Linking a cell-division gene and a suicide gene to define and improve cell therapy safety

Nature. 2018 Nov;563(7733):701-704. doi: 10.1038/s41586-018-0733-7. Epub 2018 Nov 14.

Abstract

Human pluripotent cell lines hold enormous promise for the development of cell-based therapies. Safety, however, is a crucial prerequisite condition for clinical applications. Numerous groups have attempted to eliminate potentially harmful cells through the use of suicide genes1, but none has quantitatively defined the safety level of transplant therapies. Here, using genome-engineering strategies, we demonstrate the protection of a suicide system from inactivation in dividing cells. We created a transcriptional link between the suicide gene herpes simplex virus thymidine kinase (HSV-TK) and a cell-division gene (CDK1); this combination is designated the safe-cell system. Furthermore, we used a mathematical model to quantify the safety level of the cell therapy as a function of the number of cells that is needed for the therapy and the type of genome editing that is performed. Even with the highly conservative estimates described here, we anticipate that our solution will rapidly accelerate the entry of cell-based medicine into the clinic.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • CDC2 Protein Kinase / genetics*
  • Cell Division / genetics*
  • Cell Proliferation
  • Cell- and Tissue-Based Therapy / methods*
  • Cell- and Tissue-Based Therapy / standards
  • Embryonic Stem Cells / cytology
  • Embryonic Stem Cells / metabolism
  • Female
  • Ganciclovir / pharmacology
  • Genes, Transgenic, Suicide / genetics*
  • Humans
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Patient Safety*
  • Simplexvirus / enzymology
  • Simplexvirus / genetics
  • Thymidine Kinase / genetics
  • Thymidine Kinase / metabolism

Substances

  • Thymidine Kinase
  • CDC2 Protein Kinase
  • CDK1 protein, human
  • Ganciclovir