(Q45867578)

17 December 2017

title

Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice (English)

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17 December 2017

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Fu H

1

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17 December 2017

Kang L

2

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17 December 2017

Jennings JS

3

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17 December 2017

Moy SS

4

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17 December 2017

Perez A

5

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17 December 2017

Dirosario J

6

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17 December 2017

McCarty DM

7

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17 December 2017

Muenzer J

8

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17 December 2017

publication date

26 April 2007

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17 December 2017

published in

Gene Therapy

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17 December 2017

volume

14

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17 December 2017

issue

14

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17 December 2017

page(s)

1065-1077

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17 December 2017

https://scigraph.springernature.com/pub.10.1038/sj.gt.3302961

exact match

0 references

cites work

Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase

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7 January 2021

Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders

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7 January 2021

Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres

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7 January 2021

Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome.

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7 January 2021

Enzyme replacement therapy for the mucopolysaccharide storage disorders

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7 January 2021

Sanfilippo type B syndrome (mucopolysaccharidosis III B): allelic heterogeneity corresponds to the wide spectrum of clinical phenotypes

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7 January 2021

Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts

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7 January 2021

Quantitative correlation between the residual activity of beta-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease

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7 January 2021

Simulation of hematopoiesis: implications for the gene therapy of lysosomal enzyme disorders

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7 January 2021

The cryptic life style of adeno-associated virus

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Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain

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Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery

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7 January 2021

Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible.

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7 January 2021

Prevention of neuropathology in the mouse model of Hurler syndrome

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7 January 2021

Drug and gene delivery to the brain: the vascular route

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7 January 2021

Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier

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7 January 2021

Targeted retrograde gene delivery for neuronal protection

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7 January 2021

AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease

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7 January 2021

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

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7 January 2021

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene

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7 January 2021

Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector

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7 January 2021

Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs

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7 January 2021

Osmotic opening of the blood-brain barrier: principles, mechanism, and therapeutic applications

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7 January 2021

Osmotic blood-brain barrier disruption chemotherapy for diffuse pontine gliomas

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7 January 2021

Primary CNS lymphoma treated with osmotic blood-brain barrier disruption: prolonged survival and preservation of cognitive function.

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7 January 2021

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

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7 January 2021

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

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7 January 2021

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

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7 January 2021

AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.

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7 January 2021

Effective gene therapy in an authentic model of Tay-Sachs-related diseases

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7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

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7 January 2021

Enzyme replacement therapy in alpha-mannosidosis guinea-pigs.

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7 January 2021

Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-ac

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302961

7 January 2021

Purification and characterization of recombinant human alpha-N-acetylglucosaminidase secreted by Chinese hamster ovary cells

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302961

7 January 2021

Expression and characterization of human recombinant and alpha-N-acetylglucosaminidase

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302961

7 January 2021

The lysosomal trafficking of acid sphingomyelinase is mediated by sortilin and mannose 6-phosphate receptor

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7 January 2021

Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice

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7 January 2021

Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

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7 January 2021

Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302961

7 January 2021

The molecular basis of Sanfilippo syndrome type B

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302961

7 January 2021

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302961

7 January 2021

Quantification and characterization of glycosaminoglycans at the nanogram level by a combined azure A-silver staining in agarose gels

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302961

7 January 2021

Social interaction and sensorimotor gating abnormalities in mice lacking Dvl1

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302961

7 January 2021

The conjoint importance of the hippocampus and anterior thalamic nuclei for allocentric spatial learning: evidence from a disconnection study in the rat.

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302961

7 January 2021

Identifiers

DOI

10.1038/SJ.GT.3302961

1 reference

17 December 2017

Dimensions Publication ID

0 references

1 reference

17 December 2017