Advances in genome editing technologies have made possible the introduction of precise genetic modifications to hPSCs, facilitating a range of applications in basic and translational research.
In conjunction with the Transgenic Animal and Genome Editing core at CCHMC, the PSCF now offers an hPSC gene editing service. We have successfully used CRISPR/Cas9 to introduce the following classes of genome edits in various hPSC lines:
1. Introduction of INDELs to introduce frame shift mutations to generate gene knockout hPSC lines
2. Correction of disease-associated point mutations in patient-specific iPSCs
3. Introduction of disease-associated point mutations to control iPSCs
4. Introduction of reporter cassettes to monitor transcriptional activity of endogenous genes
5. Introduction of genetic elements to the AAVS1 safe-harbor in hPSCs
- The hPSC gene editing service is available to both CCHMC/UC and external investigators.
- Gene editing projects are usually complete within 3-4 months. However, each project is different and occasionally delays are experienced. See an overview of a typical workflow below.