Spatial transcriptomics to elucidate the mechanism of anti-RANKL inhibition of fibrous dysplasia bone lesions

MDBRFDMAS
Written By Million Dollar Bike Ride

Awardee: Julia Charles

Institution: Brigham and Women's Hospital

Grant Amount: $40,321.00

Funding Period: February 1, 2024 - January 31, 2025

Summary:

Fibrous dysplasia causes fibrotic bone lesions, full of immature bone forming osteoblasts, that result in pain, deformity and fracture susceptibility. Antibody that blocks the cytokine RANKL inhibits formation of bone eroding osteoclast cells and also improves bone lesions in fibrous dysplasia, but how this works is not known. We propose to profile what both mutant and bystander wild-type cells are producing before and after RANKL is blocked to try to understand how this treatment works. This is important because blocking RANKL has side effects that limit use and understanding how it improves bone lesions could lead to developing alternative therapies.

Million Dollar Bike Ride
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Testing the specificity and efficacy of compounds that inhibit cAMP signaling, for the development of potential therapeutics for FD/MAS
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Probing Central and Peripheral Mechanisms of Pain and Dysfunction in Fibrous Dysplasia/McCune-Albright