Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressiva.

Fibrodysplasia ossificans progressiva (FOP) is a rare heritable disease characterized by progressive heterotopic ossification of connective tissues, for which there is presently no definite treatment. A recurrent activating mutation (c.617G→A; R206H) of activin receptor-like kinase 2 (ACVR1/ALK2), a...

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Main Authors: Songting Shi, Jie Cai, David J J de Gorter, Gonzalo Sanchez-Duffhues, Dwi U Kemaladewi, Willem M H Hoogaars, Annemieke Aartsma-Rus, Peter A C 't Hoen, Peter ten Dijke
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2013-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3701692?pdf=render