High-throughput screening for modulators of <i>ACVR1</i> transcription: discovery of potential therapeutics for fibrodysplasia ossificans progressiva

Abstract excerpt

The ACVR1 gene encodes a type I receptor of bone morphogenetic proteins (BMPs). Activating mutations in ACVR1 are responsible for fibrodysplasia ossificans progressiva (FOP), a rare disease characterized by congenital toe malformation and progressive heterotopic endochondral ossification leading to severe and cumulative disability. Until now, no therapy has been available to prevent soft-tissue swelling...

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