Detalhe da pesquisa
1.
Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy.
Ann Neurol
; 94(5): 969-986, 2023 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-37526361
2.
Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis.
Brain
; 145(2): 655-669, 2022 04 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-34410345
3.
PEA15 loss of function and defective cerebral development in the domestic cat.
PLoS Genet
; 16(12): e1008671, 2020 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-33290415
4.
A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system.
Mol Ther
; 29(9): 2806-2820, 2021 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34298128
5.
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease.
Gene Ther
; 28(3-4): 142-154, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32884151
6.
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.
Mol Ther
; 28(2): 411-421, 2020 02 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-31813800
7.
Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model.
Mol Ther
; 25(4): 892-903, 2017 04 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-28236574
8.
Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.
Mol Ther
; 24(4): 726-35, 2016 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-26708003
9.
In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy.
Mol Ther
; 24(7): 1247-57, 2016 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-27117222
10.
Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy.
Mol Genet Metab
; 116(1-2): 80-7, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-25971245
11.
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.
Mol Ther Methods Clin Dev
; 30: 303-314, 2023 Sep 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37601414
12.
A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis.
EBioMedicine
; 92: 104627, 2023 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-37267847
13.
Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.
Mol Ther Methods Clin Dev
; 31: 101122, 2023 Dec 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37920238
14.
Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System.
Hum Gene Ther
; 33(17-18): 889-892, 2022 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-36074937
15.
AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates.
Mol Ther Methods Clin Dev
; 27: 281-292, 2022 Dec 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36320411
16.
AAV gene therapy for Tay-Sachs disease.
Nat Med
; 28(2): 251-259, 2022 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-35145305
17.
Real-time MR tracking of AAV gene therapy with ßgal-responsive MR probe in a murine model of GM1-gangliosidosis.
Mol Ther Methods Clin Dev
; 23: 128-134, 2021 Dec 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34703836
18.
Natural history study of glycan accumulation in large animal models of GM2 gangliosidoses.
PLoS One
; 15(12): e0243006, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-33259552
19.
Canine Models of Inherited Musculoskeletal and Neurodegenerative Diseases.
Front Vet Sci
; 7: 80, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-32219101
20.
Abnormal epiphyseal development in a feline model of Sandhoff disease.
J Orthop Res
; 38(12): 2580-2591, 2020 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-32678923