Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII. (Q44564325)

4 December 2017

Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII. (English)

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4 December 2017

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Brian A Karolewski

1

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PubMed ID

16624622

4 December 2017

John H Wolfe

series ordinal

2

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4 December 2017

19 April 2006

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4 December 2017

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4 December 2017

14

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4 December 2017

14-24

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4 December 2017

1

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Prevalence of lysosomal storage disorders

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Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

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Mucopolysaccharidosis type VII in the developing mouse fetus

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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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Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain

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In utero fetal liver cell transplantation without toxic irradiation alleviates lysosomal storage in mice with mucopolysaccharidosis type VII

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In utero transplantation of fetal liver cells in the mucopolysaccharidosis type VII mouse results in low-level chimerism, but overexpression of beta-glucuronidase can delay onset of clinical signs

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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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Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase

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1 reference

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Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream.

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

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Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease

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Determination of oligosaccharides and glycolipids in amniotic fluid by electrospray ionisation tandem mass spectrometry: in utero indicators of lysosomal storage diseases

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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

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Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

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

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

7 January 2021

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Transduction of the choroid plexus and ependyma in neonatal mouse brain by vesicular stomatitis virus glycoprotein-pseudotyped lentivirus and adeno-associated virus type 5 vectors

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Retinal degeneration mutants in the mouse

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

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Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

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

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Enzyme replacement therapy for murine mucopolysaccharidosis type VII

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No evidence for germ-line transmission following prenatal and early postnatal AAV-mediated gene delivery.

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Intracerebral transplantation of mesenchymal stem cells into acid sphingomyelinase-deficient mice delays the onset of neurological abnormalities and extends their life span.

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

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

7 January 2021

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Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

7 January 2021

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Long-term gene transfer to mouse fetuses with recombinant adenovirus and adeno-associated virus (AAV) vectors

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In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression.

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10.1016/J.YMTHE.2006.02.012

7 January 2021

Identifiers

DOI

1 reference

4 December 2017

1 reference

PubMed ID

16624622