Detalhe da pesquisa
1.
Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts.
Nucleic Acids Res
; 50(15): 8418-8430, 2022 08 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-35920332
2.
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
3.
The rapidly evolving state of gene therapy.
FASEB J
; 32(4): 1733-1740, 2018 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-31282760
4.
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
5.
Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency.
Methods Mol Biol
; 2750: 11-17, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38108963
6.
Alpha-1 Antitrypsin Deficiency.
Methods Mol Biol
; 2750: 1-7, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38108962
7.
Serum Western Blot for the Detection of a c-Myc Protein Tag in Non-human Primates and Mice.
Methods Mol Biol
; 2750: 107-112, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38108971
8.
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
9.
Gene therapy for alpha-1 antitrypsin deficiency: an update.
Expert Opin Biol Ther
; 23(3): 283-291, 2023 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36825473
10.
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells.
Mol Ther Methods Clin Dev
; 23: 490-506, 2021 Dec 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34853797
11.
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
12.
Genotyping Protocol for the Alpha-1 Antitrypsin (PiZ) Mouse Model.
Methods Mol Biol
; 1639: 61-65, 2017.
Artigo
em Inglês
| MEDLINE | ID: mdl-28752446
13.
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient.
Hum Gene Ther
; 33(9-10): 477-478, 2022 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-35576406
14.
Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.
Methods Mol Biol
; 1639: 267-275, 2017.
Artigo
em Inglês
| MEDLINE | ID: mdl-28752467
15.
Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA.
Methods Mol Biol
; 1639: 211-216, 2017.
Artigo
em Inglês
| MEDLINE | ID: mdl-28752460
16.
Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups.
Hum Gene Ther
; 28(3): 228-230, 2017 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28319444
17.
Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.
Hum Gene Ther Clin Dev
; 26(3): 159-64, 2015 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-26357010
18.
Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency.
Hum Gene Ther Methods
; 26(3): 77-81, 2015 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-26067712
19.
Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.
Hum Gene Ther Methods
; 26(2): 71-6, 2015 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-25819833
20.
Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus.
Curr Protoc Microbiol
; Chapter 14: Unit 14D.3, 2013.
Artigo
em Inglês
| MEDLINE | ID: mdl-23408131