Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1R206H Transcript and Inhibit Osteogenic Differentiation.
Nucleic acid therapeutics（2022）
关键词
LNA,FOP,ACVR1,fibrodysplasia ossificans progressiva,antisense,locked nucleic acids
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