Detalhe da pesquisa
1.
Prevention of Cholestatic Liver Disease and Reduced Tumorigenicity in a Murine Model of PFIC Type 3 Using Hybrid AAV-piggyBac Gene Therapy.
Hepatology
; 70(6): 2047-2061, 2019 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-31099022
2.
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.
Nature
; 506(7488): 382-6, 2014 Feb 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-24390344
3.
Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno-associated virus/piggyBac transposase gene delivery system.
Hepatology
; 62(2): 417-28, 2015 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-26011400
4.
ACE2 Therapy Using Adeno-associated Viral Vector Inhibits Liver Fibrosis in Mice.
Mol Ther
; 23(9): 1434-43, 2015 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-25997428
5.
Adeno-associated virus-mediated rescue of neonatal lethality in argininosuccinate synthetase-deficient mice.
Mol Ther
; 21(10): 1823-31, 2013 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-23817206
6.
Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies.
Hum Gene Ther
; 34(17-18): 917-926, 2023 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-37350098
7.
Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.
J Inherit Metab Dis
; 35(4): 641-5, 2012 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-22403018
8.
Induction and prevention of severe hyperammonemia in the spfash mouse model of ornithine transcarbamylase deficiency using shRNA and rAAV-mediated gene delivery.
Mol Ther
; 19(5): 854-9, 2011 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-21386824
9.
Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice.
Mol Ther Methods Clin Dev
; 27: 352-367, 2022 Dec 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36381301
10.
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys.
Mol Ther Methods Clin Dev
; 23: 135-146, 2021 Dec 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34703837
11.
Sexually dimorphic patterns of episomal rAAV genome persistence in the adult mouse liver and correlation with hepatocellular proliferation.
Mol Ther
; 17(9): 1548-54, 2009 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-19568224
12.
AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice.
Mol Ther
; 17(8): 1340-6, 2009 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-19384294
13.
Use of a Hybrid Adeno-Associated Viral Vector Transposon System to Deliver the Insulin Gene to Diabetic NOD Mice.
Cells
; 9(10)2020 10 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33023100
14.
Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes.
JHEP Rep
; 2(1): 100065, 2020 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-32039406
15.
Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors.
Mol Ther
; 16(6): 1081-1088, 2008 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-28178471
16.
Gene delivery to the juvenile mouse liver using AAV2/8 vectors.
Mol Ther
; 16(6): 1081-8, 2008 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-18414478
17.
AAV-Mediated Gene Delivery to the Mouse Liver.
Methods Mol Biol
; 1937: 213-219, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-30706398
18.
Insights into Gene Therapy for Urea Cycle Defects by Mathematical Modeling.
Hum Gene Ther
; 30(11): 1385-1394, 2019 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-31215258
19.
Liver-Targeted Angiotensin Converting Enzyme 2 Therapy Inhibits Chronic Biliary Fibrosis in Multiple Drug-Resistant Gene 2-Knockout Mice.
Hepatol Commun
; 3(12): 1656-1673, 2019 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-31832573
20.
Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome.
Nat Genet
; 49(8): 1267-1273, 2017 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-28628105