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Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2018, Volume 123, Issue 2, pp. S95 - S95
Journal Article
Journal of Neuroscience Research, ISSN 0360-4012, 11/2016, Volume 94, Issue 11, pp. 1126 - 1137
Journal Article
Journal of neuroscience research, ISSN 0360-4012, 11/2016, Volume 94, Issue 11, pp. 1126 - 1137
Globoid cell leukodystrophy (GLD) is an autosomal recessive neurodegenerative disorder caused by a deficiency of the lysosomal enzyme galactocerebrosidase... 
gene therapy | bone marrow transplant | lysosomal storage disease | Krabbe disease | galactocerebrosidase
Journal Article
Molecular Therapy, ISSN 1525-0016, 03/2014, Volume 22, Issue 3, pp. 483 - 484
Journal Article
Journal of Agricultural Education, ISSN 1042-0541, 2019, Volume 60, Issue 2, p. 85
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2011, Volume 807, pp. 141 - 157
The liver is directly or indirectly involved in many essential processes and is affected by numerous inherited diseases. Therefore, many inherited diseases... 
Inherited metabolic disease | Adeno-associated virus | Hepatocytes | Gene therapy | Liver | Blotting, Southern | Dependovirus - genetics | Animals | Liver - metabolism | Polymerase Chain Reaction | Mice | Genetic Vectors - genetics | Genetic Therapy - methods | Inherited Metabolic Disease | Adeno-Associated Virus | Gene Therapy
Conference Proceeding
Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2018, Volume 123, Issue 2, pp. S85 - S86
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2018, Volume 123, Issue 2, pp. S104 - S104
Journal Article
EMBO Molecular Medicine, ISSN 1757-4676, 06/2013, Volume 5, Issue 6, pp. 799 - 801
Journal Article