(Q81058015)

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AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease

scientific article published on 01 September 2005

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scholarly article

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5 January 2020

AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease (English)

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Europe PubMed Central

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5 January 2020

Niemann-Pick disease

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Robert J. Desnick

6

object named as

Robert J Desnick

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5 January 2020

Edward H Schuchman

7

object named as

Edward H Schuchman

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5 January 2020

author name string

Christine M Barbon

1

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5 January 2020

Robin J Ziegler

2

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5 January 2020

Chester Li

3

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5 January 2020

Donna Armentano

4

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5 January 2020

Maribeth Cherry

5

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5 January 2020

Seng H Cheng

8

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5 January 2020

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1 September 2005

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5 January 2020

Molecular Therapy

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5 January 2020

12

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5 January 2020

3

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5 January 2020

431-440

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5 January 2020

Lysosomal storage diseases

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Characterization of human acid sphingomyelinase purified from the media of overexpressing Chinese hamster ovary cells

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Infusion of recombinant human acid sphingomyelinase into niemann-pick disease mice leads to visceral, but not neurological, correction of the pathophysiology

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Enzyme replacement and enhancement therapies: lessons from lysosomal disorders

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Case report. Pulmonary involvement in Niemann-Pick disease subtype B: CT findings.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Pulmonary involvement in Niemann-Pick disease: case report and literature review

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

The natural history of type B Niemann-Pick disease: results from a 10-year longitudinal study

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Mannose 6-phosphate receptor-mediated uptake is defective in acid sphingomyelinase-deficient macrophages: implications for Niemann-Pick disease enzyme replacement therapy

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Gene Transfer Strategies for Correction of Lysosomal Storage Disorders

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Acid sphingomyelinase deficient mice: a model of types A and B Niemann-Pick disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Analysis of the lung pathology and alveolar macrophage function in the acid sphingomyelinase--deficient mouse model of Niemann-Pick disease.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Adeno-associated virus expression systems for gene transfer

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Adeno-associated virus-mediated gene transfer for hemophilia B.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Cloning of infectious adeno-associated virus genomes in bacterial plasmids

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV).

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Use of SYTOX green dye in the flow cytometric analysis of bacterial phagocytosis

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.03.011

7 January 2021

Activation of human acid sphingomyelinase through modification or deletion of C-terminal cysteine

1 reference

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7 January 2021

Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers

1 reference

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7 January 2021

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10.1016/J.YMTHE.2005.03.011

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:16099409%20AND%20SRC:MED&resulttype=core&format=json

5 January 2020

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:16099409%20AND%20SRC:MED&resulttype=core&format=json

5 January 2020

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