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Human molecular genetics, ISSN 0964-6906, 06/2019
The focus of this review is the development of gene therapy for glycogen storage diseases (GSD). GSD results from the deficiency of specific enzymes involved... 
Journal Article
Blood, ISSN 0006-4971, 03/2015, Volume 125, Issue 10, pp. 1509 - 1510
In this issue of Blood, Crudele et al describe a novel study of adeno-associated virus (AAV) vector-mediated gene therapy that induced immune tolerance to... 
ANTIBODIES | LIVER GENE-THERAPY | EFFICACY | FACTOR-IX | MICE | PADUA | HEMATOLOGY | Hemophilia B - therapy | Factor IX - antagonists & inhibitors | Animals | Humans | Male | Hemophilia B - genetics | Genetic Therapy - methods | Index Medicus | Abridged Index Medicus
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2016, Volume 24, pp. S152 - S152
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2019, Volume 126, Issue 2, pp. S67 - S67
Journal Article
Journal of Inherited Metabolic Disease, ISSN 0141-8955, 12/2018, Volume 41, Issue 6, pp. 913 - 914
Journal Article
FASEB JOURNAL, ISSN 0892-6638, 01/2013, Volume 27, Issue 1, pp. 34 - 44
Journal Article
Thyroid, ISSN 1050-7256, 08/2019, Volume 29, Issue 8, pp. 1158 - 1167
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 01/2017, Volume 120, Issue 1-2, pp. S62 - S62
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2016, Volume 24, pp. S22 - S22
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2015, Volume 23, pp. S279 - S279
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 06/2016, Volume 126, Issue 6, pp. 2043 - 2044
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 06/2019, Volume 13, pp. 265 - 273
Glycogen storage disease type Ia (GSD Ia) is a rare inherited disease caused by mutations in the glucose-6-phosphatase (G6Pase) catalytic subunit gene ( ).... 
genome editing | glycogen storage disease type Ia | bezafibrate | autophagy | zinc-finger nuclease | gene therapy | adeno-associated virus | pan-agonist of peroxisome proliferator-activated receptors | MEDICINE, RESEARCH & EXPERIMENTAL | EFFICACY | METABOLISM | GLUCOSE | ADENOASSOCIATED VIRUS VECTORS | TRANSDUCTION | GENE-THERAPY | RECEPTORS | EFFICIENCY | CANINE | DEFICIENCY
Journal Article
MOLECULAR GENETICS AND METABOLISM, ISSN 1096-7192, 02/2012, Volume 105, Issue 2, pp. 221 - 227
Enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase (rhGAA) has improved clinical outcomes in patients with Pompe disease; however,... 
MEDICINE, RESEARCH & EXPERIMENTAL | Bompe disease | CLENBUTEROL | RAT | Acid alpha-glucosidase | Mannose-6-phosphate receptor | VII MICE | BLOOD-BRAIN-BARRIER | Glycogen storage disease type II | Enzyme replacement therapy | Acid maltase | RECOMBINANT | MUSCLE-FIBERS | MOUSE MODEL | MASS | ENDOCRINOLOGY & METABOLISM | GENETICS & HEREDITY | EXPRESSION
Journal Article
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