Detalhe da pesquisa
1.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
N Engl J Med
; 377(23): 2215-2227, 2017 12 07.
Artigo
Inglês
| MEDLINE | ID: mdl-29211678
2.
Preclinical evaluation of an anti-HCV miRNA cluster for treatment of HCV infection.
Mol Ther
; 21(3): 588-601, 2013 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-23295950
3.
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.
Blood
; 115(23): 4678-88, 2010 Jun 10.
Artigo
Inglês
| MEDLINE | ID: mdl-20335222
4.
Inhibition of hepatitis C virus replication using adeno-associated virus vector delivery of an exogenous anti-hepatitis C virus microRNA cluster.
Hepatology
; 52(6): 1877-87, 2010 Dec.
Artigo
Inglês
| MEDLINE | ID: mdl-20931557
5.
Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver.
Hum Gene Ther
; 16(3): 299-306, 2005 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-15812225
6.
Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy.
Hum Gene Ther
; 13(11): 1281-91, 2002 Jul 20.
Artigo
Inglês
| MEDLINE | ID: mdl-12162811
7.
AAV-mediated gene therapy for hemophilia.
Curr Opin Mol Ther
; 5(5): 517-23, 2003 Oct.
Artigo
Inglês
| MEDLINE | ID: mdl-14601521
8.
Viral vector-mediated RNA interference.
Curr Opin Pharmacol
; 10(5): 534-42, 2010 Oct.
Artigo
Inglês
| MEDLINE | ID: mdl-20620113
9.
Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice.
Blood
; 107(5): 1810-7, 2006 Mar 01.
Artigo
Inglês
| MEDLINE | ID: mdl-16249376
10.
Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial.
Mol Ther
; 13(6): 1064-73, 2006 Jun.
Artigo
Inglês
| MEDLINE | ID: mdl-16631412
11.
Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs.
Blood
; 108(1): 107-15, 2006 Jul 01.
Artigo
Inglês
| MEDLINE | ID: mdl-16522813
12.
Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.
Blood
; 108(10): 3321-8, 2006 Nov 15.
Artigo
Inglês
| MEDLINE | ID: mdl-16868252
13.
Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model.
Blood
; 105(9): 3458-64, 2005 May 01.
Artigo
Inglês
| MEDLINE | ID: mdl-15479726
14.
Preclinical gene therapy studies for hemophilia using adeno-associated virus (AAV) vectors.
Semin Thromb Hemost
; 30(2): 161-71, 2004 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-15118928
15.
Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.
Blood
; 103(1): 85-92, 2004 Jan 01.
Artigo
Inglês
| MEDLINE | ID: mdl-12969984
16.
Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII.
Blood
; 102(12): 3919-26, 2003 Dec 01.
Artigo
Inglês
| MEDLINE | ID: mdl-12893764
17.
Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector.
Blood
; 102(6): 2031-7, 2003 Sep 15.
Artigo
Inglês
| MEDLINE | ID: mdl-12738670
18.
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.
Blood
; 101(8): 2963-72, 2003 Apr 15.
Artigo
Inglês
| MEDLINE | ID: mdl-12515715