Detalhe da pesquisa
1.
Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy.
N Engl J Med
; 389(13): 1203-1210, 2023 Sep 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-37754285
2.
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
3.
A Translational Model for Venous Thromboembolism: MicroRNA Expression in Hibernating Black Bears.
J Surg Res
; 257: 203-212, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32858321
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.
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
6.
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
7.
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
8.
CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro.
Nat Commun
; 13(1): 6286, 2022 10 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-36271076
9.
AAV gene therapy for Tay-Sachs disease.
Nat Med
; 28(2): 251-259, 2022 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-35145305
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.
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