CRISPR/Cas-based genome and transcriptome editing offer new therapeutic perspectives for various genetically caused diseases including heart diseases. However, their implementation within the human heart muscle is very challenging and various concerns need to be addressed for its translation into clinical use.
Within this project, we aim to develop an efficient approach for the CRISPR/Cas-based gene therapy in human cardiac tissue. The strategies for genome or transcriptome editing will be applied in diseased cardiac tissue generated from iPS cells from patients with Noonan syndrome.

Insights from this preclinical study in the human heart muscle will answer fundamental questions for clinical translation of the CRISPR/Cas technology to treat inherited heart diseases.

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