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Human Gene Therapy, ISSN 1043-0342, 03/2018, Volume 29, Issue 3, pp. 312 - 326
Journal Article
Human Gene Therapy, ISSN 1043-0342, 06/2017, Volume 28, Issue 6, pp. 51 - 522
GM2 gangliosidoses, including Tay–Sachs disease and Sandhoff disease, are lysosomal storage disorders caused by deficiencies in β-N-acetylhexosaminidase (Hex).... 
Research Articles | hexosaminidase | gene therapy | adeno-associated virus | Tay-Sachs disease | intracranial delivery | AAV | LONG-TERM | MOUSE-BRAIN | MEDICINE, RESEARCH & EXPERIMENTAL | SINGLE INJECTION | SANDHOFF-DISEASE | VECTOR | DELIVERY | FELINE MODEL | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | NERVOUS-SYSTEM DISEASE | GENE-THERAPY | REDUCES LYSOSOMAL STORAGE | Dependovirus - genetics | Neurons - pathology | Thalamus - metabolism | White Matter - metabolism | Injections, Intraventricular | Macaca fascicularis | Genetic Vectors - adverse effects | Male | Thalamus - pathology | Gangliosidoses, GM2 - pathology | Necrosis - pathology | Protein Subunits - metabolism | Apathy | Gray Matter - metabolism | Female | Neurons - metabolism | beta-N-Acetylhexosaminidases - genetics | Transgenes | Gangliosidoses, GM2 - genetics | Dyskinesias - genetics | Disease Models, Animal | Protein Subunits - genetics | Gangliosidoses, GM2 - therapy | Gene Expression | Dependovirus - metabolism | Genetic Vectors - chemistry | beta-N-Acetylhexosaminidases - metabolism | Dyskinesias - pathology | Genetic Vectors - metabolism | Necrosis - metabolism | Dyskinesias - etiology | Dyskinesias - metabolism | White Matter - pathology | Protein Subunits - adverse effects | Animals | Necrosis - etiology | Gray Matter - pathology | Necrosis - genetics | beta-N-Acetylhexosaminidases - adverse effects | Gangliosidoses, GM2 - metabolism | Genetic Therapy - methods | Neuroimaging | Brain | Hex protein | Central nervous system | Lysosomal storage diseases | Ventricles (cerebral) | Monkeys | Substantia grisea | Necrosis | Proteins | Infusion | Neurotoxicity | Histopathology | Coding | Ataxia | Thalamus | Safety | Lesions | Leukocytes (eosinophilic) | Abnormalities | Injection | Magnetic resonance imaging | Sheep | Ventricle | Index Medicus
Journal Article
Journal Article
Journal Article
Journal of Neuroscience Research, ISSN 0360-4012, 11/2016, Volume 94, Issue 11, pp. 1007 - 1017
Journal Article
Journal of Neuroscience Methods, ISSN 0165-0270, 04/2014, Volume 227, pp. 10 - 17
Feline models of neurologic diseases, such as lysosomal storage diseases, leukodystrophies, Parkinson's disease, stroke and NeuroAIDS, accurately recreate many... 
Brain | Animal model | Translational | MRI | Cat | Central nervous system | Magnetic resonance imaging | Cats | Analysis
Journal Article
Experimental Neurology, ISSN 0014-4886, 01/2015, Volume 263, pp. 102 - 112
Journal Article
Molecular Therapy, ISSN 1525-0016, 04/2017, Volume 25, Issue 4, pp. 892 - 903
Journal Article
Neuroscience, ISSN 0306-4522, 2016, Volume 340, pp. 117 - 125
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 09/2015, Volume 116, Issue 1-2, pp. 80 - 87
Journal Article
Journal Article