A Novel Genetic Approach to inhibit or Eliminate the Tumorgenicity of Human Embryonic Stem Cells and/or their Derivatives After Transplantation

Pluripotent embryonic stem cells have enormous potential for providing an unlimited supply of multipotent and specialized cells, such as brain and heart cells, for transplantation and cell-based therapies that are otherwise limited by donor availability. However, this promising application is hampered by concerns that embryonic stem cells or their multipotent derivatives also possess the potential to form malignant tumors after transplantation in vivo. JHU researchers have conceived a novel method for modifying embryonic stem cells in order to provide a means for inhibiting/preventing the potential for tumor formation by undifferentiated stem cells or multipotent derivatives after transplantation. The invention involves genetically modifying the stem cells ex vivo prior to implantation. Unlike gene-based approaches, this ex vivo approach enables the isolation of clonal genetically-modified cell lines whose transgene location has been characterized to minimize the risk of inappropriate gene insertion and potential oncogenesis. Description (Set) Proposed Use (Set) This technology provides a method to reduce the potential for tumor formation by stem cell based cellular therapies after transplantation. The same approaches can be applied to engineer other multipotent stem cells as well as their derivatives to inhibit/prevent their tumorgenic potential.

Inventor(s): Li, Ronald A.

Type of Offer: Licensing



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