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Neuropediatrics, ISSN 0174-304X, 10/2016, Volume 47, Issue 5, pp. 285 - 292
gene therapy | indications | Review | neurodevelopment | hematopoietic cell transplantation | Journal Article | Review Article | Pediatrics | Clinical Neurology | Neurosciences & Neurology | Life Sciences & Biomedicine | Science & Technology | Adrenoleukodystrophy - physiopathology | Humans | Mucopolysaccharidosis I - physiopathology | Adrenoleukodystrophy - psychology | Leukodystrophy, Metachromatic - psychology | Metabolism, Inborn Errors - therapy | Neurodevelopmental Disorders - psychology | Leukodystrophy, Globoid Cell - psychology | Leukodystrophy, Globoid Cell - physiopathology | Adrenoleukodystrophy - therapy | Leukodystrophy, Globoid Cell - complications | Mucopolysaccharidosis I - therapy | Adrenoleukodystrophy - complications | Leukodystrophy, Globoid Cell - therapy | Neurodevelopmental Disorders - etiology | Leukodystrophy, Metachromatic - physiopathology | Hematopoietic Stem Cell Transplantation | Leukodystrophy, Metachromatic - therapy | Neurodevelopmental Disorders - physiopathology | Mucopolysaccharidosis I - complications | Leukodystrophy, Metachromatic - complications | Mucopolysaccharidosis I - psychology | Metabolism, Inborn Errors - psychology | Metabolism, Inborn Errors - complications | Metabolism, Inborn Errors - physiopathology
Journal Article
Archives of neurology (Chicago), ISSN 2168-6149, 08/2018, Volume 75, Issue 8, p. 1027
Journal Article
Expert review of neurotherapeutics, ISSN 1473-7175, 10/2011, Volume 11, Issue 10, pp. 1485 - 1496
metabolic genetic disease | degenerative brain disease | brain white matter | children | MRI | Pharmacology & Pharmacy | Clinical Neurology | Neurosciences & Neurology | Life Sciences & Biomedicine | Science & Technology | Neurodegenerative Diseases - pathology | Leukodystrophy, Globoid Cell - diagnosis | Leukodystrophy, Metachromatic - physiopathology | Humans | Leukodystrophy, Globoid Cell - pathology | Neurodegenerative Diseases - diagnosis | Infant | Leukodystrophy, Metachromatic - therapy | Neurodegenerative Diseases - therapy | Leukodystrophy, Globoid Cell - physiopathology | Magnetic Resonance Imaging | Leukodystrophy, Metachromatic - diagnosis | Neurodegenerative Diseases - physiopathology | Nerve Fibers, Myelinated - pathology | Leukodystrophy, Globoid Cell - therapy | Child | Leukodystrophy, Metachromatic - pathology | Infant, Newborn | Care and treatment | Usage | Magnetic resonance imaging | Children | Diagnosis | Research | Health aspects | Leukoencephalopathy | Index Medicus
Journal Article
Genetics in medicine, ISSN 1098-3600, 12/2016, Volume 18, Issue 12, pp. 1235 - 1243
newborn screening | Krabbe disease | Globoid cell leukodystrophy | Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | New York | Leukodystrophy, Globoid Cell - diagnosis | Humans | Risk Factors | Infant | Neonatal Screening | Mass Screening | Hematopoietic Stem Cell Transplantation - adverse effects | Female | Leukodystrophy, Globoid Cell - genetics | Leukodystrophy, Globoid Cell - mortality | Leukodystrophy, Globoid Cell - therapy | Infant, Newborn | Index Medicus
Journal Article
Gene, ISSN 0378-1119, 01/2015, Volume 555, Issue 1, pp. 2 - 13
β-Galactocerebrosidase | Sphingolipid | Lysosomal storage disease | Myelin | Globoid cell leukodystrophy | Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Animals | History, 20th Century | Humans | Leukodystrophy, Globoid Cell - history | History, 19th Century | Leukodystrophy, Globoid Cell - genetics | Leukodystrophy, Globoid Cell - therapy | Leukodystrophy, Globoid Cell - physiopathology | Index Medicus
Journal Article
The Journal of neuroscience, ISSN 0270-6474, 04/2015, Volume 35, Issue 16, pp. 6495 - 6505
Gene therapy | Lysosomal storage disease | Dysmyelination | Krabbe disease | Globoid cell leukodystrophy | Neurosciences | Neurosciences & Neurology | Life Sciences & Biomedicine | Science & Technology | Genetic Therapy | Motor Skills - drug effects | Body Weight - drug effects | Myelin Sheath - drug effects | Male | Body Weight - genetics | Brain - metabolism | Sciatic Nerve - metabolism | Galactosylceramidase - genetics | Bone Marrow Transplantation | Female | Leukodystrophy, Globoid Cell - therapy | Myelin Sheath - physiology | Cytokines - metabolism | Psychosine - metabolism | Leukodystrophy, Globoid Cell - pathology | Combined Modality Therapy | Leukodystrophy, Globoid Cell - drug therapy | Mice, Inbred Strains | Cycloserine - therapeutic use | Brain - drug effects | Animals | Leukodystrophy, Globoid Cell - genetics | Mice | Galactosylceramidase - metabolism | Index Medicus | gene therapy | globoid cell leukodystrophy | dysmyelination | lysosomal storage disease
Journal Article
Science (American Association for the Advancement of Science), ISSN 0036-8075, 8/2013, Volume 341, Issue 6148, pp. 864 - 864
RESEARCH ARTICLE SUMMARY | Metachromatic leukodystrophy | Nervous system diseases | Neurodegenerative diseases | Demyelinating diseases | Medical treatment | Gene therapy | Hematopoietic stem cells | Image contrast | Science & Technology - Other Topics | Multidisciplinary Sciences | Science & Technology | Transduction, Genetic | Follow-Up Studies | Humans | Hematopoietic Stem Cell Transplantation | Leukodystrophy, Metachromatic - therapy | Treatment Outcome | Hematopoietic Stem Cells - metabolism | Virus Integration | Lentivirus | Magnetic Resonance Imaging | Genetic Engineering | Brain - pathology | Genetic Vectors - toxicity | DNA Damage | Leukodystrophy, Metachromatic - pathology | Cerebroside-Sulfatase - genetics | Genetic Therapy - methods | Index Medicus
Journal Article
Glia, ISSN 0894-1491, 06/2019, Volume 67, Issue 6, pp. 1196 - 1209
metachromatic leukodystrophy | X‐linked adrenoleukodystrophy | cell death | microglia | demyelination | X-linked adrenoleukodystrophy | Neurosciences | Neurosciences & Neurology | Life Sciences & Biomedicine | Science & Technology | Microglia - metabolism | Myelin Sheath - pathology | Humans | Middle Aged | Adrenoleukodystrophy - pathology | Child, Preschool | Male | Myelin Sheath - metabolism | Adrenoleukodystrophy - metabolism | Leukodystrophy, Metachromatic - genetics | Leukodystrophy, Metachromatic - metabolism | Myelin Sheath - genetics | Adolescent | Microglia - pathology | Adult | Female | Aged | Adrenoleukodystrophy - genetics | Child | Leukodystrophy, Metachromatic - pathology | Cohort Studies | Sphingolipidoses | Transplantation | Cell death | Hematopoietic stem cells | Stem cells | Pathogenesis | Stem cell transplantation | Lipids | Demyelination | Autopsy | Oligodendrocytes | DNA fragmentation | Degeneration | Lesions | Deoxyribonucleic acid--DNA | Phenotypes | Autopsies | Demyelinating diseases | Myelin | Leukodystrophy | Atomic layer epitaxy | Substantia alba | Substrates | Microglia | Hemopoiesis | Diseases | Adrenoleukodystrophy | Morphology | Apoptosis | Index Medicus
Journal Article
Human molecular genetics, ISSN 0964-6906, 2015, Volume 24, Issue 12, pp. 3372 - 3389
Biochemistry & Molecular Biology | Genetics & Heredity | Life Sciences & Biomedicine | Science & Technology | Genetic Therapy | Central Nervous System - metabolism | Leukodystrophy, Globoid Cell - diagnosis | Humans | Apoptosis - genetics | Neural Stem Cells - cytology | Myelin Sheath - metabolism | Brain - metabolism | Gliosis - pathology | Stem Cell Transplantation | Galactosylceramidase - genetics | Leukodystrophy, Globoid Cell - metabolism | Bone Marrow Transplantation | Cell Differentiation | Leukodystrophy, Globoid Cell - mortality | Leukodystrophy, Globoid Cell - therapy | Disease Models, Animal | Gliosis - genetics | Gliosis - metabolism | Peripheral Nervous System - physiopathology | Axons - metabolism | Graft Survival | Mice, Transgenic | Cell- and Tissue-Based Therapy | Mice, Knockout | Animals | Axons - pathology | Peripheral Nervous System - metabolism | Leukodystrophy, Globoid Cell - genetics | Mice | Central Nervous System - physiopathology | Enzyme Activation | Neural Stem Cells - metabolism | Galactosylceramidase - metabolism | Index Medicus
Journal Article