Mybpc3 gene therapy for neonatal cardiomyopathy enables long-term disease prevention in mice (Q28253008)

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Saskia Schlossarek

6

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Birgit Geertz

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Ingke Braren

11

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Elisabeth Krämer

5

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Hugo A. Katus

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object named as

Hugo A Katus

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author name string

Doreen Stimpel

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Florian Weinberger

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Julia Mourot-Filiatre

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Andrea Stoehr

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Alexander Dutsch

9

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Paul J M Wijnker

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Oliver J Müller

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Thomas Voit

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Thomas Eschenhagen

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

15

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language of work or name

English

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publication date

2 December 2014

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published in

Nature Communications

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volume

5

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page(s)

5515

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cites work

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

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How do MYBPC3 mutations cause hypertrophic cardiomyopathy?

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

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

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Defective proteolytic systems in Mybpc3-targeted mice with cardiac hypertrophy

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Ubiquitin-proteasome system and hereditary cardiomyopathies

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

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

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A case of compound mutations in the MYBPC3 gene associated with biventricular hypertrophy and neonatal death.

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Sarcomere mutations in cardiomyopathy with left ventricular hypertrabeculation

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Two cases of severe neonatal hypertrophic cardiomyopathy caused by compound heterozygous mutations in the MYBPC3 gene

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

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

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

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Contractile abnormalities and altered drug response in engineered heart tissue from Mybpc3-targeted knock-in mice

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Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

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

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

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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

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Pharmacological treatment options for hypertrophic cardiomyopathy: high time for evidence

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Mechanisms of disease: hypertrophic cardiomyopathy

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Allele-specific silencing of mutant Myh6 transcripts in mice suppresses hypertrophic cardiomyopathy

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Cardiac myosin binding protein-C: a novel sarcomeric target for gene therapy

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Effect of genome size on AAV vector packaging

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Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps

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Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat.

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Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution

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Rapid and highly efficient inducible cardiac gene knockout in adult mice using AAV-mediated expression of Cre recombinase

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Hypertrophic cardiomyopathy in cardiac myosin binding protein-C knockout mice

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Asymmetric septal hypertrophy in heterozygous cMyBP-C null mice

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Evidence for FHL1 as a novel disease gene for isolated hypertrophic cardiomyopathy

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Impact of ANKRD1 mutations associated with hypertrophic cardiomyopathy on contraction parameters of engineered heart tissue.

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Hypertrophic cardiomyopathy: prognostic factors and survival analysis in 128 Egyptian patients

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7 January 2021

Homozygosity for a novel splice site mutation in the cardiac myosin-binding protein C gene causes severe neonatal hypertrophic cardiomyopathy

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7 January 2021

Next-generation sequencing (NGS) as a fast molecular diagnosis tool for left ventricular noncompaction in an infant with compound mutations in the MYBPC3 gene

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7 January 2021

Transgenic over-expression of a motor protein at high levels results in severe cardiac pathology

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7 January 2021

Rapid transition of cardiac myocytes from hyperplasia to hypertrophy during postnatal development

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7 January 2021

Identifiers

DOI

10.1038/NCOMMS6515

1 reference

Consolidated OpenCitations Corpus – April 2017

Dimensions Publication ID

ADS bibcode

2014NatCo...5.5515M

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1016814386

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1 reference

Consolidated OpenCitations Corpus – April 2017

PubMed publication ID

25463264

1 reference

Consolidated OpenCitations Corpus – April 2017