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Restoration of normal BMP signaling levels and osteogenic differentiation in FOP mesenchymal progenitor cells by mutant allele-specific targeting

Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal dominant disorder of progressive heterotopic ossification for which there is presently no cure. FOP is caused by a recurrent heterozygous activating mutation (c.617G>A; R206H) of Activin receptor type IA/Activin-like kinase-2 (ACVR1/...

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Hlavní autoři: Kaplan, Josef, Kaplan, Frederick S., Shore, Eileen M.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2011
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3390458/
https://ncbi.nlm.nih.gov/pubmed/22011642
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2011.152
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