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

• Main Authors: Maksymilian Prondzynski (Author), Elisabeth Krämer (Author), Sandra D. Laufer (Author), Aya Shibamiya (Author), Ole Pless (Author), Frederik Flenner (Author), Oliver J. Müller (Author), Julia Münch (Author), Charles Redwood (Author), Arne Hansen (Author), Monica Patten (Author), Thomas Eschenhagen (Author), Giulia Mearini (Author), Lucie Carrier (Author)
• Format: Book
• Published: Elsevier, 2017-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...