Specifically, antisense oligonucleotides, including isotersen [198] and eplontersen [199], along with siRNA agents, like patisiran [132] and vutrisiran [38], mediate mRNA silencing to prevent the pathological accumulation of misfolded transthyretin (TTR) aggregates in cardiac and neural tissues, demonstrating therapeutic efficacy in transthyretin amyloidosis. The gene discussed is TTR; the disease is amyloidosis.