UW Human Stem Cell Gene Editing Service at the Waisman Center

For all their promise, human pluripotent stem cells have issues that hinder research progress in the field. The inherent variability of human beings compared to model organisms results in a great deal of variation in the pluripotent stem cells and makes it difficult to identify robust phenotypes in these cells that can be ascribed to disease.

The “UW Human Stem Cell Editing Service”, located in the Waisman Center will provide CRISPR-Cas9 gene editing of human pluripotent stem cells (PSCs) to campus researchers. Gene editing enables the creation of isogenic human PSC lines by correcting the gene mutation in the patient iPSCs or by knocking a mutation into otherwise normal human PSCs. The isogenic cells have the identical genetic background except for the mutation, thus providing stringent controls and avoiding much of the variability inherent in iPSCs, which will greatly enhance our ability to understand how those mutations cause particular diseases. In addition to using gene editing to correct or establish mutations in hPSCs, new technologies enable strategies to regulate gene expression, delete genes and insert tags that allow rapid cell sorting.

Creating a stem cell gene-editing facility will enhance the ease and speed of many aspects of biological research on campus. The new editing core will provide technical services, including generation of genome edited human stem cells lines, quality control of genome edited cells, and training for lab personnel.