(Q45881969)

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Long-term inhibition of glycosphingolipid accumulation in Fabry model mice by a single systemic injection of AAV1 vector in the neonatal period

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

Long-term inhibition of glycosphingolipid accumulation in Fabry model mice by a single systemic injection of AAV1 vector in the neonatal period (English)

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

author name string

Koichi Ogawa

1

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

Yukihiko Hirai

2

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

Masamichi Ishizaki

3

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

Hiroshi Takahashi

4

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

Hideki Hanawa

5

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

Yoshitaka Fukunaga

6

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

Takashi Shimada

7

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

publication date

16 December 2008

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

Molecular Genetics and Metabolism

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

96

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

3

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

91-96

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

Enzymatic Defect in Fabry's Disease

1 reference

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An Atypical Variant of Fabry's Disease in Men with Left Ventricular Hypertrophy

1 reference

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7 January 2021

Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease

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7 January 2021

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

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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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7 January 2021

Enzymatic and functional correction along with long-term enzyme secretion from transduced bone marrow hematopoietic stem/progenitor and stromal cells derived from patients with Fabry disease.

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7 January 2021

Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer

1 reference

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7 January 2021

Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells

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7 January 2021

Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

1 reference

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7 January 2021

Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer

1 reference

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7 January 2021

Correction of the nonlinear dose response improves the viability of adenoviral vectors for gene therapy of Fabry disease

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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

Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

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.YMGME.2008.10.017

7 January 2021

Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

Clinical benefit of enzyme replacement therapy in Fabry disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5.

1 reference

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7 January 2021

AAV1 mediated co-expression of formylglycine-generating enzyme and arylsulfatase a efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy

1 reference

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7 January 2021

Systemic cancer gene therapy using adeno-associated virus type 1 vector expressing MDA-7/IL24.

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

alpha-Galactosidase A deficient mice: a model of Fabry disease

1 reference

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7 January 2021

Immunohistochemical characterization of transgenic mice highly expressing human lysosomal alpha-galactosidase.

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7 January 2021

Long-term expressed human alpha-galactosidase A in tissues of HalphaG transgenic mice

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7 January 2021

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

Induction of antigen-specific CD4+ T-cell anergy and deletion by in vivo viral gene transfer

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7 January 2021

Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice

1 reference

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7 January 2021

Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

1 reference

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7 January 2021

Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors

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7 January 2021

A journey to the world of glycobiology

1 reference

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Organ-specific difference in the sugar chains of gamma-glutamyltranspeptidase

1 reference

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7 January 2021

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

1 reference

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7 January 2021

Long-term correction of hyperphenylalaninemia by AAV-mediated gene transfer leads to behavioral recovery in phenylketonuria mice

1 reference

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7 January 2021

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

1 reference

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7 January 2021

Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

Fabry disease: guidelines for the evaluation and management of multi-organ system involvement

1 reference

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7 January 2021

Enzyme replacement therapy with agalsidase alfa in children with Fabry disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.017

7 January 2021

Immunology of neonatal gene transfer

1 reference

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7 January 2021

Identifiers

10.1016/J.YMGME.2008.10.017

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/19091614

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