Gaucher HMSCS present an altered osteoblast and adipose differentiation.

ANDREA CRIVARO; CONSTANZA BONDAR; JUAN MARCOS MUCCI; M. VICTORIA DELPINO; PAULA ROZENFELD

Congreso; LXVI Reunión Anual de la Sociedad Argentina de Inmunología (SAI). 14-17 Noviembre 2018.; 2018

Gaucher disease (GD) is caused by mutations on the gene encodingthe lysosomal enzyme glucocerebrosidase. Type I GD (GD1) patientspresent diverse symptoms as chronic inflammation, anemia,hepatosplenomegaly and bone alterations. The most widespreadtreatment for GD, enzyme replacement therapy (ERT), cannot completelyreverse bone problems. Despite mechanisms leading to bonedamage are not fully described, reports suggest that alterations inosteoblasts and osteoclast, as well as a chronic pro-inflammatorystate, could be involved. It is known that mesenchymal stem cells(MSCs) differentiate to osteoblasts and adipocytes, so the aim ofthis work was to evaluate, in an in vitro model, the potential of MSCsfrom control and GD patients to differentiate towards the osteoblast(GDOb) and adipocyte (GDAd) lineage. Furthermore, we sought toanalyze released cytokines from differentiated osteoblasts and thecapacity of these cells to generate osteoclasts. The expression ofdifferentiation markers were lower in GDOb at 14 days compared tocontrol Ob: BMP-2 (p=0,001), Runx2 (p=0,01), ALP (p