(Q34595095)

3 August 2017

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

1 reference

3 August 2017

7

Robert J Desnick

1 reference

3 August 2017

Robin J Ziegler

1

1 reference

3 August 2017

Maribeth Cherry

2

1 reference

3 August 2017

Christine M Barbon

3

1 reference

3 August 2017

Chester Li

4

1 reference

3 August 2017

Scott D Bercury

5

1 reference

3 August 2017

Donna Armentano

6

1 reference

3 August 2017

Seng H Cheng

8

1 reference

3 August 2017

26 December 2006

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3 August 2017

1 reference

3 August 2017

15

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3 August 2017

3

1 reference

3 August 2017

492-500

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Clinical features of and recent advances in therapy for Fabry disease

3 August 2017

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

A biochemical and pharmacological comparison of enzyme replacement therapies for the glycolipid storage disorder Fabry disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

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/10.1038%2FSJ.MT.6300066

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

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

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/10.1038%2FSJ.MT.6300066

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/10.1038%2FSJ.MT.6300066

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/10.1038%2FSJ.MT.6300066

7 January 2021

Fabry disease in mice is associated with age-dependent susceptibility to vascular thrombosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Physiological characterization of neuropathy in Fabry's disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Enzyme replacement therapy improves function of C-, Adelta-, and Abeta-nerve fibers in Fabry neuropathy.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

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/10.1038%2FSJ.MT.6300066

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/10.1038%2FSJ.MT.6300066

7 January 2021

AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Small fiber dysfunction predominates in Fabry neuropathy.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

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

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Complete prevention of atherosclerosis in apoE-deficient mice by hepatic human apoE gene transfer with adeno-associated virus serotypes 7 and 8.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia a dogs and mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Quantitative analysis of epidermal innervation in Fabry disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Enzyme replacement therapy and intraepidermal innervation density in Fabry disease.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

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/10.1038%2FSJ.MT.6300066

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/10.1038%2FSJ.MT.6300066

7 January 2021

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Local delivery of a viral vector mitigates neutralization by antiviral antibodies and results in efficient transduction of rabbit liver

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Quantitative determination of globotriaosylceramide by immunodetection of glycolipid-bound recombinant verotoxin B subunit

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300066

7 January 2021

Identifiers

DOI

10.1038/SJ.MT.6300066

1 reference

3 August 2017

1 reference