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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Autores principales: Shi, SongTing, Cai, Jie, de Gorter, David J. J., Sanchez-Duffhues, Gonzalo, Kemaladewi, Dwi U., Hoogaars, Willem M. H., Aartsma-Rus, Annemieke, ’t Hoen, Peter A. C., ten Dijke, Peter
Formato: Artigo
Lenguaje:Inglês
Publicado: Public Library of Science 2013
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Research Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3701692/
https://ncbi.nlm.nih.gov/pubmed/23861958
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0069096
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