hematol
; 94(8): 871-879, 2019 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-31106898
13.
Abnormal submaximal cardiopulmonary exercise parameters predict impaired peak exercise performance in sickle cell anemia patients.
Pediatr Blood Cancer
; 66(6): e27703, 2019 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-30848046
14.
Sickle cell anemia mice develop a unique cardiomyopathy with restrictive physiology.
Proc Natl Acad Sci U S A
; 113(35): E5182-91, 2016 08 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-27503873
15.
Correction: Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI).
Gene Ther
; 30(3-4): 405, 2023 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-34584245
16.
Diastolic dysfunction is associated with exercise impairment in patients with sickle cell anemia.
Pediatr Blood Cancer
; 65(8): e27113, 2018 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-29781568
17.
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
N Engl J Med
; 371(15): 1407-17, 2014 Oct 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-25295500
18.
Genetic diminution of circulating prothrombin ameliorates multiorgan pathologies in sickle cell disease mice.
Blood
; 126(15): 1844-55, 2015 Oct 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-26286849
19.
Losartan for the nephropathy of sickle cell anemia: A phase-2, multicenter trial.
Am J Hematol
; 92(9): E520-E528, 2017 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-28589652
20.
A reappraisal of the mechanisms underlying the cardiac complications of sickle cell anemia.
Pediatr Blood Cancer
; 64(11)2017 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-28453224