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Human Gene Therapy, ISSN 1043-0342, 03/2018, Volume 29, Issue 3, pp. 312 - 326
Journal Article
Current Gene Therapy, ISSN 1566-5232, 2018, Volume 18, Issue 2, pp. 68 - 89
Journal Article
Journal Article
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 7/2006, Volume 103, Issue 27, pp. 10373 - 10378
Tay-Sachs disease is a prototypic neurodegenerative disease. Lysosomal storage of GM2 ganglioside in Tay-Sachs and the related disorder, Sandhoff disease, is... 
Nervous system diseases | Spinal cord | Genetic vectors | Neurodegenerative diseases | Mice | Gene therapy | Sandhoff disease | Gangliosides | Tay Sachs disease | Gangliosidoses | GM2 gangliosidosis | Neurodegeneration | Adeno-associated virus | LYSOSOMAL STORAGE DISEASE | BONE-MARROW-TRANSPLANTATION | MULTIDISCIPLINARY SCIENCES | neurodegeneration | adeno-associated virus | SANDHOFF-DISEASE | VIRAL VECTORS | N-BUTYLDEOXYNOJIRIMYCIN | MOUSE MODELS | BETA-HEXOSAMINIDASE ACTIVITY | CENTRAL-NERVOUS-SYSTEM | MICE | Inflammation - pathology | Tay-Sachs Disease - therapy | beta-N-Acetylhexosaminidases - metabolism | Survival Rate | Microscopy, Electron | Body Weight - genetics | Tay-Sachs Disease - genetics | Genetic Vectors - genetics | Protein Subunits - metabolism | Mice, Knockout | beta-N-Acetylhexosaminidases - deficiency | Gene Expression Regulation, Enzymologic | Inflammation - metabolism | Animals | Tay-Sachs Disease - metabolism | Glycosphingolipids - metabolism | Inflammation - genetics | Tay-Sachs Disease - pathology | Inflammation - prevention & control | beta-N-Acetylhexosaminidases - genetics | Disease Models, Animal | Genetic Therapy - methods | Protein Subunits - genetics | Tay-Sachs disease | Research | Proteins | Nervous system | Genetic disorders | Neurological disorders | Index Medicus | Biological Sciences
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
Nature Medicine, ISSN 1078-8956, 04/2007, Volume 13, Issue 4, pp. 439 - 447
Journal Article
Molecular Therapy, ISSN 1525-0016, 03/2015, Volume 23, Issue 3, pp. 414 - 422
Journal Article
Human Molecular Genetics, ISSN 0964-6906, 02/2014, Volume 23, Issue 3, pp. 730 - 748
The GM2 gangliosidoses are progressive neurodegenerative disorders due to defects in the lysosomal beta-N-acetylhexosaminidase system. Accumulation of... 
BIOCHEMISTRY & MOLECULAR BIOLOGY | MOUSE MODEL | GM2 GANGLIOSIDOSIS | GENETICS & HEREDITY | NERVOUS-SYSTEM INFLAMMATION | HEXOSAMINIDASE ACTIVITY | GENE-THERAPY | ALPHA-SYNUCLEIN | LYSOSOMAL STORAGE DISORDERS | SANDHOFF-DISEASE |