Detalhe da pesquisa
1.
Long-term effect of gene therapy on Leber's congenital amaurosis.
N Engl J Med
; 372(20): 1887-97, 2015 May 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-25938638
2.
Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates.
J Transl Med
; 10: 122, 2012 Jun 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-22704060
3.
Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer.
Mol Ther
; 19(5): 870-5, 2011 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-21119625
4.
Development of a liver-specific Tet-on inducible system for AAV vectors and its application in the treatment of liver cancer.
Mol Ther
; 19(7): 1245-53, 2011 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-21364542
5.
Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.
Mol Ther
; 19(2): 243-50, 2011 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-20877347
6.
AAV gene therapy as a means to increase apolipoprotein (Apo) A-I and high-density lipoprotein-cholesterol levels: correction of murine ApoA-I deficiency.
J Gene Med
; 11(8): 697-707, 2009 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-19431216
7.
Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient mice.
J Gene Med
; 10(10): 1113-23, 2008 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-18720429
8.
A largely random AAV integration profile after LPLD gene therapy.
Nat Med
; 19(7): 889-91, 2013 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-23770691
9.
Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria.
Hum Gene Ther
; 24(12): 1007-17, 2013 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-24070415
10.
Adeno-associated virus liver transduction efficiency measured by in vivo [18F]FHBG positron emission tomography imaging in rodents and nonhuman primates.
Hum Gene Ther
; 22(8): 999-1009, 2011 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-21320035
11.
Novel tools for production and purification of recombinant adeno-associated viral vectors.
Methods Mol Med
; 76: 255-67, 2003.
Artigo
em Inglês
| MEDLINE | ID: mdl-12526168
12.
Reply to: NGS library preparation may generate artifactual integration sites of AAV vectors.
Nat Med
; 20(6): 578-9, 2014 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-24901561
13.
Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders.
Hum Gene Ther
; 20(8): 908-17, 2009 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-19419275
14.
Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCAD.
Hum Gene Ther
; 19(6): 579-88, 2008 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-18500942
15.
In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA.
Lab Invest
; 87(9): 893-902, 2007 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-17592477
16.
Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy.
Proc Natl Acad Sci U S A
; 103(31): 11748-53, 2006 Aug 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-16864772