Unraveling the role of Adiponectin-expressing bone marrow stromal cells in the cellular pathogenesis of Fibrous Dysplasia

Awardee: Biagio Palmisano

Institution: Sapienza University of Rome

Grant Amount: $53,791

Funding Period: February 1, 2022 - January 31, 2023


Summary:

We have previously shown that osteoclasts, the cells that normally destroy damaged bone to allow its regeneration, play a major role in the appearance and evolution of Fibrous Dysplasia (FD). We know that in growing FD lesions, the number of osteoclasts is abnormally high due to the production of a factor named RANKL by the pathological tissue. However, what we do not know yet is who produces RANKL at the very beginning of the disease, when osteoclasts destroy the healthy bone that will be then replaced by the pathological tissue. Recently, by generating a new Gs(alpha) transgenic mouse model, we have identified the cell type that is involved in this early phase of the disease. In this project, we want to investigate the characteristics of this cell type and the mechanisms through which it produces RANKL, both in the absence and in the presence of the Gs(alpha) mutation. Understanding these points may allow the development of therapies that act specifically on the very first trigger of FD lesions.

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Structure activity relationship studies of compounds to treat FD/MAS

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Reversing Brain Iron Overload in BPAN by a Natural Small Molecule