Detalhe da pesquisa
1.
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.
N Engl J Med
; 383(2): 151-158, 2020 07 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32640133
2.
Transcriptomic Analysis Reveals the Inability of Recombinant AAV8 to Activate Human Monocyte-Derived Dendritic Cells.
Int J Mol Sci
; 24(13)2023 Jun 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-37445621
3.
CHOP and c-JUN up-regulate the mutant Z α1-antitrypsin, exacerbating its aggregation and liver proteotoxicity.
J Biol Chem
; 295(38): 13213-13223, 2020 09 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-32723872
4.
Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans.
Mol Ther
; 28(3): 747-757, 2020 03 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-31982038
5.
Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.
Mol Ther
; 25(11): 2477-2489, 2017 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29032169
6.
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.
Mol Ther
; 25(6): 1387-1394, 2017 06 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-28408179
7.
Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.
Mol Ther
; 22(9): 1605-13, 2014 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-24954473
8.
Prevalence Study of Cellular Capsid-Specific Immune Responses to AAV2, 4, 5, 8, 9, and rh10 in Healthy Donors.
Hum Gene Ther
; 2024 Apr 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-38581431
9.
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques.
Mol Ther Methods Clin Dev
; 32(1): 101200, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38445045
10.
Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice.
Mol Ther Methods Clin Dev
; 25: 425-438, 2022 Jun 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35592360
11.
AAV gene therapy for Tay-Sachs disease.
Nat Med
; 28(2): 251-259, 2022 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-35145305
12.
AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation.
Mol Ther Methods Clin Dev
; 20: 660-674, 2021 Mar 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-33718516
13.
Engraftment of Human Hepatocytes in the PiZ-NSG Mouse Model.
Methods Mol Biol
; 2164: 75-85, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-32607885
14.
Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity.
Hum Gene Ther
; 30(7): 802-813, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30808235
15.
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study.
Mol Ther Methods Clin Dev
; 13: 233-242, 2019 Jun 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-30828586
16.
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
Nat Biotechnol
; 37(8): 884-894, 2019 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-31375812
17.
Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques.
Sci Transl Med
; 10(465)2018 10 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-30381409
18.
Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.
Hum Gene Ther
; 29(6): 663-673, 2018 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-29207890
19.
Regulatory and Exhausted T Cell Responses to AAV Capsid.
Hum Gene Ther
; 28(4): 338-349, 2017 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-28323492
20.
Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.
Hum Gene Ther
; 27(1): 19-31, 2016 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-26710998