Promoter Dependence of Plasmid–Pluronics Targeted α Galactosidase A Expression in Skeletal Muscle of Fabry Mice (Q57390250)

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13 January 2020

title

Promoter dependence of plasmid-pluronics targeted alpha galactosidase A expression in skeletal muscle of Fabry mice (English)

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13 January 2020

1

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Francisco Javier Novo Villaverde

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3

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13 January 2020

Hanns Lochmüller

6

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author name string

Marita Pohlschmidt

2

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13 January 2020

Bernard Higgins

4

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13 January 2020

Valery Alakhov

5

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13 January 2020

Hitoshi Sakuraba

7

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13 January 2020

Geoffrey Goldspink

8

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13 January 2020

Kay MacDermot

9

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13 January 2020

Dariusz C Górecki

10

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publication date

22 June 2005

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13 January 2020

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13 January 2020

volume

12

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issue

5

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page(s)

985-990

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Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males

13 January 2020

cites work

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Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice

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

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Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA.

7 January 2021

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

7 January 2021

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

7 January 2021

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

7 January 2021

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A combination of poloxamers increases gene expression of plasmid DNA in skeletal muscle

7 January 2021

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A muscle-specific enhancer is located at the 3' end of the myosin light-chain 1/3 gene locus

7 January 2021

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Efficient muscle-specific transgene expression after adenovirus-mediated gene transfer in mice using a 1.35 kb muscle creatine kinase promoter/enhancer

7 January 2021

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Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation

7 January 2021

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

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

7 January 2021

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Role of transfection in the priming of cytotoxic T-cells by DNA-mediated immunization

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Immune evasion by muscle-specific gene expression in dystrophic muscle

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Developing DNA vaccines that call to dendritic cells

7 January 2021

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

7 January 2021

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Muscle specific versus ubiquitous expression of Gag based HIV-1 DNA vaccines: a comparative analysis

7 January 2021

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Proteolysis in MHC class II antigen presentation: who's in charge?

7 January 2021

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Fabry disease: novel alpha-galactosidase A 3'-terminal mutations result in multiple transcripts due to aberrant 3'-end formation

7 January 2021

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Alpha-galactosidase gene mutations in Fabry disease: heterogeneous expressions of mutant enzyme proteins

7 January 2021

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Block copolymeric biotransport carriers as versatile vehicles for drug delivery

7 January 2021

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7 January 2021

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Gene transfer and expression of human alpha-galactosidase from mouse muscle in vitro and in vivo.

7 January 2021

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Immune responses, not promoter inactivation, are responsible for decreased long-term expression following plasmid gene transfer into skeletal muscle

7 January 2021

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Immune responses to dystropin: implications for gene therapy of Duchenne muscular dystrophy

7 January 2021

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7 January 2021

Identifiers

DOI

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13 January 2020

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