(Q28304022)

2

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Lucie Carrier

5

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author name string

Verena Behrens-Gawlik

1

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Christina Gedicke-Hornung

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

3

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language of work or name

English

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publication date

February 2014

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published in

Pfluegers Archiv

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volume

466

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issue

2

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page(s)

215-23

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cites work

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Trans-splicing correction of tau isoform imbalance in a mouse model of tau mis-splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Cardiac myosin binding protein-C gene splice acceptor site mutation is associated with familial hypertrophic cardiomyopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Mapping of a novel gene for familial hypertrophic cardiomyopathy to chromosome 11

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

The ubiquitin-proteasome system and nonsense-mediated mRNA decay in hypertrophic cardiomyopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

ESEfinder: A web resource to identify exonic splicing enhancers

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Development of a single vector system that enhances trans-splicing of SMN2 transcripts

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

A common MYBPC3 (cardiac myosin binding protein C) variant associated with cardiomyopathies in South Asia

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Classification of the cardiomyopathies: a position statement from the European Society Of Cardiology Working Group on Myocardial and Pericardial Diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Mechanisms of disease: hypertrophic cardiomyopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Genetics of hypertrophic and dilated cardiomyopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

2011 ACCF/AHA guideline for the diagnosis and treatment of hypertrophic cardiomyopathy: a report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Genetic therapies for RNA mis-splicing diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Development of a drug screening platform based on engineered heart tissue

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Targeting RNA splicing for disease therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Antisense-mediated exon inclusion

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): a phase 2 trial of intracoronary gene therapy of sarcoplasmic reticulum Ca2+-ATPase in patients with advanced heart failure

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Percutaneous cardiac recirculation-mediated gene transfer of an inhibitory phospholamban peptide reverses advanced heart failure in large animals

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Abnormal calcium handling properties underlie familial hypertrophic cardiomyopathy pathology in patient-specific induced pluripotent stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

RNA-targeting approaches for neuromuscular diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Exon exchange approach to repair Duchenne dystrophin transcripts

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Hypertrophic cardiomyopathy: from genetics to treatment

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Prevalence of hypertrophic cardiomyopathy in a general population of young adults. Echocardiographic analysis of 4111 subjects in the CARDIA Study. Coronary Artery Risk Development in (Young) Adults

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Cardiac disease in young trained athletes. Insights into methods for distinguishing athlete's heart from structural heart disease, with particular emphasis on hypertrophic cardiomyopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

How do MYBPC3 mutations cause hypertrophic cardiomyopathy?

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Evidence from human myectomy samples that MYBPC3 mutations cause hypertrophic cardiomyopathy through haploinsufficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Repair of Mybpc3 mRNA by 5'-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

In vivo cardiac myosin binding protein C gene transfer rescues myofilament contractile dysfunction in cardiac myosin binding protein C null mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

A cardiac myosin binding protein C mutation in the Maine Coon cat with familial hypertrophic cardiomyopathy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

A newly created splice donor site in exon 25 of the MyBP-C gene is responsible for inherited hypertrophic cardiomyopathy with incomplete disease penetrance

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Cardiac AAV9-S100A1 gene therapy rescues post-ischemic heart failure in a preclinical large animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Hypertrophic cardiomyopathy: distribution of disease genes, spectrum of mutations, and implications for a molecular diagnosis strategy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Correction of tau mis-splicing caused by FTDP-17 MAPT mutations by spliceosome-mediated RNA trans-splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Novel splice donor site mutation in the cardiac myosin-binding protein-C gene in familial hypertrophic cardiomyopathy. Characterization Of cardiac transcript and protein

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Defective proteolytic systems in Mybpc3-targeted mice with cardiac hypertrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Cardiac myosin-binding protein C in hypertrophic cardiomyopathy: mechanisms and therapeutic opportunities

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Adrenergic stress reveals septal hypertrophy and proteasome impairment in heterozygous Mybpc3-targeted knock-in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Pharmacological treatment options for hypertrophic cardiomyopathy: high time for evidence

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Cardiac myosin-binding protein C mutations and hypertrophic cardiomyopathy: haploinsufficiency, deranged phosphorylation, and cardiomyocyte dysfunction

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Contractile dysfunction irrespective of the mutant protein in human hypertrophic cardiomyopathy with normal systolic function

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Nonsense-mediated mRNA decay and ubiquitin-proteasome system regulate cardiac myosin-binding protein C mutant levels in cardiomyopathic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Spliceosome-mediated trans-splicing: the therapeutic cut and paste

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Mutations in the cardiac myosin binding protein-C gene on chromosome 11 cause familial hypertrophic cardiomyopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Regulation of alternative pre-mRNA splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Micro-exons of the cardiac myosin binding protein C gene: flanking introns contain a disproportionately large number of hypertrophic cardiomyopathy mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00424-013-1409-7

Hypertrophic cardiomyopathy

21 January 2018

1 reference

12 December 2020

Myofilament protein gene mutation screening and outcome of patients with hypertrophic cardiomyopathy

1 reference

12 December 2020

Organization and sequence of human cardiac myosin binding protein C gene (MYBPC3) and identification of mutations predicted to produce truncated proteins in familial hypertrophic cardiomyopathy

1 reference

12 December 2020

Genetic basis of hypertrophic cardiomyopathy: from bench to the clinics

1 reference

12 December 2020

Phosphorylation switches specific for the cardiac isoform of myosin binding protein-C: a modulator of cardiac contraction?

1 reference

12 December 2020

Less is more: therapeutic exon skipping for Duchenne muscular dystrophy

1 reference

12 December 2020