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Nature Communications, ISSN 2041-1723, 07/2017, Volume 8, Issue 1, pp. 16105 - 16105
Journal Article
Molecular Therapy, ISSN 1525-0016, 03/2018, Volume 26, Issue 3, pp. 890 - 901
Glycogen storage disease type III (GSDIII) is an autosomal recessive disorder caused by a deficiency of glycogen-debranching enzyme (GDE), which results in... 
dual AAV vectors | acid-alpha-glucosidase | neuromuscular disease | gene therapy | glycogen storage disease type III | glycogenosis | Cori disease | adeno-associated vector | MEDICINE, RESEARCH & EXPERIMENTAL | EFFICACY | ADENOASSOCIATED VIRUS | TRANSDUCTION | GENOME | THERAPY | GLYCOGEN-STORAGE-DISEASE | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | AAV | MOUSE MODEL | IN-VIVO | GENETICS & HEREDITY | VECTORS | Dependovirus - genetics | Glycogen Storage Disease Type III - metabolism | Genetic Vectors - administration & dosage | Male | Muscle, Skeletal - metabolism | Hepatocytes - metabolism | Glycogen - metabolism | Glycogen Debranching Enzyme System - genetics | Disease Models, Animal | Gene Transfer Techniques | Gene Expression | Glycogen Storage Disease Type III - therapy | Glycogen Debranching Enzyme System - metabolism | Glycogen Storage Disease Type III - diagnosis | Liver - metabolism | Blood Glucose | Organ Specificity | Genetic Vectors - genetics | Glycogen Storage Disease Type III - genetics | Mice, Knockout | Phenotype | Animals | Biomarkers | Mice | Enzyme Activation | Genetic Therapy - methods | Enzymes | Phenotypes | Statistical analysis | Gene transfer | Glycogen | Cardiomyopathy | Liver | Glucose | Gene expression | Hypoglycemia | Metabolism | Vectors (Biology) | Hereditary diseases | Storage diseases | Consortia | Glucose metabolism | Musculoskeletal system | Biopsy | Gene therapy | Age | Index Medicus | Original
Journal Article
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 06/2019, Volume 13, pp. 494 - 502
Sarcoglycanopathies are rare autosomic limb girdle muscular dystrophies caused by mutations in one of the genes coding for sarcoglycans. Sarcoglycans form a... 
translational medicine | miRNA biomarker | AAV | γ-sarcoglycanopathy | gene therapy | mechanical stress | limb girdle muscular dystrophy | LGMD2C | MEDICINE, RESEARCH & EXPERIMENTAL | IMAGE | GENE | MUTATION | MIRNAS | SPECTRUM | MUSCLES | Life Sciences | Biotechnology | Immunology | Quantitative Methods | Biochemistry, Molecular Biology | Genetics | Cellular Biology
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 06/2018, Volume 9, pp. 257 - 269
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 2016, Volume 3, Issue C, pp. 16049 - 16049
Crigler-Najjar syndrome is a severe metabolic disease of the liver due to a reduced activity of the UDP Glucuronosyltransferase 1A1 (UGT1A1) enzyme. In an... 
Life Sciences | Human health and pathology
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 2016, Volume 3, Issue C, pp. 16060 - 16060
Spinal muscular atrophy (SMA) is an autosomal recessive disease of variable severity caused by mutations in the gene. Deficiency of the ubiquitous SMN function... 
SURVIVAL | MEDICINE, RESEARCH & EXPERIMENTAL | CORD | AAV VECTOR | SMA MICE | DISEASE | SMN2 | GENE-TRANSFER | SMN-DELTA-7 | MOTOR-NEURON PROTEIN | DELIVERY | Life Sciences | Biochemistry, Molecular Biology
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2016, Volume 24, pp. S273 - S274
Journal Article