Ex Vivo Base Editing Therapy with Chemically Derived Hepatic Progenitors

Abstract

Ex vivo gene therapy through convergence study with progenitors and base/prime editors provides valuable approaches that can be utilized in the study and treatment of hereditary intractable diseases and models. Small molecule-mediated reprogramming of hepatocytes into bi-potent hepatic progenitors is a safe and efficient strategy for ex vivo gene therapy. Here, we described how to generate hepatic progenitors from terminally differentiated hepatocytes, deliver base/prime editors into the cells, select corrected hepatic progenitors, and transplant them into mice of inborn error of metabolism.

Keywords: Base editors; Chemically derived hepatic progenitors (CdHs); Ex vivo gene therapy; Liver progenitors; Regenerative medicine; Transplantation.