Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII. (Q39652181)

13 September 2017

Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII. (English)

1 reference

13 September 2017

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Julie Bielicki

1

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PubMed ID

20864369

13 September 2017

Chantelle McIntyre

series ordinal

2

1 reference

PubMed ID

20864369

13 September 2017

Donald S Anson

series ordinal

3

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13 September 2017

17 August 2010

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Molecular Genetics and Metabolism

13 September 2017

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13 September 2017

101

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13 September 2017

4

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370-382

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Prevalence of lysosomal storage disorders

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The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update

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Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse

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Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA.

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

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Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier

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Epinephrine enhances lysosomal enzyme delivery across the blood brain barrier by up-regulation of the mannose 6-phosphate receptor

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Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

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Clearance of albumin from mouse subarachnoid space: a measure of CSF bulk flow

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Supratentorial cerebrospinal fluid production rate in healthy adults: quantification with two-dimensional cine phase-contrast MR imaging with high temporal and spatial resolution.

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Regulation of cerebrospinal fluid production by caffeine consumption

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Reduced cerebrospinal fluid production and intracranial pressure in mice lacking choroid plexus water channel Aquaporin-1.

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Evidence for bulk flow of brain interstitial fluid: significance for physiology and pathology

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Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

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Targeted nonviral delivery vehicles to neural progenitor cells in the mouse subventricular zone

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Single-dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophy

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Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-defici

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Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector

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A rapid sensitive silver stain for polypeptides in polyacrylamide gels

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Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA.

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Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease

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Gene therapy for murine mucopolysaccharidosis type VII.

7 January 2021

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Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation

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Eosinophilic crystalline pneumonia as a major cause of death in 129S4/SvJae mice

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Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

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A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease

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Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs

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Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

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Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice

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PEGylation of a vesicular stomatitis virus G pseudotyped lentivirus vector prevents inactivation in serum

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Selective depletion or blockade of Kupffer cells leads to enhanced and prolonged hepatic transgene expression using high-capacity adenoviral vectors

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Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

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Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene

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Ultrastructure of the orbital pathway for cerebrospinal fluid drainage in rabbits

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Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

7 January 2021

1 reference

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Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector

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Targeted delivery of proteins across the blood-brain barrier

7 January 2021

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10.1016/J.YMGME.2010.08.013

7 January 2021

Identifiers

DOI

1 reference

13 September 2017

1 reference

PubMed ID

20864369