Gene therapy for muscular dystrophy - a review of promising progress (Q35184730)

7 August 2017

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title

Gene therapy for muscular dystrophy - a review of promising progress (English)

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7 August 2017

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Paul Gregorevic

1

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7 August 2017

Jeffrey S Chamberlain

series ordinal

2

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7 August 2017

language of work or name

English

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publication date

1 August 2003

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Expert Opinion on Biological Therapy

7 August 2017

published in

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7 August 2017

volume

3

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7 August 2017

issue

5

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7 August 2017

page(s)

803-814

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A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin.

7 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Dystrophin protects the sarcolemma from stresses developed during muscle contraction

21 January 2018

1 reference

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Dystrophin: the protein product of the Duchenne muscular dystrophy locus

21 January 2018

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Nitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarcolemma in Duchenne muscular dystrophy

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Human and murine dystrophin mRNA transcripts are differentially expressed during skeletal muscle, heart, and brain development

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Characterization of deletions in the dystrophin gene giving mild phenotypes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Assembly of the dystrophin-associated protein complex does not require the dystrophin COOH-terminal domain

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

The role of basal and myogenic factors in the transcriptional activation of utrophin promoter A: implications for therapeutic up-regulation in Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Direct gene transfer into mouse muscle in vivo

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Non-viral gene delivery in skeletal muscle: a protein factory

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Transfection of large plasmids in primary human myoblasts.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Improved gene transfer by direct plasmid injection associated with regeneration in mouse skeletal muscle.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Bacterial beta-galactosidase and human dystrophin genes are expressed in mouse skeletal muscle fibers after ballistic transfection.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Intramuscular plasmid DNA injection can accelerate autoimmune responses.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

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

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Functional improvement of dystrophic muscle by myostatin blockade.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Muscle-specific expression of insulin-like growth factor I counters muscle decline in mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Systemic delivery of a high-capacity adenoviral vector expressing mouse CTLA4Ig improves skeletal muscle gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Generation and growth of gutted adenoviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Lentivirus administration to rat muscle provides efficient sustained expression of erythropoietin.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

A high-titer lentiviral production system mediates efficient transduction of differentiated cells including beating cardiac myocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

In vivo gene transfer using a nonprimate lentiviral vector pseudotyped with Ross River Virus glycoproteins

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

'Advanced' generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Gene transfer with foamy virus vectors

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Herpes simplex virus type 1/adeno-associated virus hybrid vectors mediate site-specific integration at the adeno-associated virus preintegration site, AAVS1, on human chromosome 19.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Herpes simplex virus type 1/adeno-associated virus rep(+) hybrid amplicon vector improves the stability of transgene expression in human cells by site-specific integration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Expression of human dystrophin following the transplantation of genetically modified mdx myoblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Ex vivo gene transfer using adenovirus-mediated full-length dystrophin delivery to dystrophic muscles

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

A role for natural killer cells in the rapid death of cultured donor myoblasts after transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Accelerated age-related decline in replicative life-span of Duchenne muscular dystrophy myoblasts: implications for cell and gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Genetic correction of dystrophin deficiency and skeletal muscle remodeling in adult MDX mouse via transplantation of retroviral producer cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Transplantation of dermal fibroblasts expressing MyoD1 in mouse muscles

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Identification of a novel population of muscle stem cells in mice: potential for muscle regeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Skeletal muscle repair by adult human mesenchymal stem cells from synovial membrane.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Human adipose tissue is a source of multipotent stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Myogenic specification of side population cells in skeletal muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Intraarterial injection of muscle-derived CD34(+)Sca-1(+) stem cells restores dystrophin in mdx mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Stem cell route to neuromuscular therapies

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Long-term doxycycline-regulated secretion of erythropoietin by encapsulated myoblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

Skeletal myogenic potential of human and mouse neural stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.3.5.803

21 January 2018

Identifiers

DOI

10.1517/14712598.3.5.803

1 reference

7 August 2017

1 reference