Detalhe da pesquisa
1.
Reactivation of developmentally silenced globin genes by forced chromatin looping.
Cell
; 158(4): 849-860, 2014 Aug 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-25126789
2.
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study.
Blood
; 143(9): 796-806, 2024 Feb 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-37871576
3.
Outcomes of a clinical pathway for primary outpatient management of pediatric patients with low-risk febrile neutropenia.
Pediatr Blood Cancer
; 66(7): e27679, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30916887
4.
Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers.
Blood
; 128(8): 1139-43, 2016 08 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-27405777
5.
Impact of treatment regimen with moroctocog alfa (AF-CC) on bleeding rates in paediatric patients with severe haemophilia A.
Haemophilia
; 26(5): e232-e235, 2020 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-32497363
6.
Nonacog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Patients with Moderately Severe or Severe Hemophilia B in India.
Indian J Hematol Blood Transfus
; 39(4): 630-634, 2023 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-37790744
7.
Forced enhancer-promoter rewiring to alter gene expression in animal models.
Mol Ther Nucleic Acids
; 31: 452-465, 2023 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-36852088
8.
Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Patients with Hemophilia A in India.
Indian J Hematol Blood Transfus
; : 1-6, 2022 Nov 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-36467512
9.
MBD2 contributes to developmental silencing of the human ε-globin gene.
Blood Cells Mol Dis
; 46(3): 212-9, 2011 Mar 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-21296012
10.
First open-label, single-arm, prospective study of real-world use of FIX replacement therapy in a predominantly pediatric hemophilia B population in China.
Medicine (Baltimore)
; 100(21): e26077, 2021 May 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-34032739
11.
Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua.
J Thromb Haemost
; 19(5): 1212-1218, 2021 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33636038
12.
Safety and Efficacy of Moroctocog Alfa (AF-CC) in Chinese Patients with Hemophilia A: Results of Two Open-Label Studies.
J Blood Med
; 11: 439-448, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-33269010
13.
Assessment of Relative Bioavailability of Moroctocog Alfa and Moroctocog Alfa (AF-CC) in Subjects With Severe Hemophilia A.
Clin Transl Sci
; 11(3): 283-288, 2018 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-29575770
14.
Presentation of hemophagocytic lymphohistiocytosis due to a novel MUNC 13-4 mutation masked by partial therapeutic immunosuppression.
Pediatr Rheumatol Online J
; 10(1): 13, 2012 May 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-22554126
15.
Methyl binding domain protein 2 mediates gamma-globin gene silencing in adult human betaYAC transgenic mice.
Proc Natl Acad Sci U S A
; 103(17): 6617-22, 2006 Apr 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-16608912
16.
MBD2 is a critical component of a methyl cytosine-binding protein complex isolated from primary erythroid cells.
Blood
; 108(8): 2836-45, 2006 Oct 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-16778143
17.
A functional screen for Krüppel-like factors that regulate the human gamma-globin gene through the CACCC promoter element.
Blood Cells Mol Dis
; 35(2): 227-35, 2005.
Artigo
em Inglês
| MEDLINE | ID: mdl-16023392
18.
Iron nitrosyl hemoglobin formation from the reactions of hemoglobin and hydroxyurea.
Biochemistry
; 41(7): 2466-74, 2002 Feb 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-11841242