TTR and poikiloderma with neutropenia: Notably, (i) NTLA-2001, an intravenously administered lipid nanoparticle formulation encapsulating Cas9 messenger RNA (mRNA) and sgRNA targeting transthyretin (TTR), has been studied in a phase 1 clinical trial since 2020 for hereditary TTR amyloidosis with polyneuropathy (ATTRv-PN) and TTR amyloidosis-related cardiomyopathy (ATTR-CM) (NCT04601051).