Detalhe da pesquisa
1.
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury.
Mol Ther
; 32(4): 952-968, 2024 Apr 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-38327046
2.
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9.
Mol Ther
; 29(6): 2019-2029, 2021 06 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33609733
3.
IL-27 Limits Type 2 Immunopathology Following Parainfluenza Virus Infection.
PLoS Pathog
; 13(1): e1006173, 2017 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28129374
4.
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice.
Mol Ther
; 26(3): 664-668, 2018 03 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29428298
5.
Adeno-associated viral gene therapy corrects a mouse model of argininosuccinic aciduria.
Mol Genet Metab
; 125(3): 241-250, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-30253962
6.
Mucopolysaccharidosis Type VI in a Great Dane Caused by a Nonsense Mutation in the ARSB Gene.
Vet Pathol
; 55(2): 286-293, 2018 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-29157190
7.
Genetic lineage tracing analysis of the cell of origin of hepatotoxin-induced liver tumors in mice.
Hepatology
; 64(4): 1163-1177, 2016 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-27099001
8.
Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase.
Front Immunol
; 14: 1094279, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37033976
9.
Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model.
Mol Ther Methods Clin Dev
; 29: 32-39, 2023 Jun 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36936447
10.
Erratum: Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors.
Mol Ther Methods Clin Dev
; 27: 450-451, 2022 Dec 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36419470
11.
Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors.
Mol Ther Methods Clin Dev
; 27: 272-280, 2022 Dec 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36320416
12.
Determining the Minimally Effective Dose of a Clinical Candidate Adeno-Associated Virus Vector in a Mouse Model of Hemophilia A.
Hum Gene Ther
; 33(7-8): 421-431, 2022 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-34652966
13.
Efficacy and Safety of a Krabbe Disease Gene Therapy.
Hum Gene Ther
; 33(9-10): 499-517, 2022 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-35333110
14.
Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression.
Mol Ther Methods Clin Dev
; 24: 292-305, 2022 Mar 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35211641
15.
Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases.
Hum Gene Ther
; 33(21-22): 1174-1186, 2022 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-36375122
16.
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.
Sci Transl Med
; 12(569)2020 11 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-33177182
17.
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology.
Hum Gene Ther
; 31(15-16): 808-818, 2020 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-32845779
18.
Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys.
Hum Gene Ther
; 30(8): 957-966, 2019 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-31017018
19.
A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.
Hum Gene Ther Clin Dev
; 30(1): 29-39, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30693797
20.
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques.
Mol Ther Methods Clin Dev
; 10: 68-78, 2018 Sep 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-30073178