After siRNAs encapsulated in lipid nanoparticles were shown to successfully induce transthyretin knockdown in patients with TTR amyloidosis in a phase 1 study (Coelho et al., 2013), a subsequent phase 2 study evaluated patisiran as a potential therapeutic strategy in TTR mediated familial amyloidotic polyneuropathy (Suhr et al., 2015). This evidence concerns the gene TTR and amyloidosis.