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The therapy for this ultra-rare disease uses a similar strategy to Intellias liver-targeting treatments for hATTR and HAE: it consists of a Cas12i2 nuclease and guide RNA encapsulated in a lipid nanoparticle (LNP)

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Here, we confirmed that these MORF2s were localized in chloroplasts (Supplementary Fig

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This moderate pressor effect is likely due to its nitric oxide scavenging effects 2 and this drug is sometimes used as an offlabel treatment for vasoplegic syndrome 3