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In contrast, ATTR-CM, which arises from gain-of-function mutations, 260 has been the focus of all three therapeutic approaches, with multiple candidates already in clinical development (Fig

10.1038/ng.2802 Nat Genet 65 LambrechtsD.StorkebaumE.MorimotoM.Del-FaveroJ.DesmetF.MarklundS

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Antisense oligonucleotide (ASO) and RNA interference (RNAi) now enable precise gene silencing with high specificity and enhanced stability

Hence, MTs impact on cellular proliferation could be more pronounced in TRIP13 KO cells because these cells might have an already destabilized genomic state, making them more susceptible to the effects of MT

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