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When one skeleton is enough: approaches and strategies for the treatment of fibrodysplasia ossificans progressiva (FOP).


ABSTRACT: A heterozygous missense mutation in activin receptor IA/activin-like kinase-2 (ACVR1/ALK2), a bone morphogenetic protein (BMP) type I receptor, is responsible for fibrodysplasia ossificans progressiva (FOP), the most catastrophic disorder of skeletal metamorphosis in humans. The discovery of the FOP gene establishes a crucial milestone in understanding FOP, reveals a highly conserved target in the BMP signaling pathway for drug development and specifically stimulates therapeutic approaches for the development of inhibitors for ACVR1/ALK2 signaling. Effective therapies for FOP, and possibly for more common conditions of heterotopic ossification, will be based on interventions that selectively block promiscuous ACVR1/ALK2 signaling, and/or themolecular triggers, responding cells and tissue microenvironments that facilitate aberrant skeletal metamorphosis in a permissive genetic background of increased BMP pathway activity.

SUBMITTER: Kaplan FS 

PROVIDER: S-EPMC3627400 | biostudies-literature | 2008

REPOSITORIES: biostudies-literature

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When one skeleton is enough: approaches and strategies for the treatment of fibrodysplasia ossificans progressiva (FOP).

Kaplan Frederick S FS   Groppe Jay J   Shore Eileen M EM  

Drug discovery today. Therapeutic strategies 20080101 4


A heterozygous missense mutation in activin receptor IA/activin-like kinase-2 (ACVR1/ALK2), a bone morphogenetic protein (BMP) type I receptor, is responsible for fibrodysplasia ossificans progressiva (FOP), the most catastrophic disorder of skeletal metamorphosis in humans. The discovery of the FOP gene establishes a crucial milestone in understanding FOP, reveals a highly conserved target in the BMP signaling pathway for drug development and specifically stimulates therapeutic approaches for t  ...[more]

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