Leukodystrophy, Metachromatic

Known as: leukodystrophy metachromatic, Arylsulfatase A Deficiency Disease, Metachromatic Leukoencephalopathies 
An autosomal recessive inherited disorder characterized by abnormalities in the development of the myelin sheaths. It is caused by a deficiency of… (More)
National Institutes of Health

Related topics

Related topics

46 relations
ARSA geneARSA wt AlleleAbnormal behaviorArylsulfatases
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Narrower (8)

Arylsulfatase A DeficiencyCerebral Sclerosis, Diffuse, Scholz TypeCombined Saposin DeficiencyMetachromatic Leukodystrophy due to Saposin B Deficiency
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Broader (5)

Enzyme DeficiencyLeukodystrophyLysosomal Storage DiseasesSulfatidosis
(More)

Papers overview

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Highly Cited
2013
Highly Cited
2013
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disease caused by arylsulfatase A (ARSA) deficiency… (More)
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Highly Cited
2011
Highly Cited
2011
Impaired acuity of the approximate number system underlies mathematical learning disability (dyscalculia).
Many children have significant mathematical learning disabilities (MLD, or dyscalculia) despite adequate schooling. The current… (More)
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Highly Cited
2007
Highly Cited
2007
Cognitive mechanisms underlying achievement deficits in children with mathematical learning disability.
Using strict and lenient mathematics achievement cutoff scores to define a learning disability, respective groups of children who… (More)
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Highly Cited
2006
Highly Cited
2006
Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.
Metachromatic leukodystrophy (MLD) is a demyelinating lysosomal storage disorder for which new treatments are urgently needed. We… (More)
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Highly Cited
2004
Highly Cited
2004
Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.
Gene-based delivery can establish a sustained supply of therapeutic proteins within the nervous system. For diseases… (More)
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Review
2003
Review
2003
Globoid cell leukodystrophy (Krabbe's disease): update.
The classic globoid cell leukodystrophy (Krabbe's disease) is caused by genetic defects in a lysosomal enzyme… (More)
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Highly Cited
2002
Highly Cited
2002
Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH)
Patients with Hurler syndrome (mucopolysaccharidosis type-IH) and metachromatic leukodystrophy (MLD) develop significant skeletal… (More)
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Highly Cited
2001
Highly Cited
2001
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice
Metachromatic leukodystrophy (MLD) is a lipidosis caused by deficiency of arylsulfatase A (ARSA). Although the genetics of MLD… (More)
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Review
1999
Review
1999
Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis…
Over 400 patients with lysosomal and peroxisomal storage diseases have received hematopoietic stem cell transplantation from… (More)
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Highly Cited
1996
Highly Cited
1996
Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy.
Metachromatic leukodystrophy is a lysosomal sphingolipid storage disorder caused by the deficiency of arylsulfatase A. The… (More)
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