Advances in Duchenne muscular dystrophy gene therapy (Q34267400)

1 August 2017

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title

Advances in Duchenne muscular dystrophy gene therapy (English)

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Duchenne muscular dystrophy

1 August 2017

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17 March 2024

2

Gert-Jan B van Ommen

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Judith van Deutekom

series ordinal

1

object named as

Judith C. T. van Deutekom

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A14526374

17 March 2024

language of work or name

English

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National Center for Biotechnology Information

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/esummary.fcgi?db=pubmed&retmode=json&id=14526374

17 March 2024

publication date

1 October 2003

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full work available at URL

1 August 2017

https://www.nature.com/articles/nrg1180

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https://www.nature.com/articles/nrg1180.pdf

17 March 2024

file format

Portable Document Format

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17 March 2024

published in

Nature Reviews Genetics

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1 August 2017

volume

4

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1 August 2017

issue

10

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1 August 2017

page(s)

774-783

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Comprehensive detection of genomic duplications and deletions in the DMD gene, by use of multiplex amplifiable probe hybridization

1 August 2017

cites work

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Birth and population prevalence of Duchenne muscular dystrophy in The Netherlands

31 March 2020

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Calcium entry through stretch-inactivated ion channels in mdx myotubes

31 March 2020

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Animal models for muscular dystrophy show different patterns of sarcolemmal disruption.

31 March 2020

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Dystrophin deficiency, altered cell signalling and fibre hypertrophy

31 March 2020

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Activation of calcineurin and stress activated protein kinase/p38-mitogen activated protein kinase in hearts of utrophin-dystrophin knockout mice

31 March 2020

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A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase

31 March 2020

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Persistence in muscle of an adenoviral vector that lacks all viral genes

31 March 2020

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Exploiting the natural diversity in adenovirus tropism for therapy and prevention of disease

31 March 2020

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Strategies for muscle-specific targeting of adenoviral gene transfer vectors

31 March 2020

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Herpes simplex virus vector-mediated dystrophin gene transfer and expression in MDX mouse skeletal muscle

31 March 2020

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Gene transfer to muscle using herpes simplex virus-based vectors

31 March 2020

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Current protocol of a research phase I clinical trial of full-length dystrophin plasmid DNA in Duchenne/Becker muscular dystrophies. Part II: clinical protocol

31 March 2020

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Full-length dystrophin cDNA transfer into skeletal muscle of adultmdx mice by electroporation

31 March 2020

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Microbubble ultrasound improves the efficiency of gene transduction in skeletal muscle in vivo with reduced tissue damage

31 March 2020

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High-efficiency plasmid gene transfer into dystrophic muscle

31 March 2020

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Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy.

31 March 2020

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The fate of individual myoblasts after transplantation into muscles of DMD patients

31 March 2020

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

31 March 2020

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

31 March 2020

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Development of approaches to improve cell survival in myoblast transfer therapy

31 March 2020

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Enhanced migration and fusion of donor myoblasts in dystrophic and normal host muscle

31 March 2020

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Combined immunosuppression of mycophenolate mofetil and FK506 for myoblast transplantation in mdx mice

31 March 2020

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Cell Therapy of -Sarcoglycan Null Dystrophic Mice Through Intra-Arterial Delivery of Mesoangioblasts

31 March 2020

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The development and regulation of gene repair

31 March 2020

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In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide

31 March 2020

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Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides

31 March 2020

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Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping

31 March 2020

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Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

31 March 2020

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Gentamicin fails to increase dystrophin expression in dystrophin-deficient muscle

31 March 2020

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

31 March 2020

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Transgenic mdx mice expressing dystrophin with a deletion in the actin-binding domain display a "mild Becker" phenotype

31 March 2020

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Expression of Dp260 in muscle tethers the actin cytoskeleton to the dystrophin-glycoprotein complex and partially prevents dystrophy.

31 March 2020

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Forced expression of dystrophin deletion constructs reveals structure-function correlations.

31 March 2020

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

31 March 2020

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Effective restoration of dystrophin-associated proteins in vivo by adenovirus-mediated transfer of truncated dystrophin cDNAs

31 March 2020

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

31 March 2020

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Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy

31 March 2020

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

31 March 2020

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Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice

31 March 2020

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Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice

31 March 2020

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Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies.

31 March 2020

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

31 March 2020

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Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

31 March 2020

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Characterization of tissue tropism determinants of adeno-associated virus type 1.

31 March 2020

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The "rescue" of dystrophin synthesis in boys with Duchenne muscular dystrophy

31 March 2020

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Disruption of the splicing enhancer sequence within exon 27 of the dystrophin gene by a nonsense mutation induces partial skipping of the exon and is responsible for Becker muscular dystrophy

31 March 2020

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Dystrophin nonsense mutation induces different levels of exon 29 skipping and leads to variable phenotypes within one BMD family

31 March 2020

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Dystrophin-positive fibers in Duchenne dystrophy: origin and correlation to clinical course

31 March 2020

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Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion

31 March 2020

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Exon skipping during splicing of dystrophin mRNA precursor due to an intraexon deletion in the dystrophin gene of Duchenne muscular dystrophy kobe

31 March 2020

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Polypurine sequences within a downstream exon function as a splicing enhancer

31 March 2020

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Listening to silence and understanding nonsense: exonic mutations that affect splicing

31 March 2020

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Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence

31 March 2020

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Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides

31 March 2020

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The molecular basis of muscular dystrophy in the mdx mouse: a point mutation

31 March 2020

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Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides.

31 March 2020

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Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy

31 March 2020

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Dystrophin gene transcripts skipping the mdx mutation

31 March 2020

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Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

31 March 2020

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Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

31 March 2020

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Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells

31 March 2020

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

31 March 2020

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Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells

31 March 2020

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Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene.

31 March 2020

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Toxicologic effects of an oligodeoxynucleotide phosphorothioate and its analogs following intravenous administration in rats

31 March 2020

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Importance of nucleotide sequence and chemical modifications of antisense oligonucleotides

31 March 2020

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Potent and nontoxic antisense oligonucleotides containing locked nucleic acids

31 March 2020

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Antisense properties of peptide nucleic acid

31 March 2020

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Morpholino antisense oligomers: design, preparation, and properties

31 March 2020

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Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.

31 March 2020

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In vivo fate of phosphorothioate antisense oligodeoxynucleotides: predominant uptake by scavenger receptors on endothelial liver cells

31 March 2020

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Primary structure of dystrophin-related protein

31 March 2020

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The utrophin and dystrophin genes share similarities in genomic structure

31 March 2020

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

31 March 2020

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Dystrophin and utrophin: the missing links!

31 March 2020

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31 March 2020

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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

31 March 2020

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A role for dystrophin-associated glycoproteins and utrophin in agrin-induced AChR clustering

31 March 2020

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Subtle neuromuscular defects in utrophin-deficient mice

31 March 2020

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

31 March 2020

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Reciprocal expression of dystrophin and utrophin in muscles of Duchenne muscular dystrophy patients, female DMD-carriers and control subjects

31 March 2020

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The utrophin gene is transcriptionally up-regulated in regenerating muscle.

31 March 2020

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Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy

31 March 2020

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Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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Multivariate evaluation of the functional recovery obtained by the overexpression of utrophin in skeletal muscles of the mdx mouse

31 March 2020

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Non-toxic ubiquitous over-expression of utrophin in the mdx mouse

31 March 2020

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Molecular and functional analysis of the utrophin promoter

31 March 2020

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

31 March 2020

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A second promoter provides an alternative target for therapeutic up-regulation of utrophin in Duchenne muscular dystrophy

31 March 2020

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Identification of single-stranded-DNA-binding proteins that interact with muscle gene elements

31 March 2020

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Identification of a DNA element determining synaptic expression of the mouse acetylcholine receptor delta-subunit gene

31 March 2020

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Local transcriptional control of utrophin expression at the neuromuscular synapse

31 March 2020

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Muscle and neural isoforms of agrin increase utrophin expression in cultured myotubes via a transcriptional regulatory mechanism

31 March 2020

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Induction of utrophin gene expression by heregulin in skeletal muscle cells: role of the N-box motif and GA binding protein

31 March 2020

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Activation of utrophin promoter by heregulin via the ets-related transcription factor complex GA-binding protein alpha/beta

31 March 2020

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Sp1 and Sp3 physically interact and co-operate with GABP for the activation of the utrophin promoter

31 March 2020

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Nitric oxide and l-arginine cause an accumulation of utrophin at the sarcolemma: a possible compensation for dystrophin loss in Duchenne muscular dystrophy

31 March 2020

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Glucocorticoid-mediated regulation of utrophin levels in human muscle fibers

31 March 2020

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Utrophin mRNA expression in muscle is not restricted to the neuromuscular junction

31 March 2020

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A- and B-utrophin have different expression patterns and are differentially up-regulated in mdx muscle

31 March 2020

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Transcriptional activation of the utrophin promoter B by a constitutively active Ets-transcription factor

31 March 2020

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Ets, Ap-1 and GATA factor families regulate the utrophin B promoter: potential regulatory mechanisms for endothelial-specific expression

31 March 2020

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Utrophin transcription is activated by an intronic enhancer

31 March 2020

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Genetic fate of recombinant adeno-associated virus vector genomes in muscle

31 March 2020

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

31 March 2020

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Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

31 March 2020

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Persistent expression of canine factor IX in hemophilia B canines

31 March 2020

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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

31 March 2020

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Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors

31 March 2020

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Adeno-associated virus vector-mediated IL-10 gene delivery prevents type 1 diabetes in NOD mice

31 March 2020

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

31 March 2020

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Dystrophin: the protein product of the Duchenne muscular dystrophy locus

31 March 2020

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

31 March 2020

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Recovery of induced mutations for X chromosome-linked muscular dystrophy in mice

31 March 2020

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Concatamerization of adeno-associated virus circular genomes occurs through intermolecular recombination.

31 March 2020

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inferred from DOI database lookup

7 January 2021

based on heuristic

Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

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inferred from DOI database lookup

7 January 2021

based on heuristic

75

62

2024

K Fluiter

2002

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Molecular Biology and Medicine

17 March 2024

104

8

185

WD Funk

1991

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17 March 2024

Identifiers

DOI

10.1038/NRG1180

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Dimensions Publication ID

1 August 2017

1049270844

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