Detalhe da pesquisa
1.
BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression.
Blood
; 137(6): 763-774, 2021 02 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-33067633
2.
A systematic review of adeno-associated virus gene therapy clinical trials for HIV - A potential solution for patients with haemophilia and HIV?
Haemophilia
; 29(3): 784-789, 2023 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-36952285
3.
Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial.
Blood
; 124(26): 3880-6, 2014 Dec 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-25261199
4.
Impact of inhibitors on hemophilia A mortality in the United States.
Am J Hematol
; 90(5): 400-5, 2015 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-25616111
5.
Optimizing Liver Health Before and After Gene Therapy for Hemophilia A.
Blood Adv
; 2024 Jun 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-38843379
6.
Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice.
Blood
; 116(8): 1235-43, 2010 Aug 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-20445020
7.
Adeno-associated virus serotype 8 ApoA-I gene transfer reduces progression of atherosclerosis in ApoE-KO mice: comparison of intramuscular and intravenous administration.
J Cardiovasc Pharmacol
; 57(3): 325-33, 2011 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-21164355
8.
Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing.
Nat Med
; 9(8): 1015-9, 2003 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-12847523
9.
Safe and sustained overexpression of functional apolipoprotein A-I/high-density lipoprotein in apolipoprotein A-I-null mice by muscular adeno-associated viral serotype 8 vector gene transfer.
J Cardiovasc Pharmacol
; 54(5): 405-11, 2009 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-19701094
10.
Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats.
Mol Pain
; 2: 4, 2006 Jan 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-16445862
11.
RNA repair for haemophilia A.
Expert Rev Mol Med
; 8(1): 1-8, 2006 Jan 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-16401355
12.
Current and future prospects for hemophilia gene therapy.
Expert Rev Hematol
; 9(7): 649-59, 2016 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-27153210
13.
The burden of inhibitors in haemophilia patients.
Thromb Haemost
; 116 Suppl 1: S10-7, 2016 08 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-27528280
14.
Intrathecal gene transfer by adeno-associated virus for pain.
Curr Opin Mol Ther
; 7(5): 431-9, 2005 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-16248278
15.
New paradigms for gene transfer: RNA trans-splicing and small interfering RNA as therapeutic strategies.
Semin Hematol
; 41(4): 297-302, 2004 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-15508115
16.
Lentivirus vector purification using anion exchange HPLC leads to improved gene transfer.
Biotechniques
; 34(5): 1074-8, 1080, 2003 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-12765034
17.
Hemophilia gene therapy: novel rAAV vectors and RNA repair strategy.
Curr Opin Mol Ther
; 4(5): 499-504, 2002 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-12435052
18.
AAV vectors for hemophilia B gene therapy.
Mt Sinai J Med
; 71(5): 305-13, 2004 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-15543431
19.
Efficacy of EHL N9-GP for on-demand treatment of bleeding episodes in hemophilia B: analysis of pivotal trial data.
J Blood Med
; 10: 243-250, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-31413650
20.
Hemophilia clinical gene therapy: brief review.
Transl Res
; 161(4): 307-12, 2013 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-23352600