Is there a future for gene therapy? (Q33603980)

28 July 2017

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title

Is there a future for gene therapy? (English)

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28 July 2017

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Howell JM

1

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28 July 2017

publication date

1 March 1999

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28 July 2017

published in

Neuromuscular Disorders

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28 July 2017

volume

9

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28 July 2017

issue

2

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28 July 2017

page(s)

102-107

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The scope of gene therapy in humans: scientific, safety and ethical considerations

28 July 2017

cites work

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Direct gene transfer into mouse muscle in vivo

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Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs

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Dystrophin expression in muscles of mdx mice after adenovirus-mediated in vivo gene transfer

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Use of the dog model for Duchenne muscular dystrophy in gene therapy trials.

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Direct dystrophin and reporter gene transfer into dog muscle in vivo

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High-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscle of dystrophic dogs: prolongation of expression with immunosuppression.

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Implications of maturation for viral gene delivery to skeletal muscle

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Problems and potential for gene therapy in Duchenne muscular dystrophy

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Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted

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Insertion of the adenoviral E3 region into a recombinant viral vector prevents antiviral humoral and cellular immune responses and permits long-term gene expression

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Strategies to accomplish targeted gene delivery to muscle cells employing tropism-modified adenoviral vectors

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Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

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De novo protein design: fully automated sequence selection

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Lipoadenofection-mediated gene delivery to the corneal endothelium: prospects for modulating graft rejection

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Utrophin muscles in on the action

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Prevalence of dystrophin-positive fibers in 85 Duchenne muscular dystrophy patients

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7 January 2021

A FUNCTIONAL DISORDER OF MUSCLE ASSOCIATED WITH THE ABSENCE OF PHOSPHORYLASE.

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7 January 2021

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A splice-site mutation causing ovine McArdle's disease

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7 January 2021

10.1016/S0960-8966(98)00102-3

Identifiers

DOI

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28 July 2017

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