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JAMA Neurology, ISSN 2168-6149, 08/2018, Volume 75, Issue 8, p. 1027
Journal Article
1970, Handbook of clinical neurology, Volume 10, 687
Book
2011, International review of child neurology series, ISBN 1907655417
Web Resource
Journal Article
European Journal of Neurology, ISSN 1351-5101, 07/2014, Volume 21, Issue 7, pp. 983 - 988
Journal Article
Human molecular genetics, ISSN 0964-6906, 2015, Volume 24, Issue 12, pp. 3372 - 3389
Journal Article
The Journal of Pathology, ISSN 0022-3417, 04/2014, Volume 232, Issue 5, pp. 509 - 521
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 05/2005, Volume 352, Issue 20, pp. 2069 - 2081
Journal Article
Journal of Neuroscience, ISSN 0270-6474, 04/2015, Volume 35, Issue 16, pp. 6495 - 6505
Journal Article
Molecular Therapy, ISSN 1525-0016, 03/2018, Volume 26, Issue 3, pp. 874 - 889
We report a global adeno-associated virus (AAV)9-based gene therapy protocol to deliver therapeutic galactosylceramidase (GALC), a lysosomal enzyme that is... 
myelin | galactosylceramidase | Krabbe's disease | psychosine | AAV | demyelination | gene therapy | leukodystrophy | oligodendrocytes | microglia | MEDICINE, RESEARCH & EXPERIMENTAL | GALACTOCEREBROSIDASE | BONE-MARROW-TRANSPLANTATION | MUTANT TWITCHER | AXONAL-TRANSPORT | MURINE MODEL | CORD BLOOD TRANSPLANTATION | INTRAVENOUS-INJECTION | TWITCHER MICE | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | CENTRAL-NERVOUS-SYSTEM | GLOBOID-CELL LEUKODYSTROPHY | Dependovirus - genetics | Genetic Therapy | Genetic Vectors - administration & dosage | Leukodystrophy, Globoid Cell - diagnosis | Autonomic Pathways - pathology | Male | Myelin Sheath - metabolism | Behavior, Animal | Brain - metabolism | Tissue Distribution | Galactosylceramidase - genetics | Leukodystrophy, Globoid Cell - metabolism | Axons - ultrastructure | Autonomic Pathways - metabolism | Female | Autonomic Pathways - ultrastructure | Leukodystrophy, Globoid Cell - therapy | Disease Models, Animal | Gene Expression | Transduction, Genetic | Myelin Sheath - pathology | Carbohydrate Metabolism | Axons - metabolism | Treatment Outcome | Genetic Vectors - genetics | Phenotype | Animals | Axons - pathology | Leukodystrophy, Globoid Cell - genetics | Mice | Myelin Sheath - ultrastructure | Genetic Vectors - pharmacokinetics | Galactosylceramidase - metabolism | Index Medicus
Journal Article
2012, ISBN 2894726430, 226
Book
Genetics in Medicine, ISSN 1098-3600, 03/2016, Volume 18, Issue 3, pp. 239 - 248
Journal Article
Science, ISSN 0036-8075, 8/2013, Volume 341, Issue 6148, pp. 864 - 864
Journal Article
STEM CELLS, ISSN 1066-5099, 10/2011, Volume 29, Issue 10, pp. 1559 - 1571
Murine neural stem cells (mNSCs), either naive or genetically modified to express supranormal levels of β‐galactocerebrosidase (GALC), were transplanted into... 
Leukodystrophies | Stem cell transplantation | Lysosomal storage diseases | Nervous system | Neural stem cells | Gene therapy | PROGENITOR CELLS | MULTIPLE MECHANISMS | LYSOSOMAL STORAGE | CELL & TISSUE ENGINEERING | CELL BIOLOGY | TWITCHER MICE | ONCOLOGY | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | BETA-GALACTOSIDASE | CENTRAL-NERVOUS-SYSTEM | HEMATOLOGY | ENZYME REPLACEMENT THERAPY | KRABBE-DISEASE | GALACTOCEREBROSIDASE ACTIVITY | METACHROMATIC-LEUKODYSTROPHY | Humans | Brain - enzymology | Neural Stem Cells - cytology | Mice, Neurologic Mutants | Lentivirus - metabolism | Stem Cell Transplantation | Galactosylceramidase - genetics | Spinal Cord - pathology | Neural Stem Cells - transplantation | Lentivirus - genetics | Cell Differentiation | Leukodystrophy, Globoid Cell - therapy | Transgenes | Disease Models, Animal | Mice, Inbred C57BL | Cells, Cultured | Leukodystrophy, Globoid Cell - pathology | Spinal Cord - enzymology | Genetic Vectors - metabolism | Genetic Vectors - genetics | Animals | Brain - pathology | Leukodystrophy, Globoid Cell - enzymology | Leukodystrophy, Globoid Cell - genetics | Mice | Enzyme Activation | Galactosylceramidase - therapeutic use | Neural Stem Cells - metabolism | Galactosylceramidase - metabolism | Genetic Therapy - methods | Index Medicus | Original Research | Regenerative Medicine
Journal Article