Several notable examples have received clinical approval, including 2′-O-MOE gapmers (Figure 2b) targeting apolipoprotein B-100 (ApoB-100) mRNA for the treatment of homozygous familial hypercholesterolemia (mipomersen), transthyretin (TTR) mRNA in hereditary transthyretin-mediated amyloidosis (inotersen and eplontersen), apolipoprotein CIII (Apo-CIII) mRNA in familial chylomicronemia syndrome (volanesorsen and olezarsen), and superoxide dismutase 1 (SOD1) mRNA in amyotrophic lateral sclerosis (tofersen) [104,105,106,107,108,109]. This evidence concerns the gene TTR and amyotrophic lateral sclerosis.