(Q45178229)

10 December 2017

Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice (English)

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10 December 2017

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mucopolysaccharidosis I

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Yuli Liu

1

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10 December 2017

Lingfei Xu

2

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10 December 2017

Anne K Hennig

3

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10 December 2017

Attila Kovacs

4

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10 December 2017

Annabel Fu

5

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10 December 2017

Sarah Chung

6

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10 December 2017

David Lee

7

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10 December 2017

Bin Wang

8

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10 December 2017

Ramin S Herati

9

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10 December 2017

Judith Mosinger Ogilvie

10

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10 December 2017

Shi-Rong Cai

11

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10 December 2017

Katherine Parker Ponder

12

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10 December 2017

language of work or name

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1 January 2005

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10 December 2017

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10 December 2017

11

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10 December 2017

1

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10 December 2017

35-47

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Prevalence of lysosomal storage disorders

10 December 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Enzyme replacement and enhancement therapies for lysosomal diseases

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Gene therapy progress and prospects: gene therapy of lysosomal storage disorders

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Retinal function is improved in a murine model of a lysosomal storage disease following bone marrow transplantation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

An update on the frequency of mucopolysaccharide syndromes in British Columbia

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Genotype-phenotype correlations in mucopolysaccharidosis type I using enzyme kinetics, immunoquantification and in vitro turnover studies.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Inner ear pathology in the mucopolysaccharidosis VII mouse.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Enzyme replacement in a canine model of Hurler syndrome

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Enzyme-replacement therapy in mucopolysaccharidosis I.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Enzyme replacement therapy in feline mucopolysaccharidosis I.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Carbohydrate structures of recombinant human alpha-L-iduronidase secreted by Chinese hamster ovary cells

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Murine alpha-L-iduronidase: cDNA isolation and expression.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Usefulness of bone marrow transplantation in the Hurler syndrome

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Follow-up of nine patients with Hurler syndrome after bone marrow transplantation.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Electroretinographic findings in the mucopolysaccharidoses

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Ocular abnormalities in the mucopolysaccharidoses after bone marrow transplantation. Longer follow-up

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

The presenting features of mucopolysaccharidosis type IH (Hurler syndrome)

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Lysosomal storage disorders: emerging therapeutic options require early diagnosis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Myoblast gene therapy in canine mucopolysaccharidosis. I: Abrogation by an immune response to alpha-L-iduronidase

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Lipopolysaccharide potentiates the effect of hepatocyte growth factor on hepatocyte replication in rats by augmenting AP-1 activity

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.08.027

10.1016/J.YMTHE.2004.08.027

7 January 2021

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DOI

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10 December 2017

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