Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice - Wikidata - thesis-toulmin - TriplyDB

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

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

http://www.wikidata.org/entity/Q28291547

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Animal and in silico models for the study of sarcomeric cardiomyopathies Targets for therapy in sarcomeric cardiomyopathies Mybpc3 gene therapy for neonatal cardiomyopathy enables long-term disease prevention in mice MYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correction Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.Epigallocatechin-3-Gallate Accelerates Relaxation and Ca2+ Transient Decay and Desensitizes Myofilaments in Healthy and Mybpc3-Targeted Knock-in Cardiomyopathic Mice.FHL2 expression and variants in hypertrophic cardiomyopathy.INHERIT (INHibition of the renin angiotensin system in hypertrophic cardiomyopathy and the Effect on hypertrophy-a Randomised Intervention Trial with losartan)In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy.Therapeutic activity of modified U1 core spliceosomal particles Splice-switching antisense oligonucleotides as therapeutic drugs MYBPC3's alternate ending: consequences and therapeutic implications of a highly prevalent 25 bp deletion mutation Alterations in sarcomere function modify the hyperplastic to hypertrophic transition phase of mammalian cardiomyocyte development.Missing links in cardiology: long non-coding RNAs enter the arena.Development of therapeutic splice-switching oligonucleotides.In vitro correction of a pseudoexon-generating deep intronic mutation in LGMD2A by antisense oligonucleotides and modified small nuclear RNAs.I-1-deficiency negatively impacts survival in a cardiomyopathy mouse model.The Role of Cardiac Myosin Binding Protein C3 in Hypertrophic Cardiomyopathy-Progress and Novel Therapeutic Opportunities.Welcome to the splice age: antisense oligonucleotide-mediated exon skipping gains wider applicability.Evaluation of MYBPC3 trans-Splicing and Gene Replacement as Therapeutic Options in Human iPSC-Derived Cardiomyocytes.Nebivolol Desensitizes Myofilaments of a Hypertrophic Cardiomyopathy Mouse Model.Proteasome inhibition slightly improves cardiac function in mice with hypertrophic cardiomyopathy.Diltiazem prevents stress-induced contractile deficits in cardiomyocytes, but does not reverse the cardiomyopathy phenotype in Mybpc3-knock-in mice.Hypertrophic Cardiomyopathy: Genetics, Pathogenesis, Clinical Manifestations, Diagnosis, and Therapy.Functional Characterization and Rescue of a Deep Intronic Mutation in OCRL Gene Responsible for Lowe Syndrome.Activation of Autophagy Ameliorates Cardiomyopathy in Mybpc3-Targeted Knockin Mice.Ranolazine antagonizes catecholamine-induced dysfunction in isolated cardiomyocytes, but lacks long-term therapeutic effects in vivo in a mouse model of hypertrophic cardiomyopathy.RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect.Genome Editing in Cardiovascular Biology.Gene Editing and Gene-Based Therapeutics for Cardiomyopathies.Genome Editing Redefines Precision Medicine in the Cardiovascular Field.Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers.S100A4 as a Target of the E3-Ligase Asb2β and Its Effect on Engineered Heart Tissue Changes in the cardiac metabolome caused by perhexiline treatment in a mouse model of hypertrophic cardiomyopathy

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

http://www.wikidata.org/entity/Q28291547

description

2013 nî lūn-bûn

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2013 թուականի Յուլիսին հրատարակուած գիտական յօդուած

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2013 թվականի հուլիսին հրատարակված գիտական հոդված

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2013年の論文

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2013年論文

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2013年論文

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2013年論文

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2013年論文

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2013年論文

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2013年论文

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name

Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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EMBO Molecular Medicine

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Rescue of cardiomyopathy throu ...... Mybpc3-targeted knock-in mice

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Doreen Stimpel

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Verena Behrens-Gawlik

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P2860

Neonatal cardiomyopathy in mice homozygous for the Arg403Gln mutation in the alpha cardiac myosin heavy chain gene.

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

Assembly, nuclear import and function of U7 snRNPs studied by microinjection of synthetic U7 RNA into Xenopus oocytes

Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications

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

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

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

Impairment of the ubiquitin-proteasome system by truncated cardiac myosin binding protein C mutants

Cardiac myosin-binding protein C is required for complete relaxation in intact myocytes

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

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10.1002/EMMM.201202168

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Saskia Schlossarek

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