Detalhe da pesquisa
1.
Intranasal gene therapy to prevent infection by SARS-CoV-2 variants.
PLoS Pathog
; 17(7): e1009544, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-34265018
2.
Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: potential for biomarker development and insight into pathogenesis.
Hum Mol Genet
; 26(19): 3837-3849, 2017 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28934395
3.
Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes.
J Immunol
; 198(12): 4581-4587, 2017 06 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-28500077
4.
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.
Mol Ther
; 24(2): 206-216, 2016 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-26447927
5.
Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.
Proc Natl Acad Sci U S A
; 111(41): 14894-9, 2014 Oct 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-25267637
6.
Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model.
Mol Genet Metab
; 119(1-2): 124-30, 2016 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27386755
7.
Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
Mol Ther
; 23(8): 1298-1307, 2015 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-26022732
8.
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.
Blood
; 121(12): 2224-33, 2013 Mar 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-23325831
9.
Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.
Mol Ther
; 22(12): 2018-2027, 2014 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-25027660
10.
Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy.
Res Sq
; 2023 Jun 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-37461697
11.
Neonatal Fc Receptor Inhibition Enables Adeno-Associated Virus Gene Therapy Despite Pre-Existing Humoral Immunity.
Hum Gene Ther
; 34(19-20): 1022-1032, 2023 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-36719773
12.
Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy.
Commun Biol
; 6(1): 1093, 2023 10 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-37891254
13.
Adeno-associated virus-vectored erythropoietin gene therapy for anemia in cats with chronic kidney disease.
J Vet Intern Med
; 37(6): 2200-2210, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37847024
14.
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid.
Hum Gene Ther
; 34(21-22): 1095-1106, 2023 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-37624734
15.
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
16.
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
17.
A Single Injection of an Optimized Adeno-Associated Viral Vector into Cerebrospinal Fluid Corrects Neurological Disease in a Murine Model of GM1 Gangliosidosis.
Hum Gene Ther
; 31(21-22): 1169-1177, 2020 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-33045869
18.
Adeno-associated virus serotype 1-based gene therapy for FTD caused by GRN mutations.
Ann Clin Transl Neurol
; 7(10): 1843-1853, 2020 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-32937039
19.
Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration.
Mol Ther Methods Clin Dev
; 17: 969-974, 2020 Jun 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-32420410
20.
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