Gene-modified mesenchymal stem cells can serve as potent delivery vehicles but there are key safety concerns from the use of integrating vectors, which must be considered when moving toward clinical trials.

The future use of human embryonic or induced pluripotent stem cells as a source for producing MSCs would allow gene targeting through homologous recombination, or random integration and selection of those pluripotent clones that had the transgene integrated into a “safe harbor” site. The appropriate, selected pluripotent stem cell clones could be expanded greatly prior to differentiation to MSCs. We have demonstrated that human embryonic stem cell – derived MSCs home to sites of ischemia and tissue damage, and behave much like thier adult counterparts.

The current manuscript describes progress toward producing functional MSCs from hESC and iPSCs, and the future potential for this field.