(Q39120926)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

title

Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

congenital muscular dystrophy

5 March 2020

main subject

congenital disorder

0 references

1 reference

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

author name string

Yoshitsugu Aoki

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

Tetsuya Nagata

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

Toshifumi Yokota

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

Akinori Nakamura

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

Terence Partridge

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

Shin'ichi Takeda

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

publication date

23 July 2013

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

published in

Human Molecular Genetics

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

volume

22

1 reference

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5 March 2020

issue

24

1 reference

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5 March 2020

page(s)

4914-4928

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23882132%20AND%20SRC:MED&resulttype=core&format=json

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

5 March 2020

cites work

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Systemic administration of PRO051 in Duchenne's muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Dystrophin protects the sarcolemma from stresses developed during muscle contraction

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Targeted disruption of exon 52 in the mouse dystrophin gene induced muscle degeneration similar to that observed in Duchenne muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Laminin alpha2 chain-null mutant mice by targeted disruption of the Lama2 gene: a new model of merosin (laminin 2)-deficient congenital muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Pharmacokinetics and biodistribution of phosphorodiamidate morpholino antisense oligomers.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Resistance of morpholino phosphorodiamidate oligomers to enzymatic degradation

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Cellular uptake of neutral phosphorodiamidate morpholino oligomers.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Duchenne dystrophy: Electron microscopic findings pointing to a basic or early abnormality in the plasma membrane of the muscle fiber

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Our trails and trials in the subsarcolemmal cytoskeleton network and muscular dystrophy researches in the dystrophin era.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Visualization of dystrophic muscle fibers in mdx mouse by vital staining with Evans blue: evidence of apoptosis in dystrophin-deficient muscle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

RNA-targeted splice-correction therapy for neuromuscular disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Scavenger receptor-mediated uptake of cell-penetrating peptide nanocomplexes with oligonucleotides

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Cellular uptake of antisense morpholino oligomers conjugated to arginine-rich peptides.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Merosin and congenital muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Skeletal muscle laminin and MDC1A: pathogenesis and treatment strategies

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Identification of a novel mutant transcript of laminin α2 chain gene responsible for muscular dystrophy and dysmyelination in dy2J mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

ESEfinder: A web resource to identify exonic splicing enhancers

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Time course of changes in plasma membrane permeability in the dystrophin-deficient mdx mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDT341

Activation of the alternative pathway of complement by a phosphorothioate oligonucleotide: potential mechanism of action.

21 January 2018

1 reference

12 December 2020

Intracellular delivery strategies for antisense phosphorodiamidate morpholino oligomers

1 reference

12 December 2020

Merosin-deficient congenital muscular dystrophy, autosomal recessive (MDC1A, MIM#156225, LAMA2 gene coding for alpha2 chain of laminin)

1 reference

12 December 2020

Phosphorothioate oligonucleotides inhibit the intrinsic tenase complex.

1 reference

12 December 2020

Myogenic satellite cells: physiology to molecular biology

1 reference

12 December 2020

Activity, creatine kinase, and myoglobin in Duchenne muscular dystrophy: a clue to etiology?

1 reference

12 December 2020

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.

1 reference

12 December 2020

Exon-skipping therapy for Duchenne muscular dystrophy

1 reference

12 December 2020

Toward an oligonucleotide therapy for Duchenne muscular dystrophy: a complex development challenge

1 reference

12 December 2020