(Q45865114)

17 December 2017

title

Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow (English)

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17 December 2017

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mucopolysaccharidosis I

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4

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17 December 2017

Elizabeth F. Neufeld

6

object named as

Elizabeth F Neufeld

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17 December 2017

author name string

Yi Zheng

1

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17 December 2017

Nora Rozengurt

2

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17 December 2017

Sergey Ryazantsev

3

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17 December 2017

Noriko Satake

5

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17 December 2017

publication date

1 August 2003

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17 December 2017

Molecular Genetics and Metabolism

published in

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17 December 2017

volume

79

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17 December 2017

issue

4

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17 December 2017

page(s)

233-244

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17 December 2017

The human gene mutation database

cites work

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https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

Molecular genetics of mucopolysaccharidosis type I: diagnostic, clinical, and biological implications.

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The defect in Hurler and Hunter syndromes. II. Deficiency of specific factors involved in mucopolysaccharide degradation

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7 January 2021

The defect in the Hurler and Scheie syndromes: deficiency of -L-iduronidase

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7 January 2021

Recognition and receptor-mediated uptake of a lysosomal enzyme, alpha-l-iduronidase, by cultured human fibroblasts

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7 January 2021

Enzyme replacement in a canine model of Hurler syndrome

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7 January 2021

Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

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7 January 2021

Enzyme replacement therapy in feline mucopolysaccharidosis I.

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7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

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7 January 2021

Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation

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7 January 2021

Long-term clinical progress in bone marrow transplanted mucopolysaccharidosis type I patients with a defined genotype

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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

Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases

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7 January 2021

Correction of murine galactosialidosis by bone marrow-derived macrophages overexpressing human protective protein/cathepsin A under control of the colony-stimulating factor-1 receptor promoter

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7 January 2021

Retrovirus-mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts

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https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.

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7 January 2021

In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression

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7 January 2021

Murine mucopolysaccharidosis VIL: impact of therapies on the phenotype, clinical course, and pathology in a model of a lysosomal storage disease

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7 January 2021

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

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7 January 2021

Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene

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7 January 2021

Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells.

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7 January 2021

A transient three-plasmid expression system for the production of high titer retroviral vectors

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https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells

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https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors

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https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

A safe packaging line for gene transfer: separating viral genes on two different plasmids

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https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells

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https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

Direct demonstration of binding of a lysosomal enzyme, alpha-L-iduronidase, to receptors on cultured fibroblasts

1 reference

https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells

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https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

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

Long-term outcome of Hurler syndrome following bone marrow transplantation.

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7 January 2021

The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice

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7 January 2021

Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

1 reference

https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

1 reference

https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors

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https://api.crossref.org/works/10.1016%2FS1096-7192%2803%2900116-1

7 January 2021

10.1016/S1096-7192(03)00116-1

Identifiers

DOI

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17 December 2017

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17 December 2017