Detalhe da pesquisa
1.
Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy.
Mol Ther
; 32(1): 44-58, 2024 Jan 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-37952085
2.
Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.
Mol Ther
; 32(6): 1701-1720, 2024 Jun 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-38549375
3.
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
4.
Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases.
Mol Ther
; 31(1): 7-23, 2023 01 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36196048
5.
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Mol Ther
; 30(7): 2416-2428, 2022 07 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35585789
6.
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
7.
Cerebrospinal fluid and serum glycosphingolipid biomarkers in canine globoid cell leukodystrophy (Krabbe Disease).
Mol Cell Neurosci
; 102: 103451, 2020 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31794880
8.
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
9.
Canine and Feline Models of Human Genetic Diseases and Their Contributions to Advancing Clinical Therapiesâ©.
Yale J Biol Med
; 90(3): 417-431, 2017 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-28955181
10.
Clinical, electrophysiological, and biochemical markers of peripheral and central nervous system disease in canine globoid cell leukodystrophy (Krabbe's disease).
J Neurosci Res
; 94(11): 1007-17, 2016 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-27638585
11.
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
12.
Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.
Mol Ther
; 21(7): 1306-15, 2013 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-23689599
13.
CD8+ T cell depletion prevents neuropathology in a mouse model of globoid cell leukodystrophy.
J Exp Med
; 220(9)2023 09 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-37310382
14.
Efficacy and Safety of a Krabbe Disease Gene Therapy.
Hum Gene Ther
; 33(9-10): 499-517, 2022 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-35333110
15.
Recent Advancements in the Diagnosis and Treatment of Leukodystrophies.
Semin Pediatr Neurol
; 37: 100876, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33892849
16.
Krabbe disease: New hope for an old disease.
Neurosci Lett
; 752: 135841, 2021 05 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33766733
17.
Canine Models of Inherited Musculoskeletal and Neurodegenerative Diseases.
Front Vet Sci
; 7: 80, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-32219101
18.
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.
J Clin Invest
; 130(9): 4906-4920, 2020 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32773406
19.
Neurodegenerative lysosomal storage disease in European Burmese cats with hexosaminidase beta-subunit deficiency.
Mol Genet Metab
; 97(1): 53-9, 2009 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-19231264
20.
Brain-targeted ex vivo lentiviral gene therapy: Implications for MPS and beyond.
Mol Ther Methods Clin Dev
; 31: 101137, 2023 Dec 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38027070