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Activin Receptor Type I (ACVR1), also known as ALK2, is a transmembrane serine/threonine kinase receptor belonging to the bone morphogenetic protein (BMP) type I receptor family and the transforming growth factor-beta (TGF-β) superfamily. It plays a crucial role in the BMP signaling pathway, regulating skeletal development, cell proliferation, differentiation, apoptosis, adhesion, and migration. Upon ligand binding, ACVR1 forms heteromeric complexes with type II receptors, leading to the phosphorylation and activation of Smad proteins, which then regulate gene transcription. Mutations in ACVR1 are associated with diseases such as Fibrodysplasia Ossificans Progressiva (FOP) and Diffuse Intrinsic Pontine Glioma. ACVR1 is considered an important drug target for conditions involving abnormal bone formation or certain cancers related to the TGF-beta/BMP pathway.
Inhibition of ACVR1/ALK2 kinase activity, prevention of ligand binding, or disruption of Smad signaling
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