(Q39225878)

10 September 2017

title

Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy (English)

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10 September 2017

12

Stanley F Nelson

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10 September 2017

Genevieve C Kendall

1

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10 September 2017

Ekaterina I Mokhonova

2

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10 September 2017

Miriana Moran

3

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10 September 2017

Natalia E Sejbuk

4

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10 September 2017

Derek W Wang

5

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10 September 2017

Oscar Silva

6

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10 September 2017

Richard T Wang

7

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10 September 2017

Leonel Martinez

8

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10 September 2017

Qi L Lu

9

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10 September 2017

Robert Damoiseaux

10

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10 September 2017

Melissa J Spencer

11

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10 September 2017

M Carrie Miceli

13

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10 September 2017

language of work or name

English

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publication date

1 December 2012

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Science Translational Medicine

10 September 2017

published in

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10 September 2017

volume

4

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10 September 2017

issue

164

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10 September 2017

page(s)

164ra160

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Population frequencies of inherited neuromuscular diseases—A world survey

10 September 2017

cites work

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Detection of deletions spanning the Duchenne muscular dystrophy locus using a tightly linked DNA segment

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Follow-up of three patients with a large in-frame deletion of exons 45-55 in the Duchenne muscular dystrophy (DMD) gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Recombinant micro-genes and dystrophin viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

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

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Systemic administration of PRO051 in Duchenne's muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

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/10.1126%2FSCITRANSLMED.3005054

Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Recognizing and managing a malignant hyperthermia crisis: guidelines from the European Malignant Hyperthermia Group

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Pharmacotherapy of spasticity in children with cerebral palsy

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

The human dystrophin gene requires 16 hours to be transcribed and is cotranscriptionally spliced

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Biochemistry of muscle membranes in Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Dantrolene inhibition of ryanodine receptor Ca2+ release channels. Molecular mechanism and isoform selectivity

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Ryanodine receptor calcium release channels

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Hypernitrosylated ryanodine receptor calcium release channels are leaky in dystrophic muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Ryanodine receptor oxidation causes intracellular calcium leak and muscle weakness in aging.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Fixing ryanodine receptor Ca leak - a novel therapeutic strategy for contractile failure in heart and skeletal muscle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Chemical treatment enhances skipping of a mutated exon in the dystrophin gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Genetic therapies for RNA mis-splicing diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Generation of human induced pluripotent stem cells from dermal fibroblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Cellular senescence in human myoblasts is overcome by human telomerase reverse transcriptase and cyclin-dependent kinase 4: consequences in aging muscle and therapeutic strategies for muscular dystrophies

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Calcium release from ryanodine receptors in the nucleoplasmic reticulum

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

The influence of calcium signaling on the regulation of alternative splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Dantrolene stabilizes domain interactions within the ryanodine receptor.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Menstrual blood-derived cells confer human dystrophin expression in the murine model of Duchenne muscular dystrophy via cell fusion and myogenic transdifferentiation

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Reference genes for normalization of gene expression studies in human osteoarthritic articular cartilage

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3005054

10.1126/SCITRANSLMED.3005054

21 January 2018

Identifiers

DOI

1 reference

10 September 2017

1 reference