Evaluation of MYBPC3 trans -Splicing and Gene Replacement as Therapeutic Options in Human iPSC-Derived Cardiomyocytes-Reference-Cited by-同舟云学术

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

Published:2017-06 Issue: Volume:7 Page:475-486
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Prondzynski Maksymilian,Krämer Elisabeth,Laufer Sandra D.,Shibamiya Aya,Pless Ole,Flenner Frederik,Müller Oliver J.,Münch Julia,Redwood Charles,Hansen Arne,Patten Monica,Eschenhagen Thomas,Mearini Giulia,Carrier Lucie

Funder

DZHK (German Centre for Cardiovascular Research

German Ministry of Research Education (BMBF)

Seventh Framework Program of the European Union

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference57 articles.

1. Genetic therapies for RNA mis-splicing diseases;Hammond;Trends Genet.,2011

2. Genome editing. The new frontier of genome engineering with CRISPR-Cas9;Doudna;Science,2014

3. State-of-the-art human gene therapy: part I. Gene delivery technologies;Wang;Discov. Med.,2014

4. New perspectives on the prevalence of hypertrophic cardiomyopathy;Semsarian;J. Am. Coll. Cardiol.,2015

5. 2014 ESC Guidelines on diagnosis and management of hypertrophic cardiomyopathy: the Task Force for the Diagnosis and Management of Hypertrophic Cardiomyopathy of the European Society of Cardiology (ESC);Elliott;Eur. Heart J.,2014

Cited by 74 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. RNA exon editing: Splicing the way to treat human diseases;Molecular Therapy - Nucleic Acids;2024-09

2. Efficient and reproducible generation of human iPSC-derived cardiomyocytes and cardiac organoids in stirred suspension systems;Nature Communications;2024-07-15

3. An overview of the treatments for hypertrophic cardiomyopathy;Frontiers in Cardiovascular Medicine;2024-06-03

4. The Novel Frameshift Variant of the MYBPC3 Gene Associated with Hypertrophic Cardiomyopathy Significantly Decreases the Level of This Gene Transcript in the Myocardium;Russian Journal of Genetics;2024-06

5. Genetic Mutations and Mitochondrial Redox Signaling as Modulating Factors in Hypertrophic Cardiomyopathy: A Scoping Review;International Journal of Molecular Sciences;2024-05-28