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The American Journal of Human Genetics, ISSN 0002-9297, 06/2016, Volume 98, Issue 6, pp. 1193 - 1207
Journal Article
Lancet, The, ISSN 0140-6736, 2012, Volume 379, Issue 9813, pp. 335 - 341
Journal Article
Human Molecular Genetics, ISSN 0964-6906, 2017, Volume 26, Issue 21, pp. 4190 - 4202
Journal Article
Genome Research, ISSN 1088-9051, 2011, Volume 21, Issue 6, pp. 885 - 897
Journal Article
Molecular Therapy, ISSN 1525-0016, 06/2005, Volume 11, Issue 6, pp. 889 - 898
Glycogen storage disease type II (Pompe disease) causes death in infancy from cardiorespiratory failure due to acid α-glucosidase (GAA; acid maltase)... 
Pompe disease | Glycogen storage disease type II | Adeno-associated virus | Gene therapy | Acid α-glucosidase | MEDICINE, RESEARCH & EXPERIMENTAL | POMPE-DISEASE | NULL MUTATION | gene therapy | glycogen storage disease type II | adeno-associated virus | MEDIATED GENE-TRANSFER | KNOCKOUT MICE | acid alpha-glucosidase | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | KINASE CATALYTIC SUBUNIT | MOUSE MODEL | GENETICS & HEREDITY | AAV SEROTYPES | SKELETAL-MUSCLES | ACID-ALPHA-GLUCOSIDASE | NONSENSE MUTATION | Dependovirus - genetics | Genetic Vectors - administration & dosage | Myocardium - chemistry | alpha-Glucosidases - immunology | Humans | DNA, Viral - analysis | Glycogen Storage Disease Type II - therapy | Promoter Regions, Genetic - genetics | Muscle, Skeletal - chemistry | Glycogen - analysis | Gene Transfer Techniques | Muscle, Skeletal - enzymology | Glycogen Storage Disease Type II - genetics | Creatine Kinase, MM Form | Isoenzymes - genetics | alpha-Glucosidases - analysis | Injections, Intramuscular | Genetic Vectors - genetics | Mice, Knockout | Creatine Kinase - genetics | alpha-Glucosidases - genetics | Myocardium - enzymology | Animals | Antibody Formation | Antibodies - blood | Enhancer Elements, Genetic - genetics | Mice | Genetic Therapy - methods | Virus diseases | Amylases | Creatine | Glycogen | Creatine kinase | Index Medicus
Journal Article
Applied Microbiology and Biotechnology, ISSN 0175-7598, 11/2015, Volume 99, Issue 21, pp. 9123 - 9134
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