Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease. (Q51810233)

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scientific article published on 25 January 2009

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English	Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease.	scientific article published on 25 January 2009

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scholarly article

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Europe PubMed Central

12 April 2018

http://europepmc.org/abstract/MED/19231265

Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease. (English)

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Europe PubMed Central

12 April 2018

http://europepmc.org/abstract/MED/19231265

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Beth L. Thurberg

7

object named as

Beth L Thurberg

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Europe PubMed Central

12 April 2018

http://europepmc.org/abstract/MED/19231265

Edward H Schuchman

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object named as

Edward H Schuchman

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12 April 2018

http://europepmc.org/abstract/MED/19231265

author name string

Robin J Ziegler

1

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Europe PubMed Central

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http://europepmc.org/abstract/MED/19231265

Claire Brown

2

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Europe PubMed Central

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http://europepmc.org/abstract/MED/19231265

Christine M Barbon

3

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Europe PubMed Central

12 April 2018

http://europepmc.org/abstract/MED/19231265

Anne Marie D'Angona

4

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12 April 2018

http://europepmc.org/abstract/MED/19231265

Laura Andrews

6

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Europe PubMed Central

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http://europepmc.org/abstract/MED/19231265

John M McPherson

8

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12 April 2018

http://europepmc.org/abstract/MED/19231265

Kenneth P Karey

9

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12 April 2018

http://europepmc.org/abstract/MED/19231265

Seng H Cheng

10

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http://europepmc.org/abstract/MED/19231265

publication date

25 January 2009

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12 April 2018

http://europepmc.org/abstract/MED/19231265

Molecular Genetics and Metabolism

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12 April 2018

http://europepmc.org/abstract/MED/19231265

97

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http://europepmc.org/abstract/MED/19231265

1

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35-42

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12 April 2018

http://europepmc.org/abstract/MED/19231265

A prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

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.YMGME.2009.01.008

7 January 2021

Lung disease in Niemann-Pick disease

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

Type B Niemann-Pick disease: findings at chest radiography, thin-section CT, and pulmonary function testing

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7 January 2021

The demographics and distribution of type B Niemann-Pick disease: novel mutations lead to new genotype/phenotype correlations

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7 January 2021

A very unusual presentation of Niemann-Pick disease type B in an infant: similar findings to congenital lobar emphysema.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

Early pulmonary involvement in Niemann-Pick type B disease: lung lavage is not useful.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

Successful treatment of endogenous lipoid pneumonia due to Niemann-Pick Type B disease with whole-lung lavage

1 reference

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7 January 2021

Pulmonary involvement by Niemann-Pick disease. A report of six cases

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

Niemann-Pick disease: sixteen-year follow-up of allogeneic bone marrow transplantation in a type B variant.

1 reference

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7 January 2021

Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

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.YMGME.2009.01.008

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.YMGME.2009.01.008

7 January 2021

Biochemical, pathological, and clinical response to transplantation of normal bone marrow cells into acid sphingomyelinase-deficient mice

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

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.YMGME.2009.01.008

7 January 2021

Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

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.YMGME.2009.01.008

7 January 2021

Comparative effects of recombinant acid sphingomyelinase administration by different routes in niemann-pick disease mice

1 reference

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7 January 2021

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

1 reference

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7 January 2021

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

1 reference

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7 January 2021

Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

1 reference

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7 January 2021

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

1 reference

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7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

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.YMGME.2009.01.008

7 January 2021

Lung involvement and enzyme replacement therapy in Gaucher's disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

The lung as a route for systemic delivery of therapeutic proteins and peptides

1 reference

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7 January 2021

Correction of enzyme levels with allogeneic hematopoeitic progenitor cell transplantation in Niemann-Pick type B

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.01.008

7 January 2021

Identifiers

10.1016/J.YMGME.2009.01.008

1 reference

Europe PubMed Central

12 April 2018

http://europepmc.org/abstract/MED/19231265

1 reference

Europe PubMed Central

12 April 2018

http://europepmc.org/abstract/MED/19231265

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