(Q44753961)

English

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.

scientific article published in February 2004

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18 November 2019

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 (English)

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18 November 2019

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18 November 2019

Robert J. Desnick

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Robert J Desnick

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18 November 2019

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Robin J Ziegler

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18 November 2019

Scott M Lonning

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18 November 2019

Donna Armentano

3

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18 November 2019

Chester Li

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18 November 2019

David W Souza

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18 November 2019

Maribeth Cherry

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18 November 2019

Christine Ford

7

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18 November 2019

Christine M Barbon

8

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18 November 2019

Guangping Gao

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18 November 2019

Richard Peluso

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18 November 2019

Simon Godwin

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18 November 2019

Barrie J Carter

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18 November 2019

Richard J Gregory

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18 November 2019

Samuel C Wadsworth

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18 November 2019

Seng H Cheng

17

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18 November 2019

publication date

1 February 2004

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18 November 2019

Molecular Therapy

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18 November 2019

9

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18 November 2019

2

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18 November 2019

231-240

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18 November 2019

Fabry's disease: heterozygous form of different expression in two monozygous twin sisters

1 reference

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7 January 2021

Anderson-Fabry disease: clinical manifestations of disease in female heterozygotes

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7 January 2021

Lysosomal storage diseases

1 reference

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7 January 2021

Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice

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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

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

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7 January 2021

Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector

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7 January 2021

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

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7 January 2021

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

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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

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7 January 2021

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

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7 January 2021

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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

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

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7 January 2021

Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy

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

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

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7 January 2021

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Advances in the management of Anderson-Fabry disease: enzyme replacement therapy

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Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

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Humoral immune response in mice against a circulating antigen induced by adenoviral transfer is strictly dependent on expression in antigen-presenting cells

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

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