Detalhe da pesquisa
1.
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.
N Engl J Med
; 384(3): 205-215, 2021 01 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-33283990
2.
Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice.
Blood
; 119(2): 602-11, 2012 Jan 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-22031860
3.
Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells.
bioRxiv
; 2023 May 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-37292647
4.
Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy.
Mol Ther Methods Clin Dev
; 17: 589-600, 2020 Jun 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-32300607
5.
Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.
Hum Gene Ther Clin Dev
; 29(2): 69-79, 2018 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-29664709
6.
Chemical chaperones improve protein secretion and rescue mutant factor VIII in mice with hemophilia A.
PLoS One
; 7(9): e44505, 2012.
Artigo
em Inglês
| MEDLINE | ID: mdl-22973456
7.
Transgene loss and changes in the promoter methylation status as determinants for expression duration in nonviral gene transfer for factor IX.
Hum Gene Ther
; 22(1): 101-6, 2011 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-20677911
8.
Factor VIII-eGFP fusion proteins with preserved functional activity for the analysis of the early secretory pathway of factor VIII.
Thromb Haemost
; 102(5): 925-35, 2009 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-19888531
9.
Oxygen-induced changes in hemoglobin expression in Drosophila.
FEBS J
; 275(20): 5108-16, 2008 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-18795948