Abstract

An exceptional safety profile has been shown in a large number of clinical trials based on the use of mesenchymal stromal cells (MSCs) that have been performed worldwide. Consequently, MSCs are now approved by regulatory agencies in Europe, Japan, and US as treatment for pediatric Graft-versus-Host Disease and Crohn’s fistular disease. Nevertheless, reliable potency assays to predict MSC immunosuppressive efficacy in the clinical setting are still lacking. We discuss potential mechanisms of action for therapeutic effects of MSC transplantation, discuss experiemental models used to dissect tissue modulating function of MSCs, and propose approaches for identifying MSC functional effects in vivo.

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