Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping (Q30514014)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22388933%20AND%20SRC:MED&resulttype=core&format=json

24 January 2020

title

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping (English)

1 reference

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24 January 2020

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24 January 2020

Kay E Davies

1 reference

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24 January 2020

Vivienne Wilkins

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24 January 2020

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Arran Babbs

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24 January 2020

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Jordan Wright

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24 January 2020

Dave Powell

series ordinal

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24 January 2020

Luis Garcia

series ordinal

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24 January 2020

publication date

2 March 2012

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24 January 2020

published in

Human Molecular Genetics

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24 January 2020

volume

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24 January 2020

issue

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24 January 2020

page(s)

2559-2571

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describes a project that uses

24 January 2020

ImageJ

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11 June 2022

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inferred from PubMed Central ID database lookup

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Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping

20 June 2018

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

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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

20 June 2018

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AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

20 June 2018

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Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy

20 June 2018

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Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy

20 June 2018

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A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.

20 June 2018

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

20 June 2018

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20 June 2018

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20 June 2018

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20 June 2018

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20 June 2018

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20 June 2018

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

20 June 2018

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Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

20 June 2018

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20 June 2018

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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

20 June 2018

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20 June 2018

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20 June 2018

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20 June 2018

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20 June 2018

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20 June 2018

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3349427

The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion

20 June 2018

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Dystrophin immunity in Duchenne's muscular dystrophy.

20 June 2018

1 reference

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

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3349427

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3349427

In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

26 November 2018

1 reference

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

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3349427

Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice

26 November 2018

1 reference

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Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products

26 November 2018

1 reference

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Behavioural characterisation of the robotic mouse mutant

26 November 2018

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CD8(+) T-cell responses to adeno-associated virus capsid in humans

26 November 2018

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Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy

26 November 2018

1 reference

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

26 November 2018

1 reference

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Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

26 November 2018

1 reference

12 December 2020

Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression

1 reference

12 December 2020

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

1 reference

12 December 2020

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

12 December 2020

Skeletal and Cardiac Myopathies in Mice Lacking Utrophin and Dystrophin: A Model for Duchenne Muscular Dystrophy

1 reference

12 December 2020

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy

1 reference

12 December 2020

Identifiers

DOI

10.1093/HMG/DDS082

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22388933%20AND%20SRC:MED&resulttype=core&format=json

24 January 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22388933%20AND%20SRC:MED&resulttype=core&format=json

24 January 2020

PubMed publication ID

22388933

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22388933%20AND%20SRC:MED&resulttype=core&format=json