(Q45855450)

17 December 2017

title

Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping (English)

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17 December 2017

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3

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17 December 2017

Vincent Mouly

9

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17 December 2017

Nicolas Levy

12

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17 December 2017

Gillian Butler-Browne

object named as

Gillian Butler-Browne

8

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author name string

Nicolas Wein

1

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17 December 2017

Aurélie Avril

2

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17 December 2017

Cyriaque Beley

4

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17 December 2017

Soraya Chaouch

5

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17 December 2017

Pascal Laforêt

6

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17 December 2017

Anthony Behin

7

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17 December 2017

Martin Krahn

10

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17 December 2017

Luis Garcia

11

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17 December 2017

publication date

1 February 2010

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17 December 2017

published in

Human Mutation

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17 December 2017

volume

31

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17 December 2017

issue

2

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17 December 2017

page(s)

136-142

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17 December 2017

Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense

cites work

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Defective membrane repair in dysferlin-deficient muscular dystrophy

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https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Efficient control of gene expression in the hematopoietic system using a single Tet-on inducible lentiviral vector

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https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

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https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice

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https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

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

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Correction of disease-associated exon skipping by synthetic exon-specific activators

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https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Immortalized Skin Fibroblasts Expressing Conditional MyoD as a Renewable and Reliable Source of Converted Human Muscle Cells to Assess Therapeutic Strategies for Muscular Dystrophies: Validation of an Exon-Skipping Approach to Restore Dystrophin in D

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice.

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Dysferlin expression in monocytes: A source of mRNA for mutation analysis

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https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Human Splicing Finder: an online bioinformatics tool to predict splicing signals

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https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Clones of Human Satellite Cells Can Express in Vitro both Fast and Slow Myosin Heavy Chains

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https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs

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https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Analysis of the DYSF mutational spectrum in a large cohort of patients

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Identification of different genomic deletions and one duplication in the dysferlin gene using multiplex ligation-dependent probe amplification and genomic quantitative PCR.

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Dysferlin, a novel skeletal muscle gene, is mutated in Miyoshi myopathy and limb girdle muscular dystrophy

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Lentiviruses as gene transfer agents for delivery to non-dividing cells

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Phenotypic study in 40 patients with dysferlin gene mutations: high frequency of atypical phenotypes

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Lariat branch point mutation in the dysferlin gene with mild limb-girdle muscular dystrophy

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

An increased specificity score matrix for the prediction of SF2/ASF-specific exonic splicing enhancers

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Mutation impact on dysferlin inferred from database analysis and computer-based structural predictions.

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase.

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.21160

7 January 2021

https://api.crossref.org/works/10.1002%2FHUMU.21160

1 reference

7 January 2021

Identifiers

DOI

10.1002/HUMU.21160

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17 December 2017

PubMed ID

19953532

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17 December 2017