(Q28292170)

Crossref

https://api.crossref.org/works/10.1007%2FS10545-007-0540-Z

Ex vivo gene therapy using bone marrow-derived cells: combined effects of intracerebral and intravenous transplantation in a mouse model of Niemann-Pick disease

21 January 2018

1 reference

12 December 2020

Delay of myelin formation in arylsulphatase A-deficient mice

1 reference

12 December 2020

Targeting gene-modified hematopoietic cells to the central nervous system: use of green fluorescent protein uncovers microglial engraftment.

1 reference

12 December 2020

Oligodendroglial progenitor cell therapy limits central neurological deficits in mice with metachromatic leukodystrophy.

1 reference

12 December 2020

Gangliosides as apoptotic signals in ER stress response

1 reference

12 December 2020

Postnatal development of inflammation in a murine model of Niemann-Pick type C disease: immunohistochemical observations of microglia and astroglia

1 reference

12 December 2020

Hyperactivity, neuromotor defects, and impaired learning and memory in a mouse model for metachromatic leukodystrophy

1 reference

12 December 2020

Multiple sulfatase deficiency is caused by mutations in the gene encoding the human C(alpha)-formylglycine generating enzyme

1 reference

12 December 2020

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

1 reference

12 December 2020

Molecular basis for multiple sulfatase deficiency and mechanism for formylglycine generation of the human formylglycine-generating enzyme

1 reference

12 December 2020

Sulfatides trigger increase of cytosolic free calcium and enhanced expression of tumor necrosis factor-alpha and interleukin-8 mRNA in human neutrophils. Evidence for a role of L-selectin as a signaling molecule

1 reference

12 December 2020

Enzyme replacement therapy in a mouse model of aspartylglycosaminuria

1 reference

12 December 2020

The multiple sulfatase deficiency gene encodes an essential and limiting factor for the activity of sulfatases

1 reference

12 December 2020

Coexpression of formylglycine-generating enzyme is essential for synthesis and secretion of functional arylsulfatase A in a mouse model of metachromatic leukodystrophy

1 reference

12 December 2020

Activation of non-neuronal cells within the prenatal developing brain of sheep with neuronal ceroid lipofuscinosis

1 reference

12 December 2020

Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer

1 reference

12 December 2020

Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy

1 reference

12 December 2020

Sulfatides trigger cytokine gene expression and secretion in human monocytes

1 reference

12 December 2020

Ex vivo cell-mediated gene therapy for metachromatic leukodystrophy using neurospheres.

1 reference

12 December 2020

Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery.

1 reference

12 December 2020

Galactocerebrosidase-deficient oligodendrocytes maintain stable central myelin by exogenous replacement of the missing enzyme in mice

1 reference

12 December 2020

Disialoganglioside GD3 is released by microglia and induces oligodendrocyte apoptosis

1 reference

12 December 2020

1 reference

12 December 2020

Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector

1 reference

12 December 2020

AAV1 mediated co-expression of formylglycine-generating enzyme and arylsulfatase a efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy

1 reference

12 December 2020

Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH).

1 reference

12 December 2020

Decline in brainstem auditory-evoked potentials coincides with loss of spiral ganglion cells in arylsulfatase A-deficient mice

1 reference

12 December 2020

The cell biology of lysosomal storage disorders

1 reference

12 December 2020

Neuromotor alterations and cerebellar deficits in aged arylsulfatase A-deficient transgenic mice

1 reference

12 December 2020

Metachromatic leukodystrophy: consequences of sulphatide accumulation.

1 reference

12 December 2020

MR of childhood metachromatic leukodystrophy.

1 reference

12 December 2020

Storage solutions: treating lysosomal disorders of the brain

1 reference

12 December 2020

Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy

1 reference

12 December 2020

Atomic force microscopy of nonhydroxy galactocerebroside nanotubes and their self-assembly at the air-water interface, with applications to myelin

1 reference

12 December 2020

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

1 reference

12 December 2020

Neurodegeneration augments the ability of bone marrow-derived mesenchymal stem cells to fuse with Purkinje neurons in Niemann-Pick type C mice

1 reference

12 December 2020

Defective calcium homeostasis in the cerebellum in a mouse model of Niemann-Pick A disease

1 reference

12 December 2020

Cellular pathology of lysosomal storage disorders.

1 reference

12 December 2020

Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

1 reference

12 December 2020

Gene therapy of the brain in the dog model of Hurler's syndrome.

1 reference

12 December 2020

Embryonic stem cell-based reduction of central nervous system sulfatide storage in an animal model of metachromatic leukodystrophy.

1 reference

12 December 2020

Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy

1 reference

12 December 2020

Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy.

1 reference

12 December 2020

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

1 reference

12 December 2020

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

1 reference

12 December 2020

Human CD34+ cells differentiate into microglia and express recombinant therapeutic protein

1 reference

12 December 2020

Enzyme replacement and enhancement therapies: lessons from lysosomal disorders

1 reference

12 December 2020

Hippocampal pathology in the human neuronal ceroid-lipofuscinoses: distinct patterns of storage deposition, neurodegeneration and glial activation

1 reference

12 December 2020

Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

1 reference

12 December 2020

Gene therapy progress and prospects: gene therapy of lysosomal storage disorders

1 reference

12 December 2020

Cerebroside synthesis as a measure of the rate of remyelination following cuprizone-induced demyelination in brain

1 reference

12 December 2020

Lysosomal sulfatide storage in the brain of arylsulfatase A-deficient mice: cellular alterations and topographic distribution

1 reference

12 December 2020

Specific downregulation and mistargeting of the lipid raft-associated protein MAL in a glycolipid storage disorder

1 reference

12 December 2020

Structure of the arylsulfatase A gene

1 reference

12 December 2020

The status of hematopoietic stem cell transplantation in lysosomal storage disease

1 reference

12 December 2020

Sulfatide is essential for the maintenance of CNS myelin and axon structure

1 reference

12 December 2020

Reduced brain cholesterol content in arylsulfatase A-deficient mice

1 reference

12 December 2020

The role of sphingolipids in neuronal development: lessons from models of sphingolipid storage diseases

1 reference

12 December 2020

Marked elevation of the chemokine CCL18/PARC in Gaucher disease: a novel surrogate marker for assessing therapeutic intervention

1 reference

12 December 2020

Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders.

1 reference

12 December 2020

Late-onset metachromatic leukodystrophy: genotype strongly influences phenotype

1 reference

12 December 2020

Gene therapy: can neural stem cells deliver?

1 reference

12 December 2020

Viral vectors for gene delivery to the nervous system

1 reference

12 December 2020

The therapeutic potential of neural stem cells

1 reference

12 December 2020

Efficacy of enzyme replacement therapy in alpha-mannosidosis mice: a preclinical animal study

1 reference

12 December 2020

New perspectives on the function of myelin galactolipids.

1 reference

12 December 2020

Metachromatic leukodystrophy: molecular genetics and an animal model

1 reference

12 December 2020

Human embryonic stem cell-derived oligodendrocyte progenitors for the treatment of spinal cord injury

1 reference

12 December 2020

Regional and cellular neuropathology in the palmitoyl protein thioesterase-1 null mutant mouse model of infantile neuronal ceroid lipofuscinosis

1 reference

12 December 2020

The pathogenesis of glycosphingolipid storage disorders

1 reference

12 December 2020

GM1-ganglioside-mediated activation of the unfolded protein response causes neuronal death in a neurodegenerative gangliosidosis

1 reference

12 December 2020

Prevention of neuropathology in the mouse model of Hurler syndrome

1 reference

12 December 2020

Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations

1 reference

12 December 2020

The therapeutic potential of neural stem/progenitor cells in murine globoid cell leukodystrophy is conditioned by macrophage/microglia activation

1 reference

12 December 2020

Plasma chitotriosidase and CCL18: early biochemical surrogate markers in type B Niemann-Pick disease

1 reference

12 December 2020

Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and S

1 reference

12 December 2020