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Article

Evaluation of MYBPC3 trans -Splicing and Gene Replacement as Therapeutic Options in Human iPSC-Derived Cardiomyocytes

17 May 2017

Abstract excerpt

Gene therapy is a promising option for severe forms of genetic diseases. We previously provided evidence for the feasibility of trans-splicing, exon skipping, and gene replacement in a mouse model of hypertrophic cardiomyopathy (HCM) carrying a mutation in MYBPC3, encoding cardiac myosin-binding protein C (cMyBP-C). Here we used human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) from an HCM...

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PMID: 28624223
DOI: 10.1016/j.omtn.2017.05.008

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Related research

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Evaluation of MYBPC3 trans -Splicing and Gene Replacement as Therapeutic Options in Human iPSC-Derived CardiomyocytesPMID 28624223Read ↗

Repair of Mybpc3 mRNA by 5′-trans-splicing in a Mouse Model of Hypertrophic CardiomyopathySemantic proximity Read ↗

An integration-free iPSC line ZZUNEUi028-A derived from a patient with hypertrophic cardiomyopathy carrying a heterozygous mutation (c. 1504 C > T) in MYBPC3 gene.Semantic proximity Read ↗

Generation of induced pluripotent stem cell lines from two unrelated individuals with familial hypertrophic cardiomyopathy carrying MYBPC3 nonsense mutations.Semantic proximity Read ↗

Generation of two heterozygous MYBPC3 mutation-carrying human iPSC lines, SCVIi001-A and SCVIi002-A, for modeling hypertrophic cardiomyopathy.Semantic proximity Read ↗

Patient-derived induced pluripotent stem cells to study non-canonical splicing variants associated with Hypertrophic CardiomyopathySemantic proximity Read ↗

Transcriptome Sequencing of Patients With Hypertrophic Cardiomyopathy Reveals Novel Splice-Altering Variants in MYBPC3.Semantic proximity Read ↗

Variable cMyBP-C expression from cell to cell in a MYBPC3c.927-2 A>G hiPSC-CM model recapitulates HCM patient phenotype.Semantic proximity Read ↗

MYBPC3-c.772G>A mutation results in haploinsufficiency and altered myosin cycling kinetics in a patient induced stem cell derived cardiomyocyte model of hypertrophic cardiomyopathy.Semantic proximity Read ↗

Establishment and characterization of induced pluripotent stem cells from an individual with familial hypertrophic cardiomyopathy carrying the MYBPC3 c.772G>A mutationSemantic proximity Read ↗

Gene Therapy Mediates Therapeutic Improvement in Cardiac Hypertrophy and Survival in a Murine Model of <i>MYBPC3</i> -Associated CardiomyopathySemantic proximity Read ↗

Generation of two induced pluripotent stem cell lines from hypertrophic cardiomyopathy patients carrying MYBPC3 mutations.Semantic proximity Read ↗

AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy modelsSemantic proximity Read ↗

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