Evaluation of MYBPC3 trans-splicing and gene replacement as therapeutic options in human iPSC-derived cardiomyocytes

Evaluation of MYBPC3 trans-splicing and gene replacement as therapeutic options in human iPSC-derived cardiomyocytes

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 pro...

Full description

Bibliographic Details
Main Authors:	Prondzynski, M, Krämer, E, Laufer, S, Shibamiya, A, Pless, O, Flenner, F, Müller, O, Münch, J, Redwood, C, Hansen, A, Patten, M, Eschenhagen, T, Mearini, G, Carrier, L
Format:	Journal article
Language:	English
Published:	Elsevier 2017

Similar Items

The utilisation of hydrogels for iPSC-cardiomyocyte research
by: Patel, L, et al.
Published: (2023)

Activation of autophagy ameliorates cardiomyopathy in Mybpc3-targeted knockin mice
by: Singh, SR, et al.
Published: (2017)

Generation of a human iPSC-derived cardiomyocyte/fibroblast engineered heart tissue model
by: Cumberland, MJ, et al.
Published: (2023)

Geometry-dependent functional changes in iPSC-derived cardiomyocytes probed by functional imaging and RNA sequencing
by: Gaublomme, Jellert, et al.
Published: (2017)

Modeling MELAS-associated cardiac defects using patient-specific iPSC-derived cardiomyocytes and cardiac organoids
by: Phua, Qian Hua
Published: (2018)

CRISPR/Cas9-mediated engineering of subtype-specific cell lines for iPSC-derived cardiomyocyte phenotyping
by: Sontayananon, N
Published: (2022)

Comparing the effects of chemical Ca2+ dyes and R-GECO on contractility and Ca2+ transients in adult and human iPSC cardiomyocytes
by: Robinson, P, et al.
Published: (2023)

Pro-maturational effects of human iPSC-derived cortical astrocytes upon iPSC-derived cortical neurons
by: Anne Hedegaard, et al.
Published: (2020)

Fluorescent PSC-derived cardiomyocyte reporter lines: generation approaches and their applications in cardiovascular medicine
by: Sontayananon, N, et al.
Published: (2020)

Pathogenic intronic splice-affecting variants in MYBPC3 in three patients with hypertrophic cardiomyopathy
by: Wood, KA, et al.
Published: (2021)

How do MYBPC3 mutations cause hypertrophic cardiomyopathy?
by: Marston, S, et al.
Published: (2012)

Generation of human iPSC-derived cerebellar organoids
by: Tolonen, J-P, et al.
Published: (2024)

A novel human stem cell platform for probing adrenoceptor signaling in iPSC derived cardiomyocytes including those with an adult atrial phenotype
by: Ahmad, F
Published: (2017)

Modeling Alzheimer’s disease with iPSC-derived brain cells
by: Penney, Jay, et al.
Published: (2020)

Excess α-synuclein compromises phagocytosis in iPSC-derived macrophages.
by: Haenseler, W, et al.
Published: (2017)

Use of UTF1 genetic control elements as iPSC reporter
by: Morshedi, Amir, et al.
Published: (2013)

CRISPRi: a way to integrate iPSC-derived neuronal models
by: Franks, SNJ, et al.
Published: (2024)

Monte Carlo simulation of radiation transport for benchmarking Intel's iPSC/2, IPSC/860, and Touchstone Delta machine
by: Klemas, Thomas J. (Thomas Jonas)
Published: (2007)

REST and neural gene network Dysregulation in iPSC models of Alzheimer’s disease
by: Meyer, Katharina, et al.
Published: (2019)

(-)-Indolactam V and MO47: reducing heterogeneity in human iPSC culture
by: Schuessler, M
Published: (2018)

Large-scale production of human iPSC-derived macrophages for drug screening
by: Gutbier, S, et al.
Published: (2020)

Honing the double-edged sword: improving human iPSC-microglia models
by: Hedegaard, A, et al.
Published: (2020)

Clusterin is required for β-Amyloid toxicity in human iPSC-derived neurons
by: Robbins, J, et al.
Published: (2018)

REST and Neural Gene Network Dysregulation in iPSC Models of Alzheimer’s Disease
by: Meyer, Katharina, et al.
Published: (2020)

Choline Ameliorates Disease Phenotypes in Human iPSC Models of Rett Syndrome
by: Chin, Eunice W. M., et al.
Published: (2016)

Phenotyping C9-ALS/FTD iPSC-derived cortical neurons
by: Graf, MS
Published: (2023)

Studying α-Synuclein pathology using iPSC-derived dopaminergic neurons
by: Zambon, F
Published: (2017)

Resolving cell state in iPSC-derived human neural samples with multiplexed fluorescence imaging
by: Tomov, Martin L, et al.
Published: (2022)

Resolving cell state in iPSC-derived human neural samples with multiplexed fluorescence imaging
by: Tomov, Martin L, et al.
Published: (2021)

Hypertrophic cardiomyopathy mutations in MYBPC3 dysregulate myosin
by: Toepfer, C, et al.
Published: (2019)

Disease modeling of a mutation in α‐actinin 2 guides clinical therapy in hypertrophic cardiomyopathy
by: Prondzynski, M, et al.
Published: (2019)

Fatty acid-stimulated maturation of human IPSC-derived cardiomyocytes in engineered heart tissue
by: Purnama, U, et al.
Published: (2020)

APOE4 disrupts intracellular lipid homeostasis in human iPSC-derived glia
by: Sienski, G, et al.
Published: (2021)

The European General Data Protection Regulation: challenges and considerations for iPSC researchers and biobanks
by: Morrison, M, et al.
Published: (2017)

Induced pluripotent stem cell (iPSC)-derived dopaminergic models of Parkinson's disease.
by: Beevers, J, et al.
Published: (2013)

In vitro quantitative imaging assay for phagocytosis of dead neuroblastoma cells by iPSC-macrophages
by: Hall-Roberts, H, et al.
Published: (2021)

iPSC-derived microglia as a model to study inflammation in idiopathic Parkinson's disease
by: Badanjak, K, et al.
Published: (2021)

The use of iPSC-derived neurons to investigate the role of clusterin trafficking in Alzheimer’s disease
by: Foster, EM
Published: (2021)

Modelling and analysis of LRRK2 mutations in iPSC-derived dopaminergic neurons and astrocytes
by: Booth, H
Published: (2017)

NeuroLINCS Proteomics: Defining human-derived iPSC proteomes and protein signatures of pluripotency
by: Fraenkel, Ernest
Published: (2023)