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Article

Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva.

Methods in molecular biology (Clifton, N.J.) - 1 Jan 2025

Maruyama Rika, Yokota Toshifumi

Abstract excerpt

Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal-dominant disorder characterized by progressive heterotopic ossification. More than 95% of cases are caused by a recurrent mutation (617G > A; R206H) of ACVR1/ALK2, a bone morphogenetic protein (BMP) type I receptor. Recent studies revealed that ACVR1R206H induces heterotopic ossification by aberrant activation in response to Activin A. Because...

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PMID: 40720038
DOI: 10.1007/978-1-0716-4730-1_31

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ACVR1

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