Article ; Online: Recent progress in drug development for fibrodysplasia ossificans progressiva.
Molecular and cellular biochemistry
2022 Volume 477, Issue 10, Page(s) 2327–2334
Abstract: Fibrodysplasia Ossificans Progressiva (FOP) is a rare genetic disease caused by heterozygous missense mutations in Activin A receptor type I which is also known as Activin-like kinase 2 (ALK2), a type I receptor of Bone Morphogenetic Proteins(BMP). ... ...
Abstract | Fibrodysplasia Ossificans Progressiva (FOP) is a rare genetic disease caused by heterozygous missense mutations in Activin A receptor type I which is also known as Activin-like kinase 2 (ALK2), a type I receptor of Bone Morphogenetic Proteins(BMP). Patients with FOP usually undergo episodic flare-ups and the heterotopic ossification in soft and connective tissues. Molecular mechanism study indicates that Activin A, the ligand which normally transduces Transforming Growth Factor Beta signaling, abnormally activates BMP signaling through ALK2 mutants in FOP, leading to heterotopic bone formation. To date, effective therapies to FOP are unavailable. However, significant advances have recently been made in the development of FOP drugs. In this article, we review the recent advances in understanding the FOP mechanism and drug development, with a focus on the small-molecular and antibody drugs currently in the clinical trials for FOP treatment. |
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MeSH term(s) | Activins/genetics ; Activins/metabolism ; Bone Morphogenetic Proteins/metabolism ; Drug Development ; Humans ; Ligands ; Mutation ; Myositis Ossificans/drug therapy ; Myositis Ossificans/genetics ; Myositis Ossificans/metabolism ; Ossification, Heterotopic/genetics ; Ossification, Heterotopic/metabolism ; Transforming Growth Factor beta/genetics |
Chemical Substances | Bone Morphogenetic Proteins ; Ligands ; Transforming Growth Factor beta ; Activins (104625-48-1) |
Language | English |
Publishing date | 2022-05-10 |
Publishing country | Netherlands |
Document type | Journal Article ; Review |
ZDB-ID | 184833-1 |
ISSN | 1573-4919 ; 0300-8177 |
ISSN (online) | 1573-4919 |
ISSN | 0300-8177 |
DOI | 10.1007/s11010-022-04446-9 |
Database | MEDical Literature Analysis and Retrieval System OnLINE |
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