Gene therapy for metachromatic leukodystrophy. (Q30357745)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27638601%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

review article

1 reference

title

Gene therapy for metachromatic leukodystrophy (English)

1 reference

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23 March 2020

0 references

2

1 reference

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23 March 2020

author name string

Jonathan B Rosenberg

1

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23 March 2020

Patrick Aubourg

3

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23 March 2020

Ronald G Crystal

4

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23 March 2020

Dolan Sondhi

5

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23 March 2020

publication date

1 November 2016

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Journal of Neuroscience Research

23 March 2020

published in

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23 March 2020

volume

94

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23 March 2020

issue

11

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23 March 2020

page(s)

1169-1179

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Viral vectors for therapy of neurologic diseases

23 March 2020

cites work

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Mutation Update of ARSA and PSAP Genes Causing Metachromatic Leukodystrophy

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Outcome of Early Juvenile Onset Metachromatic Leukodystrophy After Unrelated Cord Blood Transplantation: A Case Series and Review of the Literature.

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Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates

3 June 2018

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Disease specific therapies in leukodystrophies and leukoencephalopathies

3 June 2018

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A clinical approach to the diagnosis of patients with leukodystrophies and genetic leukoencephelopathies

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Case definition and classification of leukodystrophies and leukoencephalopathies

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3 June 2018

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Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial

3 June 2018

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Humoral Immune Response to AAV.

3 June 2018

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Developing therapeutic approaches for metachromatic leukodystrophy

3 June 2018

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Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

3 June 2018

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Blood-brain barrier structure and function and the challenges for CNS drug delivery

3 June 2018

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Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.

3 June 2018

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Immunologic privilege in the central nervous system and the blood-brain barrier

3 June 2018

1 reference

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Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease

3 June 2018

1 reference

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Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.

3 June 2018

1 reference

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Role of sulfatide in normal and pathological cells and tissues

3 June 2018

1 reference

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The AAV vector toolkit: poised at the clinical crossroads

3 June 2018

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Developing treatment options for metachromatic leukodystrophy.

3 June 2018

1 reference

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Glial-derived neurotrophic factor gene transfer for Parkinson's disease: anterograde distribution of AAV2 vectors in the primate brain

3 June 2018

1 reference

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Haematopoietic stem cell transplantation does not retard disease progression in the psycho-cognitive variant of late-onset metachromatic leukodystrophy

3 June 2018

1 reference

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Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature.

3 June 2018

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3 June 2018

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Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations.

3 June 2018

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Metachromatic leukodystrophy--an update

3 June 2018

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Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates.

3 June 2018

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Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy

3 June 2018

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Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain.

3 June 2018

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Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis

3 June 2018

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Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

3 June 2018

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Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

3 June 2018

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Enzyme, cell and gene-based therapies for metachromatic leukodystrophy

3 June 2018

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

3 June 2018

1 reference

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Reflux-free cannula for convection-enhanced high-speed delivery of therapeutic agents

3 June 2018

1 reference

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New recombinant serotypes of AAV vectors.

3 June 2018

1 reference

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

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5027970

Gene therapy of metachromatic leukodystrophy

3 June 2018

1 reference

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

3 June 2018

1 reference

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Recombinant AAV-mediated gene delivery to the central nervous system.

3 June 2018

1 reference

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Nerve growth factor: from animal models of cholinergic neuronal degeneration to gene therapy in Alzheimer's disease

3 June 2018

1 reference

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Metachromatic Leukodystrophy: Recent Research Developments

3 June 2018

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Mannose 6-phosphate receptors: new twists in the tale

3 June 2018

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

3 June 2018

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Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

3 June 2018

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Turning blood into brain: cells bearing neuronal antigens generated in vivo from bone marrow

3 June 2018

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From marrow to brain: expression of neuronal phenotypes in adult mice.

3 June 2018

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Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome

3 June 2018

1 reference

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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

3 June 2018

1 reference

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Adeno-associated virus-mediated gene transfer to the brain: duration and modulation of expression

3 June 2018

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Gene transfer by adeno-associated virus vectors into the central nervous system

3 June 2018

1 reference

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Hematopoietic cells differentiate into both microglia and macroglia in the brains of adult mice

3 June 2018

1 reference

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

3 June 2018

1 reference

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Morphologic study of the internalization of a lysosomal enzyme by the mannose 6-phosphate receptor in cultured Chinese hamster ovary cells

3 June 2018

1 reference

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Detection of a point mutation in sphingolipid activator protein-1 mRNA in patients with a variant form of metachromatic leukodystrophy

3 June 2018

1 reference

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Sulfatide activator protein. Alternative splicing that generates three mRNAs and a newly found mutation responsible for a clinical disease

3 June 2018

1 reference

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Structure of the arylsulfatase A gene

3 June 2018

1 reference

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Insertion in the mRNA of a metachromatic leukodystrophy patient with sphingolipid activator protein-1 deficiency

3 June 2018

1 reference

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Expression of the insulin-like growth factor-II/mannose-6-phosphate receptor in multiple human tissues during fetal life and early infancy

3 June 2018

1 reference

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Terminal 22q deletion associated with a partial deficiency of arylsulphatase A

3 June 2018

1 reference

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Metachromatic leukodystrophy - mutation analysis provides further evidence of genotype-phenotype correlation

3 June 2018

1 reference

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

21 January 2018

1 reference

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system

21 January 2018

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Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

21 January 2018

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Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution

21 January 2018

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Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate.

21 January 2018

1 reference

Metachromatic leukodystrophy: natural course of cerebral MRI changes in relation to clinical course

21 January 2018

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Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response

21 January 2018

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

21 January 2018

1 reference

Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy

21 January 2018

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Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age.

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Immune responses to AAV in a phase I study for Canavan disease

21 January 2018

1 reference

Accumulation of sulfatide in neuronal and glial cells of arylsulfatase A deficient mice

21 January 2018

1 reference

Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.

21 January 2018

1 reference

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

21 January 2018

1 reference

AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy

21 January 2018

1 reference

Late-onset metachromatic leukodystrophy: genotype strongly influences phenotype

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

21 January 2018

1 reference

Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain

21 January 2018

1 reference

Adeno-associated virus vector-mediated transduction in the cat brain

21 January 2018

1 reference

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

21 January 2018

1 reference

Improvement of White Matter Changes on Neuroimaging Modalities After Stem Cell Transplant in Metachromatic Leukodystrophy

21 January 2018

1 reference

CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5027970

Occurrence, distribution, and phenotype of arylsulfatase A mutations in patients with metachromatic leukodystrophy

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5027970

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

26 November 2018

1 reference

12 December 2020

Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy

1 reference

12 December 2020

Sulfatides trigger cytokine gene expression and secretion in human monocytes

1 reference

12 December 2020

Gallbladder polyposis in metachromatic leukodystrophy

1 reference

12 December 2020

Metachromatic leukodystrophy: molecular genetics and an animal model

1 reference

12 December 2020

Identifiers

DOI

10.1002/JNR.23792

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27638601%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27638601%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

PubMed publication ID

27638601

1 reference