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.
CITATION STYLE
Kim, Y., Jeong, J., & Choi, D. (2023). Ex Vivo Base Editing Therapy with Chemically Derived Hepatic Progenitors. In Methods in Molecular Biology (Vol. 2606, pp. 171–178). Humana Press Inc. https://doi.org/10.1007/978-1-0716-2879-9_13
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