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1.
Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy.
Ann Neurol
; 94(5): 969-986, 2023 11.
Article
in English
| MEDLINE | ID: mdl-37526361
2.
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.
Article
in English
| MEDLINE | ID: mdl-32884151
3.
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.
Article
in English
| MEDLINE | ID: mdl-28236574
4.
Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy.
Mol Genet Metab
; 116(1-2): 80-7, 2015.
Article
in English
| MEDLINE | ID: mdl-25971245
5.
Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.
Mol Ther
; 21(7): 1306-15, 2013 Jul.
Article
in English
| MEDLINE | ID: mdl-23689599
6.
Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy.
Mol Ther Methods Clin Dev
; 6: 135-142, 2017 Sep 15.
Article
in English
| MEDLINE | ID: mdl-28808666
7.
AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease.
Neuroscience
; 340: 117-125, 2017 01 06.
Article
in English
| MEDLINE | ID: mdl-27793778
8.
AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.
ASN Neuro
; 7(2)2015.
Article
in English
| MEDLINE | ID: mdl-25873306
9.
Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease.
Exp Neurol
; 263: 102-12, 2015 Jan.
Article
in English
| MEDLINE | ID: mdl-25284324
10.
High resolution MRI anatomy of the cat brain at 3 Tesla.
J Neurosci Methods
; 227: 10-7, 2014 Apr 30.
Article
in English
| MEDLINE | ID: mdl-24525327
11.
Sustained normalization of neurological disease after intracranial gene therapy in a feline model.
Sci Transl Med
; 6(231): 231ra48, 2014 Apr 09.
Article
in English
| MEDLINE | ID: mdl-24718858
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