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Expression of herpes simplex virus (HSV) thymidine kinase in human embryonic stem cells (hESCs) as a strategy to prevent teratoma formation

In vitro and mouse studies identified a strategy to prevent teratoma formation in hESC-derived cellular therapies. hESCs were engineered to express HSV thymidine kinase, which is not expressed by differentiated cells or healthy human cells. In culture, the thymidine kinase inhibitor Zirgan ganciclovir induced death of engineered hESCs but not differentiated cells derived from the engineered hESCs. In mice transplanted with the engineered hESCs or partially differentiated engineered hESCs, ganciclovir prevented teratoma formation whereas saline did not. Next steps include testing the strategy in disease models.
Laboratoires Thea S.A. markets Zirgan to treat HSV infection.

SciBX 5(33); doi:10.1038/scibx.2012.883
Published online Aug. 23, 2012

Findings unpatented; available for licensing

Rong, Z. et al. J. Biol. Chem.;

published online Aug. 4, 2012;
Contact: Yang Xu, University of California, San Diego, La Jolla, Calif.