290 Strategies to improve in vivo survival of Mesenchymal Stromal Cells after injection in immunodeficient mice

Recessive dystrophic epidermolysis bullosa (RDEB) is one of the most severe skin diseases and caused by loss-of-function mutations in COL7A1 encoding type VII collagen (C7). Patients with RDEB suffer since birth from mucosal blistering develop local systemic complications resulting a poor prognosis. Mesenchymal stromal cells (MSC) have been shown to therapeutic potential improve wound healing inflammation patients due their capacity express C7 anti-inflammatory properties. Clinical trials transient improvement allogeneic MSC derived healthy donor’s bone marrow (BM-MSC). The aim this work optimize BM-MSC vitro conditioning prior injection murine models survival mucosae. We first transduced human fluorescent bioluminescent reporter lentiviral vector an efficiency 60% without modifying phenotype. Transduced were cultured under hypoxic or normoxic conditions supplemented either platelet lysate foetal calf serum. then injected subcutaneously (SC) intravenously (IV) immunodeficient mice. behaviour upon administration mice was assessed using means vivo imaging. After SC BM-MSC, signal remained localized site for at least 7 months (ongoing monitoring). When IV, bioluminescence not detected after 10 days. optimization routes biodistribution as imaging should contribute efficient persistent treatment wounded mucosae perspective clinical translation RDEB.