The Translational Genome Editing lab, led by Dr. Tobias Raabe, Ph.D. is focused on:

  • Establishing robust CRISPR-mediated methods for the genetic manipulation of human-induced pluripotent stem cells (hiPSC) and of the newly discovered human liver-derived ductal stem cells
  • The functional study of cardiovascular and liver disease associated gene mutations using induced hepatocytes derived from human liver-derived ductal stem cells
  • The use of human liver-derived ductal stem cells for liver regenerative medicine and liver gene therapy

We are especially interested in isolating and characterizing adult liver-derived ductal stem cells, which are bi-potent cells that can give rise to either bile ducts or to hepatocytes, depending on their ex vivo or in vivo environment. We use special culture conditions that allow derivation of these resident ductal stem cells from adult mouse and, importantly, human liver without artificial transgene-based immortalization. These culture conditions allow for continuous 3D culture of the ductal stem cells for more than one year with a 10-fold higher genetic stability compared to iPSC. The human liver derived ductal stem cells are physiologically and genetically virtually identical to their precursors in the parental liver tissue and thus may yield more patient-relevant data compared to other cell types that have been traditionally used for the study of liver genetics, metabolism and disease.

In addition, the Translational Genome Editing Program has several collaborations on campus, including research into cardiovascular disease, Alzheimer’s disease and schizophrenia.

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