Successful compensation for dystrophin deficiency by a helper-dependent adenovirus expressing full-length utrophin (Q38504953)

3 September 2017

Successful compensation for dystrophin deficiency by a helper-dependent adenovirus expressing full-length utrophin (English)

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3 September 2017

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Jatinderpal R Deol

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3 September 2017

Gawiyou Danialou

2

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3 September 2017

Nancy Larochelle

3

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3 September 2017

Mylène Bourget

4

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3 September 2017

Joon-Shik Moon

5

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3 September 2017

An-Bang Liu

6

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3 September 2017

Rénald Gilbert

7

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3 September 2017

Basil J Petrof

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3 September 2017

Josephine Nalbantoglu

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3 September 2017

George Karpati

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3 September 2017

31 July 2007

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3 September 2017

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3 September 2017

15

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3 September 2017

10

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3 September 2017

1767-1774

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Dystrophin: the protein product of the Duchenne muscular dystrophy locus

3 September 2017

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Characterization of deletions in the dystrophin gene giving mild phenotypes.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Very mild muscular dystrophy associated with the deletion of 46% of dystrophin

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Improved performance of a fully gutted adenovirus vector containing two full-length dystrophin cDNAs regulated by a strong promoter

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Prolonged dystrophin expression and functional correction of mdx mouse muscle following gene transfer with a helper-dependent (gutted) adenovirus-encoding murine dystrophin

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Immune response to full-length dystrophin delivered to Dmd muscle by a high-capacity adenoviral vector

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Primary structure of dystrophin-related protein

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Association of dystrophin-related protein with dystrophin-associated proteins in mdx mouse muscle

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Localization and quantitation of the chromosome 6-encoded dystrophin-related protein in normal and pathological human muscle

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Localization of the DMDL gene-encoded dystrophin-related protein using a panel of nineteen monoclonal antibodies: presence at neuromuscular junctions, in the sarcolemma of dystrophic skeletal muscle, in vascular and other smooth muscles, and in prol

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Expression of truncated utrophin leads to major functional improvements in dystrophin-deficient muscles of mice

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Skeletal muscle-specific expression of a utrophin transgene rescues utrophin-dystrophin deficient mice.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Expression of full-length utrophin prevents muscular dystrophy in mdx mice

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Prevention of pathology in mdx mice by expression of utrophin: analysis using an inducible transgenic expression system.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Sustained improvement of muscle function one year after full-length dystrophin gene transfer into mdx mice by a gutted helper-dependent adenoviral vector

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Interferon-gamma inhibits transgene expression driven by SV40 or CMV promoters but augments expression driven by the mammalian MHC I promoter

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for duchenne muscular dystrophy

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Dystrophin, its interactions with other proteins, and implications for muscular dystrophy

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Dystrophin and utrophin bind actin through distinct modes of contact

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Mitotic stability of an episomal vector containing a human scaffold/matrix-attached region is provided by association with nuclear matrix

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivo

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Site-specific integration mediated by a hybrid adenovirus/adeno-associated virus vector.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Transfer of the full-length dystrophin-coding sequence into muscle cells by a dual high-capacity hybrid viral vector with site-specific integration ability

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Efficient generation and amplification of high-capacity adeno-associated virus/adenovirus hybrid vectors.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Novel integrating adenoviral/retroviral hybrid vector for gene therapy

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

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

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

A capsid-modified helper-dependent adenovirus vector containing the beta-globin locus control region displays a nonrandom integration pattern and allows stable, erythroid-specific gene expression

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Cloning and expression of full length mouse utrophin: the differential association of utrophin and dystrophin with AChR clusters

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Efficient selection for high-expression transfectants with a novel eukaryotic vector

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Factors influencing the efficacy, longevity, and safety of electroporation-assisted plasmid-based gene transfer into mouse muscles

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

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

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

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

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Dystrophin expression in muscle following gene transfer with a fully deleted ("gutted") adenovirus is markedly improved by trans-acting adenoviral gene products.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300260

7 January 2021

Identifiers

DOI

10.1038/SJ.MT.6300260

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3 September 2017

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