Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice (Q34556906)

3 August 2017

title

Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice (English)

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Virginia Arechavala-Gomeza

3 August 2017

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4

object named as

Virginia Arechavala-Gomeza

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Keith Foster

object named as

Keith Foster

5

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Dominic J Wells

object named as

Dominic J Wells

7

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author name string

Alberto Malerba

1

1 reference

3 August 2017

Paul S Sharp

2

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3 August 2017

Ian R Graham

3

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3 August 2017

Francesco Muntoni

6

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3 August 2017

George Dickson

8

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3 August 2017

publication date

23 November 2010

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3 August 2017

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3 August 2017

volume

19

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3 August 2017

issue

2

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3 August 2017

page(s)

345-354

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describes a project that uses

3 August 2017

ImageJ

1 reference

11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC3034854/fullTextXML

inferred from PubMed Central ID database lookup

cites work

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

1 reference

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

Preclinical drug trials in the mdx mouse: assessment of reliable and sensitive outcome measures

7 July 2018

1 reference

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Vivo-Morpholinos: a non-peptide transporter delivers Morpholinos into a wide array of mouse tissues

7 July 2018

1 reference

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Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene.

7 July 2018

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Sarcolemma-localized nNOS is required to maintain activity after mild exercise

7 July 2018

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Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

7 July 2018

1 reference

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Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.

7 July 2018

1 reference

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Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.

7 July 2018

1 reference

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

7 July 2018

1 reference

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Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide

7 July 2018

1 reference

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Pharmacokinetics and biodistribution of phosphorodiamidate morpholino antisense oligomers.

7 July 2018

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

7 July 2018

1 reference

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Systemically delivered antisense oligomers upregulate gene expression in mouse tissues

7 July 2018

1 reference

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X chromosome-linked muscular dystrophy (mdx) in the mouse

7 July 2018

1 reference

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

7 July 2018

1 reference

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

7 July 2018

1 reference

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7 July 2018

1 reference

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Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

27 October 2018

1 reference

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Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice.

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

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

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

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

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

Cardioprotection for Duchenne's muscular dystrophy

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

Immunohistological intensity measurements as a tool to assess sarcolemma-associated protein expression

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034854

T-cell-dependent fibrosis in the mdx dystrophic mouse

9 December 2018

1 reference

12 December 2020

The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy

1 reference

12 December 2020

Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse

1 reference

12 December 2020

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

12 December 2020

Codon and mRNA Sequence Optimization of Microdystrophin Transgenes Improves Expression and Physiological Outcome in Dystrophic mdx Mice Following AAV2/8 Gene Transfer

1 reference

12 December 2020

Peptide-morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophy

1 reference

12 December 2020

Safety pharmacology and genotoxicity evaluation of AVI-4658

1 reference

12 December 2020

Identifiers

DOI

10.1038/MT.2010.261

1 reference

3 August 2017

1 reference

3 August 2017

1 reference