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EMBO Molecular Medicine, ISSN 1757-4676, 05/2013, Volume 5, Issue 5, pp. 691 - 706
A recently proposed therapeutic approach for lysosomal storage disorders (LSDs) relies upon the ability of transcription factor EB (TFEB) to stimulate... 
Pompe disease | TFEB | autophagy | acid alpha‐glucosidase | lysosomal storage | Acid alpha-glucosidase | Lysosomal storage | Autophagy | MEDICINE, RESEARCH & EXPERIMENTAL | EXOCYTOSIS | CHOLESTEROL ACCUMULATION | SATELLITE CELLS | LYSOSOMAL STORAGE DISORDERS | acid alpha-glucosidase | SKELETAL-MUSCLE | BIOGENESIS | MOUSE MODEL | ENZYME REPLACEMENT THERAPY | Cells, Cultured | Muscle, Skeletal - ultrastructure | Genetic Vectors - metabolism | Basic Helix-Loop-Helix Leucine Zipper Transcription Factors - chemistry | Muscle, Skeletal - metabolism | Exocytosis | Muscle, Skeletal - cytology | Glycogen Storage Disease Type II - pathology | Genetic Vectors - genetics | Mice, Knockout | Glycogen Storage Disease Type II - enzymology | alpha-Glucosidases - genetics | Animals | Glycogen - metabolism | Lysosomes - metabolism | alpha-Glucosidases - deficiency | Basic Helix-Loop-Helix Leucine Zipper Transcription Factors - genetics | Adenoviridae - genetics | Basic Helix-Loop-Helix Leucine Zipper Transcription Factors - metabolism | Mice | alpha-Glucosidases - metabolism | Disease Models, Animal | Glucose metabolism | Enzymes | Glycogen | Analysis | Stem cells | Muscles | Health aspects | Cell culture | Animal models | Transcription factors | Disease | Therapeutic applications | Lysosomal storage diseases | Metabolism | Experiments | Skeletal muscle | Storage diseases | Musculoskeletal system | Pathology | Laboratory animals | Vacuoles | Phagocytosis | Myopathy
Journal Article
Human Gene Therapy, ISSN 1043-0342, 05/2012, Volume 23, Issue 5, pp. 46 - 472
Pompe disease can be treated effectively, if immune tolerance to enzyme replacement therapy (ERT) with acid α-glucosidase (GAA) is present. An adeno-associated... 
Research Articles | MEDICINE, RESEARCH & EXPERIMENTAL | HEPATIC GENE-TRANSFER | DENDRITIC CELLS | TOLERANCE | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | FACTOR-IX | IN-VIVO | GENETICS & HEREDITY | ADENOASSOCIATED VIRUS VECTORS | MICE | ACID-ALPHA-GLUCOSIDASE | ENZYME REPLACEMENT THERAPY | T-CELLS | Cell Proliferation | Injections, Intravenous | Genetic Vectors - administration & dosage | Immunoglobulin G - blood | alpha-Glucosidases - immunology | Dendritic Cells - immunology | Humans | Glycogen Storage Disease Type II - therapy | Glycogen Storage Disease Type II - enzymology | Organ Specificity - genetics | Liver - immunology | alpha-Glucosidases - therapeutic use | T-Lymphocytes - metabolism | Transgenes | Glycogen Storage Disease Type II - genetics | Liver - metabolism | Cells, Cultured | Genetic Vectors - metabolism | Immune Tolerance | alpha-Glucosidases - analysis | Glycogen Storage Disease Type II - immunology | Dependovirus | Mice, Inbred Strains | Mice, Knockout | Enzyme Replacement Therapy - methods | alpha-Glucosidases - genetics | Animals | Dendritic Cells - enzymology | T-Lymphocytes - immunology | Mice | Genetic Therapy - methods | Cell proliferation | Spleen | Enzymes | alpha -Glucosidase | Immune response | Liver diseases | Cytokines | Dendritic cells | Glycogen | Liver | Antibodies | Adjuvants | Lymphocytes T | Immunological tolerance | Gene therapy | Chemokines | Expression vectors | Immune system
Journal Article
Journal Article
Genetics in Medicine, ISSN 1098-3600, 01/2017, Volume 19, Issue 1, pp. 90 - 97
Journal Article
Journal Article
Pediatrics, ISSN 0031-4005, 08/2003, Volume 112, Issue 2, pp. 332 - 340
Journal Article
Journal Article
Journal of Inherited Metabolic Disease, ISSN 0141-8955, 3/2015, Volume 38, Issue 2, pp. 305 - 314
Journal Article
Food Chemistry, ISSN 0308-8146, 01/2016, Volume 190, pp. 207 - 215
Journal Article
Journal Article