United Therapeutics Corporation
GENETICALLY-ENGINEERED MESENCHYMAL STEM CELLS OVEREXPRESSING AOAH AND USES THEREOF

Abstract:

Genetically-modified mesenchymal stem cells (MSCs) overexpressing an acyloxyacyl hydrolase (AOAH) polypeptide, such as MSCs comprising a recombinant nucleic acid encoding the AOAH polypeptide, are described. The MSCs may further overexpress a second polypeptide of interest, such as angiopoeitin-1 (ANGPT1). Pharmaceutical compositions comprising the genetically-modified MSCs are also described. The use of such genetically-modified MSCs and/or pharmaceutical compositions comprising same as a medicament, for example for the treatment of systemic inflammatory response syndrome (SIRS) or sepsis, is also described.