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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
Human mutation, ISSN 1059-7794, 01/2016, Volume 37, Issue 1, pp. 16 - 27
metachromatic leukodystrophy | ARSA | mutation | arylsulfatase A | saposin B | PSAP | Metachromatic leukodystrophy | Arylsulfatase A | Mutation | Saposin B | Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Leukodystrophy, Metachromatic - genetics | Phenotype | Leukodystrophy, Metachromatic - diagnosis | Genetic Association Studies | Humans | Alleles | Databases, Genetic | Saposins - genetics | Genotype | Cerebroside-Sulfatase - genetics | Sphingolipidoses | Enzymes | Nervous system diseases | Genetic aspects | Genes | Proteins | Genetic disorders | Cells | Index Medicus
Journal Article
Annals of neurology, ISSN 0364-5134, 02/2018, Volume 83, Issue 2, pp. 418 - 432
Neurosciences | Clinical Neurology | Neurosciences & Neurology | Life Sciences & Biomedicine | Science & Technology | Gene Transfer Techniques | Neuroprotective Agents | Animals | Humans | Mice, Inbred C57BL | Mice, Transgenic | Mice | Lysosomal Storage Diseases - pathology | Metallothionein | Neuroprotection | Oxidative stress | Animal models | Disease | Central nervous system | Disorders | Lysosomal storage diseases | Neuroprotective agents | Proteins | Purkinje cells | Lysosomal enzymes | Rodents | Degeneration | Phenotypes | Gene transfer | Transgenic mice | Pharmacology | Gene expression | Chemical compounds | Survival | Microglia | Storage | Metallothioneins | Metabolic disorders | Apoptosis | Index Medicus
Journal Article
Blood, ISSN 0006-4971, 05/2011, Volume 117, Issue 20, pp. 5332 - 5339
Life Sciences & Biomedicine | Hematology | Science & Technology | Hematologic and hematopoietic diseases | Biological and medical sciences | Medical sciences | Gene Transfer Techniques | Virus Integration - genetics | Humans | Hematopoietic Stem Cell Transplantation | Clinical Trials as Topic | DNA-Binding Proteins - genetics | Interleukin Receptor Common gamma Subunit - genetics | DNA-Binding Proteins - deficiency | Mice, Knockout | Transplantation Chimera - genetics | Interleukin Receptor Common gamma Subunit - deficiency | Animals | Adrenoleukodystrophy - therapy | Lentivirus - genetics | Mice | Adrenoleukodystrophy - genetics | Genetic Vectors | Genetic Therapy - methods | Index Medicus | Abridged Index Medicus
Journal Article
Annals of neurology, ISSN 0364-5134, 02/2018, Volume 83, Issue 2, pp. 418 - 432
Journal Article
Annals of neurology, ISSN 0364-5134, 01/2014, Volume 75, Issue 1, pp. 127 - 137
Journal Article
Current gene therapy, ISSN 1566-5232, 04/2008, Volume 8, Issue 2, pp. 135 - 146
Transduction | Ex vivo manipulation | Gene therapy | Hematopoietic stem cells | Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Viruses - genetics | Hematopoietic Stem Cells - cytology | Humans | Hematopoietic Stem Cells - metabolism | Transduction, Genetic - methods | Genetic Vectors - genetics | Genetic Therapy - methods
Journal Article
Human mutation, ISSN 1059-7794, 01/2016, Volume 37, Issue 1, pp. 16 - 27
Journal Article
Gene, ISSN 0378-1119, 03/2014, Volume 537, Issue 2, pp. 348 - 351
Arylsulfatase A | Metachromatic Leukodystrophy | Molecular diagnosis | Enzymatic activity | Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Leukodystrophy, Metachromatic - genetics | Leukodystrophy, Metachromatic - diagnosis | Humans | Alleles | Female | Heterozygote | Infant | Male | Cerebroside-Sulfatase - blood | Cerebroside-Sulfatase - genetics | Sphingolipidoses | Enzymes | Diagnosis | Analysis | Index Medicus
Journal Article